<clinical_study>
  <!-- This xml conforms to an XML Schema at:
    https://clinicaltrials.gov/ct2/html/images/info/public.xsd -->
  <required_header>
    <download_date>ClinicalTrials.gov processed this data on September 20, 2023</download_date>
    <link_text>Link to the current ClinicalTrials.gov record.</link_text>
    <url>https://clinicaltrials.gov/ct2/show/NCT03039686</url>
  </required_header>
  <id_info>
    <org_study_id>CN001-016</org_study_id>
    <secondary_id>2016-001654-18</secondary_id>
    <secondary_id>WN40227</secondary_id>
    <nct_id>NCT03039686</nct_id>
  </id_info>
  <brief_title>Clinical Trial to Evaluate the Efficacy, Safety, and Tolerability of RO7239361 in Ambulatory Boys With Duchenne Muscular Dystrophy</brief_title>
  <official_title>A Randomized, Double Blind, Placebo-Controlled, Study to Assess the Efficacy, Safety, and Tolerability of RO7239361 in Ambulatory Boys With Duchenne Muscular Dystrophy</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>Hoffmann-La Roche</agency>
      <agency_class>Industry</agency_class>
    </lead_sponsor>
  </sponsors>
  <source>Hoffmann-La Roche</source>
  <oversight_info>
    <has_dmc>Yes</has_dmc>
    <is_fda_regulated_drug>Yes</is_fda_regulated_drug>
    <is_fda_regulated_device>No</is_fda_regulated_device>
  </oversight_info>
  <brief_summary>
    <textblock>
      This is a multi-center, randomized, double-blind, placebo-controlled study to assess the&#xD;
      efficacy, safety and tolerability of two different weekly doses of RO7239361 in ambulatory&#xD;
      boys with Duchenne Muscular Dystrophy (DMD).&#xD;
    </textblock>
  </brief_summary>
  <overall_status>Completed</overall_status>
  <start_date type="Actual">July 6, 2017</start_date>
  <completion_date type="Actual">April 28, 2020</completion_date>
  <primary_completion_date type="Actual">April 28, 2020</primary_completion_date>
  <phase>Phase 2/Phase 3</phase>
  <study_type>Interventional</study_type>
  <has_expanded_access>No</has_expanded_access>
  <study_design_info>
    <allocation>Randomized</allocation>
    <intervention_model>Parallel Assignment</intervention_model>
    <primary_purpose>Treatment</primary_purpose>
    <masking>Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)</masking>
  </study_design_info>
  <primary_outcome>
    <measure>Baseline for the North Star Ambulatory Assessment (NSAA) Total Score</measure>
    <time_frame>Baseline</time_frame>
    <description>The NSAA is a functional scale specifically designed for ambulant boys with Duchenne muscular dystrophy (DMD) that can provide information about motor function. The NSAA is a 17-item test of standing, ability to transition from lying to sitting, sitting to standing, and other mobility assessments. Each of the 17 items is evaluated on an ordinal scale of 0-2: 0 = unable to achieve independently, 1 = modified method but achieves goal independent of physical assistance from another, or 2 = normal with no obvious modification of activity. Total score range is 0 to 34. Higher scores reflect better performance.</description>
  </primary_outcome>
  <primary_outcome>
    <measure>Change From Baseline in the North Star Ambulatory Assessment (NSAA) Total Score at Week 48</measure>
    <time_frame>Baseline, Week 48</time_frame>
    <description>The NSAA is a functional scale specifically designed for ambulant boys with Duchenne muscular dystrophy (DMD) that can provide information about motor function. The NSAA is a 17-item test of standing, ability to transition from lying to sitting, sitting to standing, and other mobility assessments. Each of the 17 items is evaluated on an ordinal scale of 0-2: 0 = unable to achieve independently, 1 = modified method but achieves goal independent of physical assistance from another, or 2 = normal with no obvious modification of activity. Total score range is 0 to 34. Higher scores reflect better performance. A positive change from baseline indicates an improvement. Based on the mixed-effect model of repeated measures (MMRM).</description>
  </primary_outcome>
  <secondary_outcome>
    <measure>Baseline Time for 4 Stair Climb</measure>
    <time_frame>Baseline</time_frame>
    <description>The time to complete the 4 stair climb was measured at baseline.</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Change From Baseline at Week 48 in 4 Stair Climb Velocity (4SCV)</measure>
    <time_frame>Baseline, Week 48</time_frame>
    <description>4SCV was calculated as the ratio of the number of stairs climbed (4) divided by the number of seconds taken to complete the 4-stair climb. The results were converted into velocity (distance/time). A positive change from baseline indicates an improvement. Based on the mixed-effect model of repeated measures (MMRM).</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Baseline for the Time to Stand From Supine</measure>
    <time_frame>Baseline</time_frame>
    <description>The time required for a participant to stand from supine position. A longer time reflects a worse outcome.</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Change From Baseline at Week 48 in Stand From Supine Velocity</measure>
    <time_frame>Baseline, Week 48</time_frame>
    <description>The time required for a participant to stand from supine position. A longer time reflects a worse outcome. A negative change from baseline indicates an improvement. Based on the mixed-effect model of repeated measures (MMRM).</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Baseline Time for 10 Meter Walk/Run</measure>
    <time_frame>Baseline</time_frame>
    <description>The time required for a participant to run or walk a distance of 10 meters as quickly as possible. A longer time reflects a worse outcome.</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Change From Baseline at Week 48 in 10 M Walk/Run Velocity</measure>
    <time_frame>Baseline, Week 48</time_frame>
    <description>The time required for a participant to run or walk a distance of 10 meters as quickly as possible calculated as velocity (distance/time). A positive change from baseline indicates an improvement. Based on the mixed-effect model of repeated measures (MMRM).</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Baseline for the Pediatric Outcome Data Collection Instrument (PODCI) Transfer and Basic Mobility Subscale</measure>
    <time_frame>Baseline</time_frame>
    <description>The PODCI is designed to be completed by the parent/guardian of a child who has knowledge of the child's conditions. The Transfer and Basic Mobility scale is one of the subscales of the PODCI. The results are standardized into a scale of 0-100 with a higher score reflecting better performance.</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Change From Baseline at Week 48 in Pediatric Outcome Data Collection Instrument (PODCI) Transfer and Basic Mobility Subscale</measure>
    <time_frame>Baseline, Week 48</time_frame>
    <description>The PODCI is designed to be completed by the parent/guardian of a child who has knowledge of the child's conditions. The Transfer and Basic Mobility scale is one of the subscales of the PODCI. The results are standardized into a scale of 0-100 with a higher score reflecting better performance. A positive change from baseline indicates an improvement. Based on the mixed-effect model of repeated measures (MMRM).</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Change From Baseline at Week 48 in Proximal Lower Extremity Flexor Strength</measure>
    <time_frame>Baseline, Week 48</time_frame>
    <description>Proximal lower extremity flexor (knee extension and knee flexion) strength was measured using manual myometry. A higher score reflects a better outcome. A positive change from baseline indicates an improvement.</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Baseline for the 6 Minute Walk Distance (6MWD)</measure>
    <time_frame>Baseline</time_frame>
    <description>The 6MWD measured the distance a participant was able to traverse while walking for 6 minutes. A longer distance reflects a better outcome.</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Change From Baseline at Week 48 in 6 Minute Walk Distance (6MWD)</measure>
    <time_frame>Baseline, Week 48</time_frame>
    <description>The 6MWD measured the distance a participant was able to traverse while walking for 6 minutes. A longer distance reflects a better outcome. A positive change from baseline indicates an improvement. Based on the mixed-effect model of repeated measures (MMRM).</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Percentage of Participants for Each Clinical Global Impression of Change (CGI-C) Assessment Status at Week 48</measure>
    <time_frame>Baseline, Week 48</time_frame>
    <description>The CGI-C was used to assess the participant's overall condition on a 7-point scale, using the status markers &quot;very much improved, much improved, slightly improved, no change, slightly worse, much worse or very much worse&quot; at Week 48 as compared to baseline.</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Change From Baseline at Week 48 in 95th Percentile Stride Velocity</measure>
    <time_frame>Baseline, Week 48</time_frame>
    <description>Stride velocity was recorded with the ActiMyo device in a subset of the overall study population. The ActiMyo device measures the daily movement and activity levels of the participant. The device consists of two sensors worn on each ankle. A higher velocity reflects a better outcome. A positive change from baseline indicates an improvement.</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Number of Participants With Adverse Events (AEs)</measure>
    <time_frame>During DB period (48 weeks) and Whole study (up to approximately 34 months)</time_frame>
    <description>An adverse event is any untoward medical occurrence in a subject administered a pharmaceutical product and which does not necessarily have to have a causal relationship with the treatment. An adverse event can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding, for example), symptom, or disease temporally associated with the use of a pharmaceutical product, whether or not considered related to the pharmaceutical product. Preexisting conditions which worsen during a study are also considered as adverse events.</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Number of Participants With AEs Leading to Discontinuation</measure>
    <time_frame>During DB period (48 weeks) and Whole study (up to approximately 34 months)</time_frame>
    <description>An adverse event is any untoward medical occurrence in a subject administered a pharmaceutical product and which does not necessarily have to have a causal relationship with the treatment. An adverse event can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding, for example), symptom, or disease temporally associated with the use of a pharmaceutical product, whether or not considered related to the pharmaceutical product. Preexisting conditions which worsen during a study are also considered as adverse events. Reported here is the number of participants with AEs that led to study discontinuation.</description>
  </secondary_outcome>
  <number_of_arms>3</number_of_arms>
  <enrollment type="Actual">166</enrollment>
  <condition>Duchenne Muscular Dystrophy</condition>
  <arm_group>
    <arm_group_label>RO7239361 Low Dose</arm_group_label>
    <arm_group_type>Experimental</arm_group_type>
    <description>Participants received low dose RO7239361 SC on specified days of the 48-week DB period. Following the DB period participants received low dose RO7239361 on specified days for up to 192 weeks during the open-label period followed by 24 weeks of follow-up.</description>
  </arm_group>
  <arm_group>
    <arm_group_label>RO7239361 High Dose</arm_group_label>
    <arm_group_type>Experimental</arm_group_type>
    <description>Participants received high dose RO7239361 SC on specified days of the 48-week DB period. Following the DB period participants received high dose RO7239361 on specified days for up to 192 weeks during the open-label period followed by 24 weeks of follow-up.</description>
  </arm_group>
  <arm_group>
    <arm_group_label>Placebo</arm_group_label>
    <arm_group_type>Placebo Comparator</arm_group_type>
    <description>Participants received matching placebo solution subcutaneously (SC) on specified days of the 48-week double-blind (DB) period. Following the DB period participants received low dose or high dose RO7239361 on specified days for up to 192 weeks during the open-label period followed by 24 weeks of follow-up.</description>
  </arm_group>
  <intervention>
    <intervention_type>Drug</intervention_type>
    <intervention_name>RO7239361</intervention_name>
    <description>Take RO7239361 subcutaneously on specified days over a 48 week blinded period</description>
    <arm_group_label>RO7239361 High Dose</arm_group_label>
    <arm_group_label>RO7239361 Low Dose</arm_group_label>
  </intervention>
  <intervention>
    <intervention_type>Drug</intervention_type>
    <intervention_name>Placebo for RO7239361</intervention_name>
    <description>Take placebo subcutaneously on specified days over a 48 week blinded period</description>
    <arm_group_label>Placebo</arm_group_label>
  </intervention>
  <eligibility>
    <criteria>
      <textblock>
        Inclusion Criteria:&#xD;
&#xD;
          -  Diagnosed with DMD by confirmed medical history and genetic testing&#xD;
&#xD;
          -  Able to walk without assistance&#xD;
&#xD;
          -  Minimum North Star Ambulatory Assessment score of 15 at screening&#xD;
&#xD;
          -  Able to walk up 4 stairs in 8 seconds or less&#xD;
&#xD;
          -  Weigh at least 15 kg (33 lbs)&#xD;
&#xD;
          -  Taking corticosteroids for DMD&#xD;
&#xD;
        Exclusion Criteria:&#xD;
&#xD;
          -  Any behavior or mental issue that will affect the ability to complete the required&#xD;
             study procedures&#xD;
&#xD;
          -  Previously or currently taking medications like androgens or human growth hormone&#xD;
&#xD;
          -  Use of a ventilator during the day&#xD;
&#xD;
          -  Unable to have blood samples collected or receive an injection under the skin&#xD;
&#xD;
          -  Concomitant or previous participation at any time in a gene therapy study&#xD;
&#xD;
        Other protocol defined Inclusion/Exclusion Criteria could apply.&#xD;
      </textblock>
    </criteria>
    <gender>Male</gender>
    <minimum_age>6 Years</minimum_age>
    <maximum_age>11 Years</maximum_age>
    <healthy_volunteers>No</healthy_volunteers>
  </eligibility>
  <overall_official>
    <last_name>Clinical Trials</last_name>
    <role>Study Director</role>
    <affiliation>Hoffmann-La Roche</affiliation>
  </overall_official>
  <location>
    <facility>
      <name>Neuromuscular Research Center</name>
      <address>
        <city>Phoenix</city>
        <state>Arizona</state>
        <zip>85028</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Stanford University</name>
      <address>
        <city>Palo Alto</city>
        <state>California</state>
        <zip>94304</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>University of California Davis Medical Center</name>
      <address>
        <city>Sacramento</city>
        <state>California</state>
        <zip>95817</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Yale University School of Medicine ; Pulmonary &amp; Critical Care</name>
      <address>
        <city>New Haven</city>
        <state>Connecticut</state>
        <zip>06510</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>University of Florida</name>
      <address>
        <city>Gainesville</city>
        <state>Florida</state>
        <zip>32607</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Nemours Children's Hospital</name>
      <address>
        <city>Orlando</city>
        <state>Florida</state>
        <zip>32827</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Rare Disease Research, LLC</name>
      <address>
        <city>Atlanta</city>
        <state>Georgia</state>
        <zip>30318</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Rush University Medical Center - PPDS</name>
      <address>
        <city>Chicago</city>
        <state>Illinois</state>
        <zip>60612</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>University of Iowa</name>
      <address>
        <city>Iowa City</city>
        <state>Iowa</state>
        <zip>52242</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>University of Kansas Medical Center</name>
      <address>
        <city>Kansas City</city>
        <state>Kansas</state>
        <zip>66160</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Kennedy Krieger Institute</name>
      <address>
        <city>Baltimore</city>
        <state>Maryland</state>
        <zip>21205</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>University of Massachusetts Memorial Childrens Medical Center; Department of Neurology</name>
      <address>
        <city>Worcester</city>
        <state>Massachusetts</state>
        <zip>01655</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Saint Louis Children's Hospital</name>
      <address>
        <city>Saint Louis</city>
        <state>Missouri</state>
        <zip>63110</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Las Vegas Clinic</name>
      <address>
        <city>Las Vegas</city>
        <state>Nevada</state>
        <zip>89145</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Cincinnati Childrens Hospital Medical Center;Investigational Pharmacy</name>
      <address>
        <city>Cincinnati</city>
        <state>Ohio</state>
        <zip>45229</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Nationwide Childrens Hospital; Research Institute at Nationwide Childrens Hospital</name>
      <address>
        <city>Columbus</city>
        <state>Ohio</state>
        <zip>43205</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Seattle Children's Hospital</name>
      <address>
        <city>Seattle</city>
        <state>Washington</state>
        <zip>98105</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Instituto centenario</name>
      <address>
        <city>Buenos Aires</city>
        <zip>C1204AAD</zip>
        <country>Argentina</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Children's Hospital Westmead; Paediatrics &amp; Child Health</name>
      <address>
        <city>Westmead</city>
        <state>New South Wales</state>
        <zip>2145</zip>
        <country>Australia</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Lady Cilento Children's Hospital; Neurosciences Department</name>
      <address>
        <city>South Brisbane</city>
        <state>Queensland</state>
        <zip>4101</zip>
        <country>Australia</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Royal Children's Hospital</name>
      <address>
        <city>Parkville</city>
        <state>Victoria</state>
        <zip>3052</zip>
        <country>Australia</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>UZ Gent</name>
      <address>
        <city>Gent</city>
        <zip>9000</zip>
        <country>Belgium</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>London Health Sciences Centre; Children's Hospital; Pediatrics</name>
      <address>
        <city>London</city>
        <state>Ontario</state>
        <zip>N6A 5W9</zip>
        <country>Canada</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Children'S Hospital of Eastern Ontario</name>
      <address>
        <city>Ottawa</city>
        <state>Ontario</state>
        <zip>K1H 8L1</zip>
        <country>Canada</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Hospices Civils de Lyon</name>
      <address>
        <city>Lyon</city>
        <zip>69004</zip>
        <country>France</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Hotel Dieu; Service Pharmacie Essais Cliniques</name>
      <address>
        <city>Nantes</city>
        <zip>44093</zip>
        <country>France</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Hopital Armand Trousseau; centre reference Maladies Neuro-musculaires Est parisien Neuropediatrie</name>
      <address>
        <city>Paris Cedex 12</city>
        <zip>75571</zip>
        <country>France</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Hopitaux Universitaires de Strasbourg</name>
      <address>
        <city>Strasbourg</city>
        <zip>67091</zip>
        <country>France</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Universitatsklinikum Essen; Innere Klinik</name>
      <address>
        <city>Essen</city>
        <zip>45122</zip>
        <country>Germany</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Fondazione Serena Onlus - CENTRO CLINICO NEMO</name>
      <address>
        <city>Milano</city>
        <state>Emilia-Romagna</state>
        <zip>20162</zip>
        <country>Italy</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Fondazione Policlinico Universitario A Gemelli; Servizio di Farmacia</name>
      <address>
        <city>Rome</city>
        <state>Lazio</state>
        <zip>00168</zip>
        <country>Italy</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Az. Osp. Universitaria Pol. G. Martino; Dip. Neuroscienze, Scienze Psichiatriche e Anest.</name>
      <address>
        <city>Messina</city>
        <state>Sicilia</state>
        <zip>98125</zip>
        <country>Italy</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Hyogo College of Medicine Hospital</name>
      <address>
        <city>Hyogo</city>
        <zip>663-8501</zip>
        <country>Japan</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Miyagi Children's Hospital</name>
      <address>
        <city>Miyagi</city>
        <zip>989-3126</zip>
        <country>Japan</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Shinshu University Hospital</name>
      <address>
        <city>Nagano</city>
        <zip>390-8621</zip>
        <country>Japan</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>National Hospital Organization Osaka Toneyama Medical Center</name>
      <address>
        <city>Osaka</city>
        <zip>560-8552</zip>
        <country>Japan</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>National Center of Neurology and Psychiatry</name>
      <address>
        <city>Tokyo</city>
        <zip>187-8551</zip>
        <country>Japan</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Leids Universitair Medisch Centrum</name>
      <address>
        <city>Leiden</city>
        <zip>2333 ZA</zip>
        <country>Netherlands</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Radboud University Nijmegen Medical Centre; Ophthalmology</name>
      <address>
        <city>Nijmegen</city>
        <zip>6525 EX</zip>
        <country>Netherlands</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Hospital Sant Joan De Deu</name>
      <address>
        <city>Esplugues De Llobregas</city>
        <state>Barcelona</state>
        <zip>08950</zip>
        <country>Spain</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Hospital Universitari i Politecnic La Fe de Valencia; Servicio de Farmacia</name>
      <address>
        <city>Valencia</city>
        <zip>46026</zip>
        <country>Spain</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Drottning Silvias Barn- och ungdomssjukhus; Kliniken for barnmedicin</name>
      <address>
        <city>Goeteborg</city>
        <zip>41685</zip>
        <country>Sweden</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Alder Hey Children s Hospital; Department of Pediatrics</name>
      <address>
        <city>Liverpool</city>
        <zip>L12 2AP</zip>
        <country>United Kingdom</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>UCL Institute of Child Health &amp; Great Ormond Street Hospital for Children</name>
      <address>
        <city>London</city>
        <zip>WC1N 1EH</zip>
        <country>United Kingdom</country>
      </address>
    </facility>
  </location>
  <location_countries>
    <country>Argentina</country>
    <country>Australia</country>
    <country>Belgium</country>
    <country>Canada</country>
    <country>France</country>
    <country>Germany</country>
    <country>Italy</country>
    <country>Japan</country>
    <country>Netherlands</country>
    <country>Spain</country>
    <country>Sweden</country>
    <country>United Kingdom</country>
    <country>United States</country>
  </location_countries>
  <link>
    <url>http://www.fda.gov/Safety/Recalls/</url>
    <description>FDA Safety Alerts and Recalls</description>
  </link>
  <link>
    <url>http://roche-duchenne-clinicaltrials.com</url>
    <description>Provides information about the Roche clinical trial NCT03039686 and molecule being investigated in Duchenne</description>
  </link>
  <verification_date>November 2020</verification_date>
  <study_first_submitted>January 27, 2017</study_first_submitted>
  <study_first_submitted_qc>January 31, 2017</study_first_submitted_qc>
  <study_first_posted type="Estimate">February 1, 2017</study_first_posted>
  <results_first_submitted>October 21, 2020</results_first_submitted>
  <results_first_submitted_qc>November 25, 2020</results_first_submitted_qc>
  <results_first_posted type="Actual">December 21, 2020</results_first_posted>
  <last_update_submitted>November 25, 2020</last_update_submitted>
  <last_update_submitted_qc>November 25, 2020</last_update_submitted_qc>
  <last_update_posted type="Actual">December 21, 2020</last_update_posted>
  <responsible_party>
    <responsible_party_type>Sponsor</responsible_party_type>
  </responsible_party>
  <keyword>muscular dystrophy</keyword>
  <keyword>Duchenne's Muscular Dystrophy</keyword>
  <keyword>DMD</keyword>
  <condition_browse>
    <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
    <mesh_term>Muscular Dystrophies</mesh_term>
    <mesh_term>Muscular Dystrophy, Duchenne</mesh_term>
  </condition_browse>
  <provided_document_section>
    <provided_document>
      <document_type>Study Protocol</document_type>
      <document_has_protocol>Yes</document_has_protocol>
      <document_has_icf>No</document_has_icf>
      <document_has_sap>No</document_has_sap>
      <document_date>August 16, 2018</document_date>
      <document_url>https://ClinicalTrials.gov/ProvidedDocs/86/NCT03039686/Prot_000.pdf</document_url>
    </provided_document>
    <provided_document>
      <document_type>Statistical Analysis Plan</document_type>
      <document_has_protocol>No</document_has_protocol>
      <document_has_icf>No</document_has_icf>
      <document_has_sap>Yes</document_has_sap>
      <document_date>October 11, 2019</document_date>
      <document_url>https://ClinicalTrials.gov/ProvidedDocs/86/NCT03039686/SAP_001.pdf</document_url>
    </provided_document>
  </provided_document_section>

  <clinical_results>

    <participant_flow>
      <group_list>
        <group group_id="P1">
          <title>Placebo</title>
          <description>Participants received matching placebo solution subcutaneously (SC) on specified days of the 48-week double-blind (DB) period. Following the DB period participants received low dose or high dose RO7239361 on specified days for up to 192 weeks during the open-label period followed by 24 weeks of follow-up.</description>
        </group>
        <group group_id="P2">
          <title>RO7239361 Low Dose</title>
          <description>Participants received low dose RO7239361 SC on specified days of the 48-week DB period. Following the DB period participants received low dose RO7239361 on specified days for up to 192 weeks during the open-label period followed by 24 weeks of follow-up.</description>
        </group>
        <group group_id="P3">
          <title>RO7239361 High Dose</title>
          <description>Participants received high dose RO7239361 SC on specified days of the 48-week DB period. Following the DB period participants received high dose RO7239361 on specified days for up to 192 weeks during the open-label period followed by 24 weeks of follow-up.</description>
        </group>
      </group_list>
      <period_list>
        <period>
          <title>Double-blind Period</title>
          <milestone_list>
            <milestone>
              <title>STARTED</title>
              <participants_list>
                <participants group_id="P1" count="56"/>
                <participants group_id="P2" count="55"/>
                <participants group_id="P3" count="55"/>
              </participants_list>
            </milestone>
            <milestone>
              <title>COMPLETED</title>
              <participants_list>
                <participants group_id="P1" count="29"/>
                <participants group_id="P2" count="26"/>
                <participants group_id="P3" count="32"/>
              </participants_list>
            </milestone>
            <milestone>
              <title>NOT COMPLETED</title>
              <participants_list>
                <participants group_id="P1" count="27"/>
                <participants group_id="P2" count="29"/>
                <participants group_id="P3" count="23"/>
              </participants_list>
            </milestone>
          </milestone_list>
          <drop_withdraw_reason_list>
            <drop_withdraw_reason>
              <title>Administrative Reason by Sponsor</title>
              <participants_list>
                <participants group_id="P1" count="24"/>
                <participants group_id="P2" count="25"/>
                <participants group_id="P3" count="21"/>
              </participants_list>
            </drop_withdraw_reason>
            <drop_withdraw_reason>
              <title>Subject Request to Discontinue Study Treatment</title>
              <participants_list>
                <participants group_id="P1" count="1"/>
                <participants group_id="P2" count="2"/>
                <participants group_id="P3" count="0"/>
              </participants_list>
            </drop_withdraw_reason>
            <drop_withdraw_reason>
              <title>Withdrawal by Subject</title>
              <participants_list>
                <participants group_id="P1" count="2"/>
                <participants group_id="P2" count="2"/>
                <participants group_id="P3" count="1"/>
              </participants_list>
            </drop_withdraw_reason>
            <drop_withdraw_reason>
              <title>Death</title>
              <participants_list>
                <participants group_id="P1" count="0"/>
                <participants group_id="P2" count="0"/>
                <participants group_id="P3" count="1"/>
              </participants_list>
            </drop_withdraw_reason>
          </drop_withdraw_reason_list>
        </period>
        <period>
          <title>Open Label Period</title>
          <milestone_list>
            <milestone>
              <title>STARTED</title>
              <participants_list>
                <participants group_id="P1" count="0">Double-blind (DB) placebo participants switched to RO7239361 (RO) for the open label phase.</participants>
                <participants group_id="P2" count="38">14 DB placebo participants switched to the RO Low Dose for the open-label phase.</participants>
                <participants group_id="P3" count="42">13 DB placebo participants switched to the RO High Dose for the open-label phase.</participants>
              </participants_list>
            </milestone>
            <milestone>
              <title>Completed DB, Did Not Start OL</title>
              <participants_list>
                <participants group_id="P1" count="2"/>
                <participants group_id="P2" count="2"/>
                <participants group_id="P3" count="3"/>
              </participants_list>
            </milestone>
            <milestone>
              <title>COMPLETED</title>
              <participants_list>
                <participants group_id="P1" count="0"/>
                <participants group_id="P2" count="0"/>
                <participants group_id="P3" count="0"/>
              </participants_list>
            </milestone>
            <milestone>
              <title>NOT COMPLETED</title>
              <participants_list>
                <participants group_id="P1" count="0"/>
                <participants group_id="P2" count="38"/>
                <participants group_id="P3" count="42"/>
              </participants_list>
            </milestone>
          </milestone_list>
          <drop_withdraw_reason_list>
            <drop_withdraw_reason>
              <title>Administrative Reason by Sponsor</title>
              <participants_list>
                <participants group_id="P1" count="0"/>
                <participants group_id="P2" count="34"/>
                <participants group_id="P3" count="41"/>
              </participants_list>
            </drop_withdraw_reason>
            <drop_withdraw_reason>
              <title>Subject Request to Discontinue Study Treatment</title>
              <participants_list>
                <participants group_id="P1" count="0"/>
                <participants group_id="P2" count="1"/>
                <participants group_id="P3" count="0"/>
              </participants_list>
            </drop_withdraw_reason>
            <drop_withdraw_reason>
              <title>Withdrawal by Subject</title>
              <participants_list>
                <participants group_id="P1" count="0"/>
                <participants group_id="P2" count="3"/>
                <participants group_id="P3" count="1"/>
              </participants_list>
            </drop_withdraw_reason>
          </drop_withdraw_reason_list>
        </period>
      </period_list>
    </participant_flow>

    <baseline>
      <group_list>
        <group group_id="B1">
          <title>Placebo</title>
          <description>Participants received matching placebo solution subcutaneously (SC) on specified days of the 48-week double-blind (DB) period. Following the DB period participants received low dose or high dose RO7239361 on specified days for up to 192 weeks during the open-label period followed by 24 weeks of follow-up.</description>
        </group>
        <group group_id="B2">
          <title>RO7239361 Low Dose</title>
          <description>Participants received low dose RO7239361 SC on specified days of the 48-week DB period. Following the DB period participants received low dose RO7239361 on specified days for up to 192 weeks during the open-label period followed by 24 weeks of follow-up.</description>
        </group>
        <group group_id="B3">
          <title>RO7239361 High Dose</title>
          <description>Participants received high dose RO7239361 SC on specified days of the 48-week DB period. Following the DB period participants received high dose RO7239361 on specified days for up to 192 weeks during the open-label period followed by 24 weeks of follow-up.</description>
        </group>
        <group group_id="B4">
          <title>Total</title>
          <description>Total of all reporting groups</description>
        </group>
      </group_list>
      <analyzed_list>
        <analyzed>
          <units>Participants</units>
          <scope>Overall</scope>
          <count_list>
            <count group_id="B1" value="56"/>
            <count group_id="B2" value="55"/>
            <count group_id="B3" value="55"/>
            <count group_id="B4" value="166"/>
          </count_list>
        </analyzed>
      </analyzed_list>
      <measure_list>
        <measure>
          <title>Age</title>
          <units>years</units>
          <param>Mean</param>
          <dispersion>Standard Deviation</dispersion>
          <class_list>
            <class>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="B1" value="8.4" spread="1.7"/>
                    <measurement group_id="B2" value="8.5" spread="1.8"/>
                    <measurement group_id="B3" value="8.4" spread="1.5"/>
                    <measurement group_id="B4" value="8.5" spread="1.7"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
        <measure>
          <title>Age, Customized</title>
          <units>participants</units>
          <param>Number</param>
          <class_list>
            <class>
              <title>Children (6-11 years)</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="B1" value="56"/>
                    <measurement group_id="B2" value="55"/>
                    <measurement group_id="B3" value="55"/>
                    <measurement group_id="B4" value="166"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
        <measure>
          <title>Sex: Female, Male</title>
          <units>Participants</units>
          <param>Count of Participants</param>
          <class_list>
            <class>
              <category_list>
                <category>
                  <title>Female</title>
                  <measurement_list>
                    <measurement group_id="B1" value="0"/>
                    <measurement group_id="B2" value="0"/>
                    <measurement group_id="B3" value="0"/>
                    <measurement group_id="B4" value="0"/>
                  </measurement_list>
                </category>
                <category>
                  <title>Male</title>
                  <measurement_list>
                    <measurement group_id="B1" value="56"/>
                    <measurement group_id="B2" value="55"/>
                    <measurement group_id="B3" value="55"/>
                    <measurement group_id="B4" value="166"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
        <measure>
          <title>Ethnicity (NIH/OMB)</title>
          <units>Participants</units>
          <param>Count of Participants</param>
          <class_list>
            <class>
              <category_list>
                <category>
                  <title>Hispanic or Latino</title>
                  <measurement_list>
                    <measurement group_id="B1" value="7"/>
                    <measurement group_id="B2" value="5"/>
                    <measurement group_id="B3" value="6"/>
                    <measurement group_id="B4" value="18"/>
                  </measurement_list>
                </category>
                <category>
                  <title>Not Hispanic or Latino</title>
                  <measurement_list>
                    <measurement group_id="B1" value="43"/>
                    <measurement group_id="B2" value="41"/>
                    <measurement group_id="B3" value="43"/>
                    <measurement group_id="B4" value="127"/>
                  </measurement_list>
                </category>
                <category>
                  <title>Unknown or Not Reported</title>
                  <measurement_list>
                    <measurement group_id="B1" value="6"/>
                    <measurement group_id="B2" value="9"/>
                    <measurement group_id="B3" value="6"/>
                    <measurement group_id="B4" value="21"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
        <measure>
          <title>Race/Ethnicity, Customized</title>
          <units>participants</units>
          <param>Number</param>
          <class_list>
            <class>
              <title>Asian</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="B1" value="9"/>
                    <measurement group_id="B2" value="7"/>
                    <measurement group_id="B3" value="7"/>
                    <measurement group_id="B4" value="23"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Native Hawaiian or other Pacific Islander</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="B1" value="0"/>
                    <measurement group_id="B2" value="0"/>
                    <measurement group_id="B3" value="1"/>
                    <measurement group_id="B4" value="1"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>White</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="B1" value="42"/>
                    <measurement group_id="B2" value="45"/>
                    <measurement group_id="B3" value="45"/>
                    <measurement group_id="B4" value="132"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Other</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="B1" value="5"/>
                    <measurement group_id="B2" value="3"/>
                    <measurement group_id="B3" value="2"/>
                    <measurement group_id="B4" value="10"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
      </measure_list>
    </baseline>

    <outcome_list>
      <outcome>
        <type>Primary</type>
        <title>Baseline for the North Star Ambulatory Assessment (NSAA) Total Score</title>
        <description>The NSAA is a functional scale specifically designed for ambulant boys with Duchenne muscular dystrophy (DMD) that can provide information about motor function. The NSAA is a 17-item test of standing, ability to transition from lying to sitting, sitting to standing, and other mobility assessments. Each of the 17 items is evaluated on an ordinal scale of 0-2: 0 = unable to achieve independently, 1 = modified method but achieves goal independent of physical assistance from another, or 2 = normal with no obvious modification of activity. Total score range is 0 to 34. Higher scores reflect better performance.</description>
        <time_frame>Baseline</time_frame>
        <population>Intent-to-Treat (ITT) population included all enrolled participants who received a randomization treatment assignment.</population>
        <group_list>
          <group group_id="O1">
            <title>Placebo</title>
            <description>Participants received matching placebo solution subcutaneously (SC) on specified days of the 48-week double-blind (DB) period. Following the DB period participants received low dose or high dose RO7239361 on specified days for up to 192 weeks during the open-label period followed by 24 weeks of follow-up.</description>
          </group>
          <group group_id="O2">
            <title>RO7239361 Low Dose</title>
            <description>Participants received low dose RO7239361 SC on specified days of the 48-week DB period. Following the DB period participants received low dose RO7239361 on specified days for up to 192 weeks during the open-label period followed by 24 weeks of follow-up.</description>
          </group>
          <group group_id="O3">
            <title>RO7239361 High Dose</title>
            <description>Participants received high dose RO7239361 SC on specified days of the 48-week DB period. Following the DB period participants received high dose RO7239361 on specified days for up to 192 weeks during the open-label period followed by 24 weeks of follow-up.</description>
          </group>
        </group_list>
        <measure>
          <title>Baseline for the North Star Ambulatory Assessment (NSAA) Total Score</title>
          <description>The NSAA is a functional scale specifically designed for ambulant boys with Duchenne muscular dystrophy (DMD) that can provide information about motor function. The NSAA is a 17-item test of standing, ability to transition from lying to sitting, sitting to standing, and other mobility assessments. Each of the 17 items is evaluated on an ordinal scale of 0-2: 0 = unable to achieve independently, 1 = modified method but achieves goal independent of physical assistance from another, or 2 = normal with no obvious modification of activity. Total score range is 0 to 34. Higher scores reflect better performance.</description>
          <population>Intent-to-Treat (ITT) population included all enrolled participants who received a randomization treatment assignment.</population>
          <units>score on a scale</units>
          <param>Mean</param>
          <dispersion>Standard Deviation</dispersion>
          <analyzed_list>
            <analyzed>
              <units>Participants</units>
              <scope>Measure</scope>
              <count_list>
                <count group_id="O1" value="56"/>
                <count group_id="O2" value="55"/>
                <count group_id="O3" value="55"/>
              </count_list>
            </analyzed>
          </analyzed_list>
          <class_list>
            <class>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="23.1" spread="6.4"/>
                    <measurement group_id="O2" value="24.5" spread="5.5"/>
                    <measurement group_id="O3" value="22.7" spread="6.7"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
      </outcome>
      <outcome>
        <type>Primary</type>
        <title>Change From Baseline in the North Star Ambulatory Assessment (NSAA) Total Score at Week 48</title>
        <description>The NSAA is a functional scale specifically designed for ambulant boys with Duchenne muscular dystrophy (DMD) that can provide information about motor function. The NSAA is a 17-item test of standing, ability to transition from lying to sitting, sitting to standing, and other mobility assessments. Each of the 17 items is evaluated on an ordinal scale of 0-2: 0 = unable to achieve independently, 1 = modified method but achieves goal independent of physical assistance from another, or 2 = normal with no obvious modification of activity. Total score range is 0 to 34. Higher scores reflect better performance. A positive change from baseline indicates an improvement. Based on the mixed-effect model of repeated measures (MMRM).</description>
        <time_frame>Baseline, Week 48</time_frame>
        <population>Intent-to-Treat (ITT) population included all enrolled participants who received a randomization treatment assignment. MMRM analysis included all participants at baseline and the following number of participants by Week 48: Placebo n=30, Low Dose n=29, High Dose n=33.</population>
        <group_list>
          <group group_id="O1">
            <title>Placebo</title>
            <description>Participants received matching placebo solution subcutaneously (SC) on specified days of the 48-week double-blind (DB) period. Following the DB period participants received low dose or high dose RO7239361 on specified days for up to 192 weeks during the open-label period followed by 24 weeks of follow-up.</description>
          </group>
          <group group_id="O2">
            <title>RO7239361 Low Dose</title>
            <description>Participants received low dose RO7239361 SC on specified days of the 48-week DB period. Following the DB period participants received low dose RO7239361 on specified days for up to 192 weeks during the open-label period followed by 24 weeks of follow-up.</description>
          </group>
          <group group_id="O3">
            <title>RO7239361 High Dose</title>
            <description>Participants received high dose RO7239361 SC on specified days of the 48-week DB period. Following the DB period participants received high dose RO7239361 on specified days for up to 192 weeks during the open-label period followed by 24 weeks of follow-up.</description>
          </group>
        </group_list>
        <measure>
          <title>Change From Baseline in the North Star Ambulatory Assessment (NSAA) Total Score at Week 48</title>
          <description>The NSAA is a functional scale specifically designed for ambulant boys with Duchenne muscular dystrophy (DMD) that can provide information about motor function. The NSAA is a 17-item test of standing, ability to transition from lying to sitting, sitting to standing, and other mobility assessments. Each of the 17 items is evaluated on an ordinal scale of 0-2: 0 = unable to achieve independently, 1 = modified method but achieves goal independent of physical assistance from another, or 2 = normal with no obvious modification of activity. Total score range is 0 to 34. Higher scores reflect better performance. A positive change from baseline indicates an improvement. Based on the mixed-effect model of repeated measures (MMRM).</description>
          <population>Intent-to-Treat (ITT) population included all enrolled participants who received a randomization treatment assignment. MMRM analysis included all participants at baseline and the following number of participants by Week 48: Placebo n=30, Low Dose n=29, High Dose n=33.</population>
          <units>score on a scale</units>
          <param>Least Squares Mean</param>
          <dispersion>Standard Error</dispersion>
          <analyzed_list>
            <analyzed>
              <units>Participants</units>
              <scope>Measure</scope>
              <count_list>
                <count group_id="O1" value="56"/>
                <count group_id="O2" value="55"/>
                <count group_id="O3" value="55"/>
              </count_list>
            </analyzed>
          </analyzed_list>
          <class_list>
            <class>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="-2.99" spread="0.65"/>
                    <measurement group_id="O2" value="-3.44" spread="0.67"/>
                    <measurement group_id="O3" value="-2.41" spread="0.64"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
        <analysis_list>
          <analysis>
            <group_id_list>
              <group_id>O1</group_id>
              <group_id>O2</group_id>
            </group_id_list>
            <non_inferiority_type>Superiority</non_inferiority_type>
            <param_type>Mean Difference (Final Values)</param_type>
            <param_value>-0.45</param_value>
            <dispersion_type>Standard Error of the Mean</dispersion_type>
            <dispersion_value>0.87</dispersion_value>
            <ci_percent>95</ci_percent>
            <ci_n_sides>2-Sided</ci_n_sides>
            <ci_lower_limit>-2.17</ci_lower_limit>
            <ci_upper_limit>1.27</ci_upper_limit>
            <estimate_desc>Based on the MMRM using an unstructured covariance matrix -change = Baseline + Age Interactive response system (IXRS) Stratum + Corticosteroid Regimen IXRS Stratum + Analysis Visit*Treatment.</estimate_desc>
          </analysis>
          <analysis>
            <group_id_list>
              <group_id>O1</group_id>
              <group_id>O3</group_id>
            </group_id_list>
            <non_inferiority_type>Superiority</non_inferiority_type>
            <param_type>Mean Difference (Final Values)</param_type>
            <param_value>0.58</param_value>
            <dispersion_type>Standard Error of the Mean</dispersion_type>
            <dispersion_value>0.85</dispersion_value>
            <ci_percent>95</ci_percent>
            <ci_n_sides>2-Sided</ci_n_sides>
            <ci_lower_limit>-1.10</ci_lower_limit>
            <ci_upper_limit>2.26</ci_upper_limit>
            <estimate_desc>Based on the MMRM using an unstructured covariance matrix -change = Baseline + Age IXRS Stratum + Corticosteroid Regimen IXRS Stratum + Analysis Visit*Treatment.</estimate_desc>
          </analysis>
        </analysis_list>
      </outcome>
      <outcome>
        <type>Secondary</type>
        <title>Baseline Time for 4 Stair Climb</title>
        <description>The time to complete the 4 stair climb was measured at baseline.</description>
        <time_frame>Baseline</time_frame>
        <population>Intent-to-Treat (ITT) population included all enrolled participants who received a randomization treatment assignment.</population>
        <group_list>
          <group group_id="O1">
            <title>Placebo</title>
            <description>Participants received matching placebo solution subcutaneously (SC) on specified days of the 48-week double-blind (DB) period. Following the DB period participants received low dose or high dose RO7239361 on specified days for up to 192 weeks during the open-label period followed by 24 weeks of follow-up.</description>
          </group>
          <group group_id="O2">
            <title>RO7239361 Low Dose</title>
            <description>Participants received low dose RO7239361 SC on specified days of the 48-week DB period. Following the DB period participants received low dose RO7239361 on specified days for up to 192 weeks during the open-label period followed by 24 weeks of follow-up.</description>
          </group>
          <group group_id="O3">
            <title>RO7239361 High Dose</title>
            <description>Participants received high dose RO7239361 SC on specified days of the 48-week DB period. Following the DB period participants received high dose RO7239361 on specified days for up to 192 weeks during the open-label period followed by 24 weeks of follow-up.</description>
          </group>
        </group_list>
        <measure>
          <title>Baseline Time for 4 Stair Climb</title>
          <description>The time to complete the 4 stair climb was measured at baseline.</description>
          <population>Intent-to-Treat (ITT) population included all enrolled participants who received a randomization treatment assignment.</population>
          <units>seconds (secs)</units>
          <param>Mean</param>
          <dispersion>Standard Deviation</dispersion>
          <analyzed_list>
            <analyzed>
              <units>Participants</units>
              <scope>Measure</scope>
              <count_list>
                <count group_id="O1" value="56"/>
                <count group_id="O2" value="55"/>
                <count group_id="O3" value="55"/>
              </count_list>
            </analyzed>
          </analyzed_list>
          <class_list>
            <class>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="3.81" spread="1.55"/>
                    <measurement group_id="O2" value="3.85" spread="1.61"/>
                    <measurement group_id="O3" value="3.92" spread="1.91"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
      </outcome>
      <outcome>
        <type>Secondary</type>
        <title>Change From Baseline at Week 48 in 4 Stair Climb Velocity (4SCV)</title>
        <description>4SCV was calculated as the ratio of the number of stairs climbed (4) divided by the number of seconds taken to complete the 4-stair climb. The results were converted into velocity (distance/time). A positive change from baseline indicates an improvement. Based on the mixed-effect model of repeated measures (MMRM).</description>
        <time_frame>Baseline, Week 48</time_frame>
        <population>Intent-to-Treat (ITT) population included all enrolled participants who received a randomization treatment assignment. MMRM analysis included all participants at baseline and the following number of participants by Week 48: Placebo n=30, Low Dose n=29, High Dose n=33.</population>
        <group_list>
          <group group_id="O1">
            <title>Placebo</title>
            <description>Participants received matching placebo solution subcutaneously (SC) on specified days of the 48-week double-blind (DB) period. Following the DB period participants received low dose or high dose RO7239361 on specified days for up to 192 weeks during the open-label period followed by 24 weeks of follow-up.</description>
          </group>
          <group group_id="O2">
            <title>RO7239361 Low Dose</title>
            <description>Participants received low dose RO7239361 SC on specified days of the 48-week DB period. Following the DB period participants received low dose RO7239361 on specified days for up to 192 weeks during the open-label period followed by 24 weeks of follow-up.</description>
          </group>
          <group group_id="O3">
            <title>RO7239361 High Dose</title>
            <description>Participants received high dose RO7239361 SC on specified days of the 48-week DB period. Following the DB period participants received high dose RO7239361 on specified days for up to 192 weeks during the open-label period followed by 24 weeks of follow-up.</description>
          </group>
        </group_list>
        <measure>
          <title>Change From Baseline at Week 48 in 4 Stair Climb Velocity (4SCV)</title>
          <description>4SCV was calculated as the ratio of the number of stairs climbed (4) divided by the number of seconds taken to complete the 4-stair climb. The results were converted into velocity (distance/time). A positive change from baseline indicates an improvement. Based on the mixed-effect model of repeated measures (MMRM).</description>
          <population>Intent-to-Treat (ITT) population included all enrolled participants who received a randomization treatment assignment. MMRM analysis included all participants at baseline and the following number of participants by Week 48: Placebo n=30, Low Dose n=29, High Dose n=33.</population>
          <units>stairs/sec</units>
          <param>Least Squares Mean</param>
          <dispersion>Standard Error</dispersion>
          <analyzed_list>
            <analyzed>
              <units>Participants</units>
              <scope>Measure</scope>
              <count_list>
                <count group_id="O1" value="56"/>
                <count group_id="O2" value="55"/>
                <count group_id="O3" value="55"/>
              </count_list>
            </analyzed>
          </analyzed_list>
          <class_list>
            <class>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="-0.15" spread="0.07"/>
                    <measurement group_id="O2" value="-0.15" spread="0.07"/>
                    <measurement group_id="O3" value="-0.07" spread="0.07"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
        <analysis_list>
          <analysis>
            <group_id_list>
              <group_id>O1</group_id>
              <group_id>O2</group_id>
            </group_id_list>
            <non_inferiority_type>Superiority</non_inferiority_type>
            <param_type>Mean Difference (Final Values)</param_type>
            <param_value>-0.00</param_value>
            <dispersion_type>Standard Error of the Mean</dispersion_type>
            <dispersion_value>0.10</dispersion_value>
            <ci_percent>95</ci_percent>
            <ci_n_sides>2-Sided</ci_n_sides>
            <ci_lower_limit>-0.21</ci_lower_limit>
            <ci_upper_limit>0.20</ci_upper_limit>
            <estimate_desc>Based on the MMRM using an unstructured covariance matrix - change = Baseline + Age IXRS Stratum + Corticosteroid Regimen IXRS Stratum + Analysis Visit*Treatment.</estimate_desc>
          </analysis>
          <analysis>
            <group_id_list>
              <group_id>O1</group_id>
              <group_id>O3</group_id>
            </group_id_list>
            <non_inferiority_type>Superiority</non_inferiority_type>
            <param_type>Mean Difference (Final Values)</param_type>
            <param_value>0.08</param_value>
            <dispersion_type>Standard Error of the Mean</dispersion_type>
            <dispersion_value>0.10</dispersion_value>
            <ci_percent>95</ci_percent>
            <ci_n_sides>2-Sided</ci_n_sides>
            <ci_lower_limit>-0.12</ci_lower_limit>
            <ci_upper_limit>0.27</ci_upper_limit>
            <estimate_desc>Based on the MMRM using an unstructured covariance matrix - change = Baseline + Age IXRS Stratum + Corticosteroid Regimen IXRS Stratum + Analysis Visit*Treatment.</estimate_desc>
          </analysis>
        </analysis_list>
      </outcome>
      <outcome>
        <type>Secondary</type>
        <title>Baseline for the Time to Stand From Supine</title>
        <description>The time required for a participant to stand from supine position. A longer time reflects a worse outcome.</description>
        <time_frame>Baseline</time_frame>
        <population>Intent-to-Treat (ITT) population included all enrolled participants who received a randomization treatment assignment.</population>
        <group_list>
          <group group_id="O1">
            <title>Placebo</title>
            <description>Participants received matching placebo solution subcutaneously (SC) on specified days of the 48-week double-blind (DB) period. Following the DB period participants received low dose or high dose RO7239361 on specified days for up to 192 weeks during the open-label period followed by 24 weeks of follow-up.</description>
          </group>
          <group group_id="O2">
            <title>RO7239361 Low Dose</title>
            <description>Participants received low dose RO7239361 SC on specified days of the 48-week DB period. Following the DB period participants received low dose RO7239361 on specified days for up to 192 weeks during the open-label period followed by 24 weeks of follow-up.</description>
          </group>
          <group group_id="O3">
            <title>RO7239361 High Dose</title>
            <description>Participants received high dose RO7239361 SC on specified days of the 48-week DB period. Following the DB period participants received high dose RO7239361 on specified days for up to 192 weeks during the open-label period followed by 24 weeks of follow-up.</description>
          </group>
        </group_list>
        <measure>
          <title>Baseline for the Time to Stand From Supine</title>
          <description>The time required for a participant to stand from supine position. A longer time reflects a worse outcome.</description>
          <population>Intent-to-Treat (ITT) population included all enrolled participants who received a randomization treatment assignment.</population>
          <units>secs</units>
          <param>Mean</param>
          <dispersion>Standard Deviation</dispersion>
          <analyzed_list>
            <analyzed>
              <units>Participants</units>
              <scope>Measure</scope>
              <count_list>
                <count group_id="O1" value="56"/>
                <count group_id="O2" value="55"/>
                <count group_id="O3" value="55"/>
              </count_list>
            </analyzed>
          </analyzed_list>
          <class_list>
            <class>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="6.28" spread="4.75"/>
                    <measurement group_id="O2" value="6.15" spread="4.07"/>
                    <measurement group_id="O3" value="7.24" spread="9.22"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
      </outcome>
      <outcome>
        <type>Secondary</type>
        <title>Change From Baseline at Week 48 in Stand From Supine Velocity</title>
        <description>The time required for a participant to stand from supine position. A longer time reflects a worse outcome. A negative change from baseline indicates an improvement. Based on the mixed-effect model of repeated measures (MMRM).</description>
        <time_frame>Baseline, Week 48</time_frame>
        <population>Intent-to-Treat (ITT) population included all enrolled participants who received a randomization treatment assignment. MMRM analysis included all participants at baseline and the following number of participants by Week 48: Placebo n=28, Low Dose n=28, High Dose n=32.</population>
        <group_list>
          <group group_id="O1">
            <title>Placebo</title>
            <description>Participants received matching placebo solution subcutaneously (SC) on specified days of the 48-week double-blind (DB) period. Following the DB period participants received low dose or high dose RO7239361 on specified days for up to 192 weeks during the open-label period followed by 24 weeks of follow-up.</description>
          </group>
          <group group_id="O2">
            <title>RO7239361 Low Dose</title>
            <description>Participants received low dose RO7239361 SC on specified days of the 48-week DB period. Following the DB period participants received low dose RO7239361 on specified days for up to 192 weeks during the open-label period followed by 24 weeks of follow-up.</description>
          </group>
          <group group_id="O3">
            <title>RO7239361 High Dose</title>
            <description>Participants received high dose RO7239361 SC on specified days of the 48-week DB period. Following the DB period participants received high dose RO7239361 on specified days for up to 192 weeks during the open-label period followed by 24 weeks of follow-up.</description>
          </group>
        </group_list>
        <measure>
          <title>Change From Baseline at Week 48 in Stand From Supine Velocity</title>
          <description>The time required for a participant to stand from supine position. A longer time reflects a worse outcome. A negative change from baseline indicates an improvement. Based on the mixed-effect model of repeated measures (MMRM).</description>
          <population>Intent-to-Treat (ITT) population included all enrolled participants who received a randomization treatment assignment. MMRM analysis included all participants at baseline and the following number of participants by Week 48: Placebo n=28, Low Dose n=28, High Dose n=32.</population>
          <units>1/sec</units>
          <param>Least Squares Mean</param>
          <dispersion>Standard Error</dispersion>
          <analyzed_list>
            <analyzed>
              <units>Participants</units>
              <scope>Measure</scope>
              <count_list>
                <count group_id="O1" value="56"/>
                <count group_id="O2" value="55"/>
                <count group_id="O3" value="55"/>
              </count_list>
            </analyzed>
          </analyzed_list>
          <class_list>
            <class>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="-0.05" spread="0.01"/>
                    <measurement group_id="O2" value="-0.02" spread="0.01"/>
                    <measurement group_id="O3" value="-0.02" spread="0.01"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
        <analysis_list>
          <analysis>
            <group_id_list>
              <group_id>O1</group_id>
              <group_id>O2</group_id>
            </group_id_list>
            <non_inferiority_type>Superiority</non_inferiority_type>
            <param_type>Mean Difference (Final Values)</param_type>
            <param_value>0.03</param_value>
            <dispersion_type>Standard Error of the Mean</dispersion_type>
            <dispersion_value>0.02</dispersion_value>
            <ci_percent>95</ci_percent>
            <ci_n_sides>2-Sided</ci_n_sides>
            <ci_lower_limit>-0.00</ci_lower_limit>
            <ci_upper_limit>0.06</ci_upper_limit>
            <estimate_desc>Based on the MMRM using an unstructured covariance matrix - change = Baseline + Age IXRS Stratum + Corticosteroid Regimen IXRS Stratum + Analysis Visit*Treatment.</estimate_desc>
          </analysis>
          <analysis>
            <group_id_list>
              <group_id>O1</group_id>
              <group_id>O3</group_id>
            </group_id_list>
            <non_inferiority_type>Superiority</non_inferiority_type>
            <param_type>Mean Difference (Final Values)</param_type>
            <param_value>0.03</param_value>
            <dispersion_type>Standard Error of the Mean</dispersion_type>
            <dispersion_value>0.02</dispersion_value>
            <ci_percent>95</ci_percent>
            <ci_n_sides>2-Sided</ci_n_sides>
            <ci_lower_limit>-0.00</ci_lower_limit>
            <ci_upper_limit>0.06</ci_upper_limit>
            <estimate_desc>Based on the MMRM using an unstructured covariance matrix - change = Baseline + Age IXRS Stratum + Corticosteroid Regimen IXRS Stratum + Analysis Visit*Treatment.</estimate_desc>
          </analysis>
        </analysis_list>
      </outcome>
      <outcome>
        <type>Secondary</type>
        <title>Baseline Time for 10 Meter Walk/Run</title>
        <description>The time required for a participant to run or walk a distance of 10 meters as quickly as possible. A longer time reflects a worse outcome.</description>
        <time_frame>Baseline</time_frame>
        <population>Intent-to-Treat (ITT) population included all enrolled participants who received a randomization treatment assignment.</population>
        <group_list>
          <group group_id="O1">
            <title>Placebo</title>
            <description>Participants received matching placebo solution subcutaneously (SC) on specified days of the 48-week double-blind (DB) period. Following the DB period participants received low dose or high dose RO7239361 on specified days for up to 192 weeks during the open-label period followed by 24 weeks of follow-up.</description>
          </group>
          <group group_id="O2">
            <title>RO7239361 Low Dose</title>
            <description>Participants received low dose RO7239361 SC on specified days of the 48-week DB period. Following the DB period participants received low dose RO7239361 on specified days for up to 192 weeks during the open-label period followed by 24 weeks of follow-up.</description>
          </group>
          <group group_id="O3">
            <title>RO7239361 High Dose</title>
            <description>Participants received high dose RO7239361 SC on specified days of the 48-week DB period. Following the DB period participants received high dose RO7239361 on specified days for up to 192 weeks during the open-label period followed by 24 weeks of follow-up.</description>
          </group>
        </group_list>
        <measure>
          <title>Baseline Time for 10 Meter Walk/Run</title>
          <description>The time required for a participant to run or walk a distance of 10 meters as quickly as possible. A longer time reflects a worse outcome.</description>
          <population>Intent-to-Treat (ITT) population included all enrolled participants who received a randomization treatment assignment.</population>
          <units>secs</units>
          <param>Mean</param>
          <dispersion>Standard Deviation</dispersion>
          <analyzed_list>
            <analyzed>
              <units>Participants</units>
              <scope>Measure</scope>
              <count_list>
                <count group_id="O1" value="56"/>
                <count group_id="O2" value="55"/>
                <count group_id="O3" value="55"/>
              </count_list>
            </analyzed>
          </analyzed_list>
          <class_list>
            <class>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="5.38" spread="1.48"/>
                    <measurement group_id="O2" value="5.51" spread="1.68"/>
                    <measurement group_id="O3" value="5.68" spread="2.30"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
      </outcome>
      <outcome>
        <type>Secondary</type>
        <title>Change From Baseline at Week 48 in 10 M Walk/Run Velocity</title>
        <description>The time required for a participant to run or walk a distance of 10 meters as quickly as possible calculated as velocity (distance/time). A positive change from baseline indicates an improvement. Based on the mixed-effect model of repeated measures (MMRM).</description>
        <time_frame>Baseline, Week 48</time_frame>
        <population>Intent-to-Treat (ITT) population included all enrolled participants who received a randomization treatment assignment. MMRM analysis included all participants at baseline and the following number of participants by Week 48: Placebo n=30, Low Dose n=29, High Dose n=31.</population>
        <group_list>
          <group group_id="O1">
            <title>Placebo</title>
            <description>Participants received matching placebo solution subcutaneously (SC) on specified days of the 48-week double-blind (DB) period. Following the DB period participants received low dose or high dose RO7239361 on specified days for up to 192 weeks during the open-label period followed by 24 weeks of follow-up.</description>
          </group>
          <group group_id="O2">
            <title>RO7239361 Low Dose</title>
            <description>Participants received low dose RO7239361 SC on specified days of the 48-week DB period. Following the DB period participants received low dose RO7239361 on specified days for up to 192 weeks during the open-label period followed by 24 weeks of follow-up.</description>
          </group>
          <group group_id="O3">
            <title>RO7239361 High Dose</title>
            <description>Participants received high dose RO7239361 SC on specified days of the 48-week DB period. Following the DB period participants received high dose RO7239361 on specified days for up to 192 weeks during the open-label period followed by 24 weeks of follow-up.</description>
          </group>
        </group_list>
        <measure>
          <title>Change From Baseline at Week 48 in 10 M Walk/Run Velocity</title>
          <description>The time required for a participant to run or walk a distance of 10 meters as quickly as possible calculated as velocity (distance/time). A positive change from baseline indicates an improvement. Based on the mixed-effect model of repeated measures (MMRM).</description>
          <population>Intent-to-Treat (ITT) population included all enrolled participants who received a randomization treatment assignment. MMRM analysis included all participants at baseline and the following number of participants by Week 48: Placebo n=30, Low Dose n=29, High Dose n=31.</population>
          <units>m/sec</units>
          <param>Least Squares Mean</param>
          <dispersion>Standard Error</dispersion>
          <analyzed_list>
            <analyzed>
              <units>Participants</units>
              <scope>Measure</scope>
              <count_list>
                <count group_id="O1" value="56"/>
                <count group_id="O2" value="55"/>
                <count group_id="O3" value="55"/>
              </count_list>
            </analyzed>
          </analyzed_list>
          <class_list>
            <class>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="-0.23" spread="0.06"/>
                    <measurement group_id="O2" value="-0.14" spread="0.07"/>
                    <measurement group_id="O3" value="-0.23" spread="0.06"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
        <analysis_list>
          <analysis>
            <group_id_list>
              <group_id>O1</group_id>
              <group_id>O2</group_id>
            </group_id_list>
            <non_inferiority_type>Superiority</non_inferiority_type>
            <param_type>Mean Difference (Final Values)</param_type>
            <param_value>0.09</param_value>
            <dispersion_type>Standard Error of the Mean</dispersion_type>
            <dispersion_value>0.09</dispersion_value>
            <ci_percent>95</ci_percent>
            <ci_n_sides>2-Sided</ci_n_sides>
            <ci_lower_limit>-0.08</ci_lower_limit>
            <ci_upper_limit>0.27</ci_upper_limit>
            <estimate_desc>Based on the MMRM using an unstructured covariance matrix - change = Baseline + Age IXRS Stratum + Corticosteroid Regimen IXRS Stratum + Analysis Visit*Treatment.</estimate_desc>
          </analysis>
          <analysis>
            <group_id_list>
              <group_id>O1</group_id>
              <group_id>O3</group_id>
            </group_id_list>
            <non_inferiority_type>Superiority</non_inferiority_type>
            <param_type>Mean Difference (Final Values)</param_type>
            <param_value>0.00</param_value>
            <dispersion_type>Standard Error of the Mean</dispersion_type>
            <dispersion_value>0.09</dispersion_value>
            <ci_percent>95</ci_percent>
            <ci_n_sides>2-Sided</ci_n_sides>
            <ci_lower_limit>-0.17</ci_lower_limit>
            <ci_upper_limit>0.18</ci_upper_limit>
            <estimate_desc>Based on the MMRM using an unstructured covariance matrix - change = Baseline + Age IXRS Stratum + Corticosteroid Regimen IXRS Stratum + Analysis Visit*Treatment.</estimate_desc>
          </analysis>
        </analysis_list>
      </outcome>
      <outcome>
        <type>Secondary</type>
        <title>Baseline for the Pediatric Outcome Data Collection Instrument (PODCI) Transfer and Basic Mobility Subscale</title>
        <description>The PODCI is designed to be completed by the parent/guardian of a child who has knowledge of the child's conditions. The Transfer and Basic Mobility scale is one of the subscales of the PODCI. The results are standardized into a scale of 0-100 with a higher score reflecting better performance.</description>
        <time_frame>Baseline</time_frame>
        <population>Intent-to-Treat (ITT) population included all enrolled participants who received a randomization treatment assignment.</population>
        <group_list>
          <group group_id="O1">
            <title>Placebo</title>
            <description>Participants received matching placebo solution subcutaneously (SC) on specified days of the 48-week double-blind (DB) period. Following the DB period participants received low dose or high dose RO7239361 on specified days for up to 192 weeks during the open-label period followed by 24 weeks of follow-up.</description>
          </group>
          <group group_id="O2">
            <title>RO7239361 Low Dose</title>
            <description>Participants received low dose RO7239361 SC on specified days of the 48-week DB period. Following the DB period participants received low dose RO7239361 on specified days for up to 192 weeks during the open-label period followed by 24 weeks of follow-up.</description>
          </group>
          <group group_id="O3">
            <title>RO7239361 High Dose</title>
            <description>Participants received high dose RO7239361 SC on specified days of the 48-week DB period. Following the DB period participants received high dose RO7239361 on specified days for up to 192 weeks during the open-label period followed by 24 weeks of follow-up.</description>
          </group>
        </group_list>
        <measure>
          <title>Baseline for the Pediatric Outcome Data Collection Instrument (PODCI) Transfer and Basic Mobility Subscale</title>
          <description>The PODCI is designed to be completed by the parent/guardian of a child who has knowledge of the child's conditions. The Transfer and Basic Mobility scale is one of the subscales of the PODCI. The results are standardized into a scale of 0-100 with a higher score reflecting better performance.</description>
          <population>Intent-to-Treat (ITT) population included all enrolled participants who received a randomization treatment assignment.</population>
          <units>score on a scale</units>
          <param>Mean</param>
          <dispersion>Standard Deviation</dispersion>
          <analyzed_list>
            <analyzed>
              <units>Participants</units>
              <scope>Measure</scope>
              <count_list>
                <count group_id="O1" value="56"/>
                <count group_id="O2" value="55"/>
                <count group_id="O3" value="55"/>
              </count_list>
            </analyzed>
          </analyzed_list>
          <class_list>
            <class>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="85.59" spread="10.21"/>
                    <measurement group_id="O2" value="86.54" spread="9.52"/>
                    <measurement group_id="O3" value="84.47" spread="14.76"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
      </outcome>
      <outcome>
        <type>Secondary</type>
        <title>Change From Baseline at Week 48 in Pediatric Outcome Data Collection Instrument (PODCI) Transfer and Basic Mobility Subscale</title>
        <description>The PODCI is designed to be completed by the parent/guardian of a child who has knowledge of the child's conditions. The Transfer and Basic Mobility scale is one of the subscales of the PODCI. The results are standardized into a scale of 0-100 with a higher score reflecting better performance. A positive change from baseline indicates an improvement. Based on the mixed-effect model of repeated measures (MMRM).</description>
        <time_frame>Baseline, Week 48</time_frame>
        <population>Intent-to-Treat (ITT) population included all enrolled participants who received a randomization treatment assignment. MMRM analysis included all participants at baseline and the following number of participants by Week 48: Placebo n=31, Low Dose n=29, High Dose n=34.</population>
        <group_list>
          <group group_id="O1">
            <title>Placebo</title>
            <description>Participants received matching placebo solution subcutaneously (SC) on specified days of the 48-week double-blind (DB) period. Following the DB period participants received low dose or high dose RO7239361 on specified days for up to 192 weeks during the open-label period followed by 24 weeks of follow-up.</description>
          </group>
          <group group_id="O2">
            <title>RO7239361 Low Dose</title>
            <description>Participants received low dose RO7239361 SC on specified days of the 48-week DB period. Following the DB period participants received low dose RO7239361 on specified days for up to 192 weeks during the open-label period followed by 24 weeks of follow-up.</description>
          </group>
          <group group_id="O3">
            <title>RO7239361 High Dose</title>
            <description>Participants received high dose RO7239361 SC on specified days of the 48-week DB period. Following the DB period participants received high dose RO7239361 on specified days for up to 192 weeks during the open-label period followed by 24 weeks of follow-up.</description>
          </group>
        </group_list>
        <measure>
          <title>Change From Baseline at Week 48 in Pediatric Outcome Data Collection Instrument (PODCI) Transfer and Basic Mobility Subscale</title>
          <description>The PODCI is designed to be completed by the parent/guardian of a child who has knowledge of the child's conditions. The Transfer and Basic Mobility scale is one of the subscales of the PODCI. The results are standardized into a scale of 0-100 with a higher score reflecting better performance. A positive change from baseline indicates an improvement. Based on the mixed-effect model of repeated measures (MMRM).</description>
          <population>Intent-to-Treat (ITT) population included all enrolled participants who received a randomization treatment assignment. MMRM analysis included all participants at baseline and the following number of participants by Week 48: Placebo n=31, Low Dose n=29, High Dose n=34.</population>
          <units>score on a scale</units>
          <param>Least Squares Mean</param>
          <dispersion>Standard Error</dispersion>
          <analyzed_list>
            <analyzed>
              <units>Participants</units>
              <scope>Measure</scope>
              <count_list>
                <count group_id="O1" value="56"/>
                <count group_id="O2" value="55"/>
                <count group_id="O3" value="55"/>
              </count_list>
            </analyzed>
          </analyzed_list>
          <class_list>
            <class>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="-5.47" spread="1.79"/>
                    <measurement group_id="O2" value="-7.47" spread="1.83"/>
                    <measurement group_id="O3" value="-4.51" spread="1.77"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
        <analysis_list>
          <analysis>
            <group_id_list>
              <group_id>O1</group_id>
              <group_id>O2</group_id>
            </group_id_list>
            <non_inferiority_type>Superiority</non_inferiority_type>
            <param_type>Mean Difference (Final Values)</param_type>
            <param_value>-2.00</param_value>
            <dispersion_type>Standard Error of the Mean</dispersion_type>
            <dispersion_value>2.41</dispersion_value>
            <ci_percent>95</ci_percent>
            <ci_n_sides>2-Sided</ci_n_sides>
            <ci_lower_limit>-6.76</ci_lower_limit>
            <ci_upper_limit>2.77</ci_upper_limit>
            <estimate_desc>Based on the MMRM using an unstructured covariance matrix - change = Baseline + Age IXRS Stratum + Corticosteroid Regimen IXRS Stratum + Analysis Visit*Treatment.</estimate_desc>
          </analysis>
          <analysis>
            <group_id_list>
              <group_id>O1</group_id>
              <group_id>O3</group_id>
            </group_id_list>
            <non_inferiority_type>Superiority</non_inferiority_type>
            <param_type>Mean Difference (Final Values)</param_type>
            <param_value>0.96</param_value>
            <dispersion_type>Standard Error of the Mean</dispersion_type>
            <dispersion_value>2.36</dispersion_value>
            <ci_percent>95</ci_percent>
            <ci_n_sides>2-Sided</ci_n_sides>
            <ci_lower_limit>-3.71</ci_lower_limit>
            <ci_upper_limit>5.63</ci_upper_limit>
            <estimate_desc>Based on the MMRM using an unstructured covariance matrix - change = Baseline + Age IXRS Stratum + Corticosteroid Regimen IXRS Stratum + Analysis Visit*Treatment.</estimate_desc>
          </analysis>
        </analysis_list>
      </outcome>
      <outcome>
        <type>Secondary</type>
        <title>Change From Baseline at Week 48 in Proximal Lower Extremity Flexor Strength</title>
        <description>Proximal lower extremity flexor (knee extension and knee flexion) strength was measured using manual myometry. A higher score reflects a better outcome. A positive change from baseline indicates an improvement.</description>
        <time_frame>Baseline, Week 48</time_frame>
        <population>Intent-to-Treat (ITT) population included all enrolled participants who received a randomization treatment assignment. Number analyzed is the number of participants with data available for analyses at the given timepoint.</population>
        <group_list>
          <group group_id="O1">
            <title>Placebo</title>
            <description>Participants received matching placebo solution subcutaneously (SC) on specified days of the 48-week double-blind (DB) period. Following the DB period participants received low dose or high dose RO7239361 on specified days for up to 192 weeks during the open-label period followed by 24 weeks of follow-up.</description>
          </group>
          <group group_id="O2">
            <title>RO7239361 Low Dose</title>
            <description>Participants received low dose RO7239361 SC on specified days of the 48-week DB period. Following the DB period participants received low dose RO7239361 on specified days for up to 192 weeks during the open-label period followed by 24 weeks of follow-up.</description>
          </group>
          <group group_id="O3">
            <title>RO7239361 High Dose</title>
            <description>Participants received high dose RO7239361 SC on specified days of the 48-week DB period. Following the DB period participants received high dose RO7239361 on specified days for up to 192 weeks during the open-label period followed by 24 weeks of follow-up.</description>
          </group>
        </group_list>
        <measure>
          <title>Change From Baseline at Week 48 in Proximal Lower Extremity Flexor Strength</title>
          <description>Proximal lower extremity flexor (knee extension and knee flexion) strength was measured using manual myometry. A higher score reflects a better outcome. A positive change from baseline indicates an improvement.</description>
          <population>Intent-to-Treat (ITT) population included all enrolled participants who received a randomization treatment assignment. Number analyzed is the number of participants with data available for analyses at the given timepoint.</population>
          <units>kilogram (kg)</units>
          <param>Mean</param>
          <dispersion>Standard Deviation</dispersion>
          <analyzed_list>
            <analyzed>
              <units>Participants</units>
              <scope>Measure</scope>
              <count_list>
                <count group_id="O1" value="56"/>
                <count group_id="O2" value="55"/>
                <count group_id="O3" value="55"/>
              </count_list>
            </analyzed>
          </analyzed_list>
          <class_list>
            <class>
              <title>Baseline: Knee Extenders</title>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="O1" value="56"/>
                    <count group_id="O2" value="55"/>
                    <count group_id="O3" value="54"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="5.58" spread="2.87"/>
                    <measurement group_id="O2" value="6.25" spread="3.63"/>
                    <measurement group_id="O3" value="5.76" spread="3.53"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Change from Baseline at Week 48: Knee Extenders</title>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="O1" value="30"/>
                    <count group_id="O2" value="28"/>
                    <count group_id="O3" value="33"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="-1.19" spread="2.13"/>
                    <measurement group_id="O2" value="-0.47" spread="2.43"/>
                    <measurement group_id="O3" value="-0.88" spread="2.97"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Baseline: Knee Flexors</title>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="O1" value="56"/>
                    <count group_id="O2" value="55"/>
                    <count group_id="O3" value="54"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="5.04" spread="2.58"/>
                    <measurement group_id="O2" value="5.70" spread="3.08"/>
                    <measurement group_id="O3" value="5.04" spread="2.72"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Change from Baseline at Week 48: Knee Flexors</title>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="O1" value="30"/>
                    <count group_id="O2" value="28"/>
                    <count group_id="O3" value="33"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="0.15" spread="2.24"/>
                    <measurement group_id="O2" value="0.08" spread="2.53"/>
                    <measurement group_id="O3" value="-0.13" spread="2.29"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
      </outcome>
      <outcome>
        <type>Secondary</type>
        <title>Baseline for the 6 Minute Walk Distance (6MWD)</title>
        <description>The 6MWD measured the distance a participant was able to traverse while walking for 6 minutes. A longer distance reflects a better outcome.</description>
        <time_frame>Baseline</time_frame>
        <population>Intent-to-Treat (ITT) population included all enrolled participants who received a randomization treatment assignment.</population>
        <group_list>
          <group group_id="O1">
            <title>Placebo</title>
            <description>Participants received matching placebo solution subcutaneously (SC) on specified days of the 48-week double-blind (DB) period. Following the DB period participants received low dose or high dose RO7239361 on specified days for up to 192 weeks during the open-label period followed by 24 weeks of follow-up.</description>
          </group>
          <group group_id="O2">
            <title>RO7239361 Low Dose</title>
            <description>Participants received low dose RO7239361 SC on specified days of the 48-week DB period. Following the DB period participants received low dose RO7239361 on specified days for up to 192 weeks during the open-label period followed by 24 weeks of follow-up.</description>
          </group>
          <group group_id="O3">
            <title>RO7239361 High Dose</title>
            <description>Participants received high dose RO7239361 SC on specified days of the 48-week DB period. Following the DB period participants received high dose RO7239361 on specified days for up to 192 weeks during the open-label period followed by 24 weeks of follow-up.</description>
          </group>
        </group_list>
        <measure>
          <title>Baseline for the 6 Minute Walk Distance (6MWD)</title>
          <description>The 6MWD measured the distance a participant was able to traverse while walking for 6 minutes. A longer distance reflects a better outcome.</description>
          <population>Intent-to-Treat (ITT) population included all enrolled participants who received a randomization treatment assignment.</population>
          <units>meters (m)</units>
          <param>Mean</param>
          <dispersion>Standard Deviation</dispersion>
          <analyzed_list>
            <analyzed>
              <units>Participants</units>
              <scope>Measure</scope>
              <count_list>
                <count group_id="O1" value="56"/>
                <count group_id="O2" value="55"/>
                <count group_id="O3" value="55"/>
              </count_list>
            </analyzed>
          </analyzed_list>
          <class_list>
            <class>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="388.33" spread="69.59"/>
                    <measurement group_id="O2" value="399.73" spread="68.35"/>
                    <measurement group_id="O3" value="370.73" spread="93.35"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
      </outcome>
      <outcome>
        <type>Secondary</type>
        <title>Change From Baseline at Week 48 in 6 Minute Walk Distance (6MWD)</title>
        <description>The 6MWD measured the distance a participant was able to traverse while walking for 6 minutes. A longer distance reflects a better outcome. A positive change from baseline indicates an improvement. Based on the mixed-effect model of repeated measures (MMRM).</description>
        <time_frame>Baseline, Week 48</time_frame>
        <population>Intent-to-Treat (ITT) population included all enrolled participants who received a randomization treatment assignment. MMRM analysis included all participants at baseline and the following number of participants by Week 48: Placebo n=29, Low Dose n=25, High Dose n=31.</population>
        <group_list>
          <group group_id="O1">
            <title>Placebo</title>
            <description>Participants received matching placebo solution subcutaneously (SC) on specified days of the 48-week double-blind (DB) period. Following the DB period participants received low dose or high dose RO7239361 on specified days for up to 192 weeks during the open-label period followed by 24 weeks of follow-up.</description>
          </group>
          <group group_id="O2">
            <title>RO7239361 Low Dose</title>
            <description>Participants received low dose RO7239361 SC on specified days of the 48-week DB period. Following the DB period participants received low dose RO7239361 on specified days for up to 192 weeks during the open-label period followed by 24 weeks of follow-up.</description>
          </group>
          <group group_id="O3">
            <title>RO7239361 High Dose</title>
            <description>Participants received high dose RO7239361 SC on specified days of the 48-week DB period. Following the DB period participants received high dose RO7239361 on specified days for up to 192 weeks during the open-label period followed by 24 weeks of follow-up.</description>
          </group>
        </group_list>
        <measure>
          <title>Change From Baseline at Week 48 in 6 Minute Walk Distance (6MWD)</title>
          <description>The 6MWD measured the distance a participant was able to traverse while walking for 6 minutes. A longer distance reflects a better outcome. A positive change from baseline indicates an improvement. Based on the mixed-effect model of repeated measures (MMRM).</description>
          <population>Intent-to-Treat (ITT) population included all enrolled participants who received a randomization treatment assignment. MMRM analysis included all participants at baseline and the following number of participants by Week 48: Placebo n=29, Low Dose n=25, High Dose n=31.</population>
          <units>meters (m)</units>
          <param>Least Squares Mean</param>
          <dispersion>Standard Error</dispersion>
          <analyzed_list>
            <analyzed>
              <units>Participants</units>
              <scope>Measure</scope>
              <count_list>
                <count group_id="O1" value="56"/>
                <count group_id="O2" value="55"/>
                <count group_id="O3" value="55"/>
              </count_list>
            </analyzed>
          </analyzed_list>
          <class_list>
            <class>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="-41.3" spread="8.7"/>
                    <measurement group_id="O2" value="-39.6" spread="9.0"/>
                    <measurement group_id="O3" value="-30.0" spread="8.7"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
        <analysis_list>
          <analysis>
            <group_id_list>
              <group_id>O1</group_id>
              <group_id>O2</group_id>
            </group_id_list>
            <non_inferiority_type>Superiority</non_inferiority_type>
            <param_type>Mean Difference (Final Values)</param_type>
            <param_value>1.7</param_value>
            <dispersion_type>Standard Error of the Mean</dispersion_type>
            <dispersion_value>11.5</dispersion_value>
            <ci_percent>95</ci_percent>
            <ci_n_sides>2-Sided</ci_n_sides>
            <ci_lower_limit>-21.1</ci_lower_limit>
            <ci_upper_limit>24.6</ci_upper_limit>
            <estimate_desc>Based on the MMRM using an unstructured covariance matrix - change = Baseline + Age IXRS Stratum + Corticosteroid Regimen IXRS Stratum + Analysis Visit*Treatment.</estimate_desc>
          </analysis>
          <analysis>
            <group_id_list>
              <group_id>O1</group_id>
              <group_id>O3</group_id>
            </group_id_list>
            <non_inferiority_type>Superiority</non_inferiority_type>
            <param_type>Mean Difference (Final Values)</param_type>
            <param_value>11.3</param_value>
            <dispersion_type>Standard Error of the Mean</dispersion_type>
            <dispersion_value>11.3</dispersion_value>
            <ci_percent>95</ci_percent>
            <ci_n_sides>2-Sided</ci_n_sides>
            <ci_lower_limit>-11.0</ci_lower_limit>
            <ci_upper_limit>33.6</ci_upper_limit>
            <estimate_desc>Based on the MMRM using an unstructured covariance matrix - change = Baseline + Age IXRS Stratum + Corticosteroid Regimen IXRS Stratum + Analysis Visit*Treatment.</estimate_desc>
          </analysis>
        </analysis_list>
      </outcome>
      <outcome>
        <type>Secondary</type>
        <title>Percentage of Participants for Each Clinical Global Impression of Change (CGI-C) Assessment Status at Week 48</title>
        <description>The CGI-C was used to assess the participant's overall condition on a 7-point scale, using the status markers &quot;very much improved, much improved, slightly improved, no change, slightly worse, much worse or very much worse&quot; at Week 48 as compared to baseline.</description>
        <time_frame>Baseline, Week 48</time_frame>
        <population>Intent-to-Treat (ITT) population included all enrolled participants who received a randomization treatment assignment. Included in the analysis are only those subjects for whom an efficacy assessment was completed at Week 48.</population>
        <group_list>
          <group group_id="O1">
            <title>Placebo</title>
            <description>Participants received matching placebo solution subcutaneously (SC) on specified days of the 48-week double-blind (DB) period. Following the DB period participants received low dose or high dose RO7239361 on specified days for up to 192 weeks during the open-label period followed by 24 weeks of follow-up.</description>
          </group>
          <group group_id="O2">
            <title>RO7239361 Low Dose</title>
            <description>Participants received low dose RO7239361 SC on specified days of the 48-week DB period. Following the DB period participants received low dose RO7239361 on specified days for up to 192 weeks during the open-label period followed by 24 weeks of follow-up.</description>
          </group>
          <group group_id="O3">
            <title>RO7239361 High Dose</title>
            <description>Participants received high dose RO7239361 SC on specified days of the 48-week DB period. Following the DB period participants received high dose RO7239361 on specified days for up to 192 weeks during the open-label period followed by 24 weeks of follow-up.</description>
          </group>
        </group_list>
        <measure>
          <title>Percentage of Participants for Each Clinical Global Impression of Change (CGI-C) Assessment Status at Week 48</title>
          <description>The CGI-C was used to assess the participant's overall condition on a 7-point scale, using the status markers &quot;very much improved, much improved, slightly improved, no change, slightly worse, much worse or very much worse&quot; at Week 48 as compared to baseline.</description>
          <population>Intent-to-Treat (ITT) population included all enrolled participants who received a randomization treatment assignment. Included in the analysis are only those subjects for whom an efficacy assessment was completed at Week 48.</population>
          <units>percentage of participants</units>
          <param>Number</param>
          <analyzed_list>
            <analyzed>
              <units>Participants</units>
              <scope>Measure</scope>
              <count_list>
                <count group_id="O1" value="36"/>
                <count group_id="O2" value="37"/>
                <count group_id="O3" value="31"/>
              </count_list>
            </analyzed>
          </analyzed_list>
          <class_list>
            <class>
              <title>Very much improved</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="0"/>
                    <measurement group_id="O2" value="0"/>
                    <measurement group_id="O3" value="0"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Much improved</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="5.6"/>
                    <measurement group_id="O2" value="2.7"/>
                    <measurement group_id="O3" value="3.2"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Minimally improved</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="13.9"/>
                    <measurement group_id="O2" value="13.5"/>
                    <measurement group_id="O3" value="19.4"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>No change</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="58.3"/>
                    <measurement group_id="O2" value="54.1"/>
                    <measurement group_id="O3" value="51.6"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Minimally worse</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="16.7"/>
                    <measurement group_id="O2" value="18.9"/>
                    <measurement group_id="O3" value="22.6"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Much worse</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="5.6"/>
                    <measurement group_id="O2" value="10.8"/>
                    <measurement group_id="O3" value="3.2"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Very much worse</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="0"/>
                    <measurement group_id="O2" value="0"/>
                    <measurement group_id="O3" value="0"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
      </outcome>
      <outcome>
        <type>Secondary</type>
        <title>Change From Baseline at Week 48 in 95th Percentile Stride Velocity</title>
        <description>Stride velocity was recorded with the ActiMyo device in a subset of the overall study population. The ActiMyo device measures the daily movement and activity levels of the participant. The device consists of two sensors worn on each ankle. A higher velocity reflects a better outcome. A positive change from baseline indicates an improvement.</description>
        <time_frame>Baseline, Week 48</time_frame>
        <population>Intent-to-Treat (ITT) population included all enrolled participants who received a randomization treatment assignment.</population>
        <group_list>
          <group group_id="O1">
            <title>Placebo</title>
            <description>Participants received matching placebo solution subcutaneously (SC) on specified days of the 48-week double-blind (DB) period. Following the DB period participants received low dose or high dose RO7239361 on specified days for up to 192 weeks during the open-label period followed by 24 weeks of follow-up.</description>
          </group>
          <group group_id="O2">
            <title>RO7239361 Low Dose</title>
            <description>Participants received low dose RO7239361 SC on specified days of the 48-week DB period. Following the DB period participants received low dose RO7239361 on specified days for up to 192 weeks during the open-label period followed by 24 weeks of follow-up.</description>
          </group>
          <group group_id="O3">
            <title>RO7239361 High Dose</title>
            <description>Participants received high dose RO7239361 SC on specified days of the 48-week DB period. Following the DB period participants received high dose RO7239361 on specified days for up to 192 weeks during the open-label period followed by 24 weeks of follow-up.</description>
          </group>
        </group_list>
        <measure>
          <title>Change From Baseline at Week 48 in 95th Percentile Stride Velocity</title>
          <description>Stride velocity was recorded with the ActiMyo device in a subset of the overall study population. The ActiMyo device measures the daily movement and activity levels of the participant. The device consists of two sensors worn on each ankle. A higher velocity reflects a better outcome. A positive change from baseline indicates an improvement.</description>
          <population>Intent-to-Treat (ITT) population included all enrolled participants who received a randomization treatment assignment.</population>
          <units>m/sec</units>
          <param>Mean</param>
          <dispersion>Standard Deviation</dispersion>
          <analyzed_list>
            <analyzed>
              <units>Participants</units>
              <scope>Measure</scope>
              <count_list>
                <count group_id="O1" value="17"/>
                <count group_id="O2" value="19"/>
                <count group_id="O3" value="15"/>
              </count_list>
            </analyzed>
          </analyzed_list>
          <class_list>
            <class>
              <title>Baseline</title>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="O1" value="17"/>
                    <count group_id="O2" value="19"/>
                    <count group_id="O3" value="15"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="1.69" spread="0.33"/>
                    <measurement group_id="O2" value="1.54" spread="0.35"/>
                    <measurement group_id="O3" value="1.57" spread="0.46"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Change from Baseline at Week 48</title>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="O1" value="5"/>
                    <count group_id="O2" value="7"/>
                    <count group_id="O3" value="4"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="-0.25" spread="0.39"/>
                    <measurement group_id="O2" value="-0.22" spread="0.22"/>
                    <measurement group_id="O3" value="-0.28" spread="0.29"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
      </outcome>
      <outcome>
        <type>Secondary</type>
        <title>Number of Participants With Adverse Events (AEs)</title>
        <description>An adverse event is any untoward medical occurrence in a subject administered a pharmaceutical product and which does not necessarily have to have a causal relationship with the treatment. An adverse event can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding, for example), symptom, or disease temporally associated with the use of a pharmaceutical product, whether or not considered related to the pharmaceutical product. Preexisting conditions which worsen during a study are also considered as adverse events.</description>
        <time_frame>During DB period (48 weeks) and Whole study (up to approximately 34 months)</time_frame>
        <population>Safety population included all enrolled participants who received at least 1 dose of study therapy. Data are presented for the arms in the DB period as well as for all RO7239361-treated participants in the whole study.</population>
        <group_list>
          <group group_id="O1">
            <title>Placebo DB</title>
            <description>Participants received matching placebo solution subcutaneously (SC) on specified days of the 48-week double-blind (DB) period.</description>
          </group>
          <group group_id="O2">
            <title>RO7239361 Low Dose DB</title>
            <description>Participants received low dose RO7239361 SC on specified days of the 48-week DB period.</description>
          </group>
          <group group_id="O3">
            <title>RO7239361 High Dose DB</title>
            <description>Participants received high dose RO7239361 SC on specified days of the 48-week DB period.</description>
          </group>
          <group group_id="O4">
            <title>RO7239361 Low Dose</title>
            <description>Participants received low dose RO7239361 SC on specified days of the 48-week DB period. Following the DB period participants received low dose RO7239361 on specified days for up to 192 weeks during the open-label period followed by 24 weeks of follow-up.</description>
          </group>
          <group group_id="O5">
            <title>RO7239361 High Dose Whole Study</title>
            <description>Participants received high dose SC on specified days of the 48-week DB period. Following the DB period participants received high dose RO7239361 on specified days for up to 192 weeks during the open-label period followed by 24 weeks of follow-up.</description>
          </group>
        </group_list>
        <measure>
          <title>Number of Participants With Adverse Events (AEs)</title>
          <description>An adverse event is any untoward medical occurrence in a subject administered a pharmaceutical product and which does not necessarily have to have a causal relationship with the treatment. An adverse event can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding, for example), symptom, or disease temporally associated with the use of a pharmaceutical product, whether or not considered related to the pharmaceutical product. Preexisting conditions which worsen during a study are also considered as adverse events.</description>
          <population>Safety population included all enrolled participants who received at least 1 dose of study therapy. Data are presented for the arms in the DB period as well as for all RO7239361-treated participants in the whole study.</population>
          <units>participants</units>
          <param>Number</param>
          <analyzed_list>
            <analyzed>
              <units>Participants</units>
              <scope>Measure</scope>
              <count_list>
                <count group_id="O1" value="56"/>
                <count group_id="O2" value="55"/>
                <count group_id="O3" value="55"/>
                <count group_id="O4" value="69"/>
                <count group_id="O5" value="68"/>
              </count_list>
            </analyzed>
          </analyzed_list>
          <class_list>
            <class>
              <title>Double-Blind Period</title>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="O1" value="56"/>
                    <count group_id="O2" value="55"/>
                    <count group_id="O3" value="55"/>
                    <count group_id="O4" value="0"/>
                    <count group_id="O5" value="0"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="46"/>
                    <measurement group_id="O2" value="48"/>
                    <measurement group_id="O3" value="49"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Whole Study</title>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="O1" value="0"/>
                    <count group_id="O2" value="0"/>
                    <count group_id="O3" value="0"/>
                    <count group_id="O4" value="69"/>
                    <count group_id="O5" value="68"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O4" value="57"/>
                    <measurement group_id="O5" value="56"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
      </outcome>
      <outcome>
        <type>Secondary</type>
        <title>Number of Participants With AEs Leading to Discontinuation</title>
        <description>An adverse event is any untoward medical occurrence in a subject administered a pharmaceutical product and which does not necessarily have to have a causal relationship with the treatment. An adverse event can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding, for example), symptom, or disease temporally associated with the use of a pharmaceutical product, whether or not considered related to the pharmaceutical product. Preexisting conditions which worsen during a study are also considered as adverse events. Reported here is the number of participants with AEs that led to study discontinuation.</description>
        <time_frame>During DB period (48 weeks) and Whole study (up to approximately 34 months)</time_frame>
        <population>Safety population included all enrolled participants who received at least 1 dose of study therapy. Data are presented for the arms in the DB period as well as for all RO7239361-treated participants in the whole study.</population>
        <group_list>
          <group group_id="O1">
            <title>Placebo DB</title>
            <description>Participants received matching placebo solution subcutaneously (SC) on specified days of the 48-week double-blind (DB) period.</description>
          </group>
          <group group_id="O2">
            <title>RO7239361 Low Dose DB</title>
            <description>Participants received low dose RO7239361 SC on specified days of the 48-week DB period.</description>
          </group>
          <group group_id="O3">
            <title>RO7239361 High Dose DB</title>
            <description>Participants received high dose RO7239361 SC on specified days of the 48-week DB period.</description>
          </group>
          <group group_id="O4">
            <title>RO7239361 Low Dose</title>
            <description>Participants received low dose RO7239361 SC on specified days of the 48-week DB period. Following the DB period participants received low dose RO7239361 on specified days for up to 192 weeks during the open-label period followed by 24 weeks of follow-up.</description>
          </group>
          <group group_id="O5">
            <title>RO7239361 High Dose</title>
            <description>Participants received high dose RO7239361 SC on specified days of the 48-week DB period. Following the DB period participants received high dose RO7239361 on specified days for up to 192 weeks during the open-label period followed by 24 weeks of follow-up.</description>
          </group>
        </group_list>
        <measure>
          <title>Number of Participants With AEs Leading to Discontinuation</title>
          <description>An adverse event is any untoward medical occurrence in a subject administered a pharmaceutical product and which does not necessarily have to have a causal relationship with the treatment. An adverse event can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding, for example), symptom, or disease temporally associated with the use of a pharmaceutical product, whether or not considered related to the pharmaceutical product. Preexisting conditions which worsen during a study are also considered as adverse events. Reported here is the number of participants with AEs that led to study discontinuation.</description>
          <population>Safety population included all enrolled participants who received at least 1 dose of study therapy. Data are presented for the arms in the DB period as well as for all RO7239361-treated participants in the whole study.</population>
          <units>participants</units>
          <param>Number</param>
          <analyzed_list>
            <analyzed>
              <units>Participants</units>
              <scope>Measure</scope>
              <count_list>
                <count group_id="O1" value="56"/>
                <count group_id="O2" value="55"/>
                <count group_id="O3" value="55"/>
                <count group_id="O4" value="69"/>
                <count group_id="O5" value="68"/>
              </count_list>
            </analyzed>
          </analyzed_list>
          <class_list>
            <class>
              <title>Double-Blind Period</title>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="O1" value="56"/>
                    <count group_id="O2" value="55"/>
                    <count group_id="O3" value="55"/>
                    <count group_id="O4" value="0"/>
                    <count group_id="O5" value="0"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="0"/>
                    <measurement group_id="O2" value="0"/>
                    <measurement group_id="O3" value="0"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Whole Study</title>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="O1" value="0"/>
                    <count group_id="O2" value="0"/>
                    <count group_id="O3" value="0"/>
                    <count group_id="O4" value="69"/>
                    <count group_id="O5" value="68"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O4" value="0"/>
                    <measurement group_id="O5" value="0"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
      </outcome>
    </outcome_list>

    <reported_events>
      <time_frame>Up to approximately 34 months</time_frame>
      <desc>Safety population included all enrolled participants who received at least 1 dose of study therapy.</desc>
      <group_list>
        <group group_id="E1">
          <title>Placebo DB</title>
          <description>Participants received matching placebo solution subcutaneously (SC) on specified days of the 48-week double-blind (DB) period.</description>
        </group>
        <group group_id="E2">
          <title>RO7239361 Low Dose DB</title>
          <description>Participants received low dose RO7239361 SC on specified days of the 48-week DB period.</description>
        </group>
        <group group_id="E3">
          <title>RO7239361 High Dose DB</title>
          <description>Participants received high dose RO7239361 SC on specified days of the 48-week DB period.</description>
        </group>
        <group group_id="E4">
          <title>Placebo, Then RO7239361 Low Dose OL</title>
          <description>Participants received matching placebo solution SC on specified days of the 48-week DB period. Following the DB period participants received low dose RO7239361 on specified days for up to 192 weeks during the open-label (OL) period followed by 24 weeks of follow-up.</description>
        </group>
        <group group_id="E5">
          <title>Placebo, Then RO7239361 High Dose OL</title>
          <description>Participants received matching placebo solution SC on specified days of the 48-week DB period. Following the DB period participants received high dose RO7239361 on specified days for up to 192 weeks during the open-label period followed by 24 weeks of follow-up.</description>
        </group>
        <group group_id="E6">
          <title>RO7239361 Low Dose, Then RO7239361 Low Dose OL</title>
          <description>Participants received low dose RO7239361 SC on specified days of the 48-week DB period. Following the DB period participants received low dose RO7239361 on specified days for up to 192 weeks during the open-label period followed by 24 weeks of follow-up.</description>
        </group>
        <group group_id="E7">
          <title>RO7239361 High Dose, Then RO7239361 High Dose OL</title>
          <description>Participants received high dose RO7239361 SC on specified days of the 48-week DB period. Following the DB period participants received high dose RO7239361 on specified days for up to 192 weeks during the open-label period followed by 24 weeks of follow-up</description>
        </group>
      </group_list>
      <serious_events>
        <default_vocab>MedDRA 23.0</default_vocab>
        <default_assessment>Systematic Assessment</default_assessment>
        <category_list>
          <category>
            <title>Total</title>
            <event_list>
              <event>
                <sub_title>Total, all-cause mortality</sub_title>
                <counts group_id="E1" subjects_affected="0" subjects_at_risk="56"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="55"/>
                <counts group_id="E3" subjects_affected="1" subjects_at_risk="55"/>
                <counts group_id="E4" subjects_affected="0" subjects_at_risk="14"/>
                <counts group_id="E5" subjects_affected="0" subjects_at_risk="13"/>
                <counts group_id="E6" subjects_affected="0" subjects_at_risk="24"/>
                <counts group_id="E7" subjects_affected="0" subjects_at_risk="29"/>
              </event>
              <event>
                <sub_title>Total, serious adverse events</sub_title>
                <counts group_id="E1" subjects_affected="3" subjects_at_risk="56"/>
                <counts group_id="E2" subjects_affected="2" subjects_at_risk="55"/>
                <counts group_id="E3" subjects_affected="4" subjects_at_risk="55"/>
                <counts group_id="E4" subjects_affected="0" subjects_at_risk="14"/>
                <counts group_id="E5" subjects_affected="0" subjects_at_risk="13"/>
                <counts group_id="E6" subjects_affected="0" subjects_at_risk="24"/>
                <counts group_id="E7" subjects_affected="0" subjects_at_risk="29"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Cardiac disorders</title>
            <event_list>
              <event>
                <sub_title>Cardiac arrest</sub_title>
                <counts group_id="E1" events="0" subjects_affected="0" subjects_at_risk="56"/>
                <counts group_id="E2" events="0" subjects_affected="0" subjects_at_risk="55"/>
                <counts group_id="E3" events="1" subjects_affected="1" subjects_at_risk="55"/>
                <counts group_id="E4" events="0" subjects_affected="0" subjects_at_risk="14"/>
                <counts group_id="E5" events="0" subjects_affected="0" subjects_at_risk="13"/>
                <counts group_id="E6" events="0" subjects_affected="0" subjects_at_risk="24"/>
                <counts group_id="E7" events="0" subjects_affected="0" subjects_at_risk="29"/>
              </event>
              <event>
                <sub_title>Myocarditis</sub_title>
                <counts group_id="E1" events="1" subjects_affected="1" subjects_at_risk="56"/>
                <counts group_id="E2" events="0" subjects_affected="0" subjects_at_risk="55"/>
                <counts group_id="E3" events="0" subjects_affected="0" subjects_at_risk="55"/>
                <counts group_id="E4" events="0" subjects_affected="0" subjects_at_risk="14"/>
                <counts group_id="E5" events="0" subjects_affected="0" subjects_at_risk="13"/>
                <counts group_id="E6" events="0" subjects_affected="0" subjects_at_risk="24"/>
                <counts group_id="E7" events="0" subjects_affected="0" subjects_at_risk="29"/>
              </event>
              <event>
                <sub_title>Supraventricular tachycardia</sub_title>
                <counts group_id="E1" events="0" subjects_affected="0" subjects_at_risk="56"/>
                <counts group_id="E2" events="0" subjects_affected="0" subjects_at_risk="55"/>
                <counts group_id="E3" events="1" subjects_affected="1" subjects_at_risk="55"/>
                <counts group_id="E4" events="0" subjects_affected="0" subjects_at_risk="14"/>
                <counts group_id="E5" events="0" subjects_affected="0" subjects_at_risk="13"/>
                <counts group_id="E6" events="0" subjects_affected="0" subjects_at_risk="24"/>
                <counts group_id="E7" events="0" subjects_affected="0" subjects_at_risk="29"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>General disorders</title>
            <event_list>
              <event>
                <sub_title>Influenza like illness</sub_title>
                <counts group_id="E1" events="1" subjects_affected="1" subjects_at_risk="56"/>
                <counts group_id="E2" events="0" subjects_affected="0" subjects_at_risk="55"/>
                <counts group_id="E3" events="0" subjects_affected="0" subjects_at_risk="55"/>
                <counts group_id="E4" events="0" subjects_affected="0" subjects_at_risk="14"/>
                <counts group_id="E5" events="0" subjects_affected="0" subjects_at_risk="13"/>
                <counts group_id="E6" events="0" subjects_affected="0" subjects_at_risk="24"/>
                <counts group_id="E7" events="0" subjects_affected="0" subjects_at_risk="29"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Hepatobiliary disorders</title>
            <event_list>
              <event>
                <sub_title>Hyperbilirubinaemia</sub_title>
                <counts group_id="E1" events="0" subjects_affected="0" subjects_at_risk="56"/>
                <counts group_id="E2" events="0" subjects_affected="0" subjects_at_risk="55"/>
                <counts group_id="E3" events="1" subjects_affected="1" subjects_at_risk="55"/>
                <counts group_id="E4" events="0" subjects_affected="0" subjects_at_risk="14"/>
                <counts group_id="E5" events="0" subjects_affected="0" subjects_at_risk="13"/>
                <counts group_id="E6" events="0" subjects_affected="0" subjects_at_risk="24"/>
                <counts group_id="E7" events="0" subjects_affected="0" subjects_at_risk="29"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Infections and infestations</title>
            <event_list>
              <event>
                <sub_title>Gastrointestinal viral infection</sub_title>
                <counts group_id="E1" events="1" subjects_affected="1" subjects_at_risk="56"/>
                <counts group_id="E2" events="0" subjects_affected="0" subjects_at_risk="55"/>
                <counts group_id="E3" events="0" subjects_affected="0" subjects_at_risk="55"/>
                <counts group_id="E4" events="0" subjects_affected="0" subjects_at_risk="14"/>
                <counts group_id="E5" events="0" subjects_affected="0" subjects_at_risk="13"/>
                <counts group_id="E6" events="0" subjects_affected="0" subjects_at_risk="24"/>
                <counts group_id="E7" events="0" subjects_affected="0" subjects_at_risk="29"/>
              </event>
              <event>
                <sub_title>Viral infection</sub_title>
                <counts group_id="E1" events="0" subjects_affected="0" subjects_at_risk="56"/>
                <counts group_id="E2" events="1" subjects_affected="1" subjects_at_risk="55"/>
                <counts group_id="E3" events="0" subjects_affected="0" subjects_at_risk="55"/>
                <counts group_id="E4" events="0" subjects_affected="0" subjects_at_risk="14"/>
                <counts group_id="E5" events="0" subjects_affected="0" subjects_at_risk="13"/>
                <counts group_id="E6" events="0" subjects_affected="0" subjects_at_risk="24"/>
                <counts group_id="E7" events="0" subjects_affected="0" subjects_at_risk="29"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Injury, poisoning and procedural complications</title>
            <event_list>
              <event>
                <sub_title>Humerus fracture</sub_title>
                <counts group_id="E1" events="0" subjects_affected="0" subjects_at_risk="56"/>
                <counts group_id="E2" events="1" subjects_affected="1" subjects_at_risk="55"/>
                <counts group_id="E3" events="0" subjects_affected="0" subjects_at_risk="55"/>
                <counts group_id="E4" events="0" subjects_affected="0" subjects_at_risk="14"/>
                <counts group_id="E5" events="0" subjects_affected="0" subjects_at_risk="13"/>
                <counts group_id="E6" events="0" subjects_affected="0" subjects_at_risk="24"/>
                <counts group_id="E7" events="0" subjects_affected="0" subjects_at_risk="29"/>
              </event>
              <event>
                <sub_title>Overdose</sub_title>
                <counts group_id="E1" events="0" subjects_affected="0" subjects_at_risk="56"/>
                <counts group_id="E2" events="0" subjects_affected="0" subjects_at_risk="55"/>
                <counts group_id="E3" events="1" subjects_affected="1" subjects_at_risk="55"/>
                <counts group_id="E4" events="0" subjects_affected="0" subjects_at_risk="14"/>
                <counts group_id="E5" events="0" subjects_affected="0" subjects_at_risk="13"/>
                <counts group_id="E6" events="0" subjects_affected="0" subjects_at_risk="24"/>
                <counts group_id="E7" events="0" subjects_affected="0" subjects_at_risk="29"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Metabolism and nutrition disorders</title>
            <event_list>
              <event>
                <sub_title>Hypocalcaemia</sub_title>
                <counts group_id="E1" events="1" subjects_affected="1" subjects_at_risk="56"/>
                <counts group_id="E2" events="0" subjects_affected="0" subjects_at_risk="55"/>
                <counts group_id="E3" events="0" subjects_affected="0" subjects_at_risk="55"/>
                <counts group_id="E4" events="0" subjects_affected="0" subjects_at_risk="14"/>
                <counts group_id="E5" events="0" subjects_affected="0" subjects_at_risk="13"/>
                <counts group_id="E6" events="0" subjects_affected="0" subjects_at_risk="24"/>
                <counts group_id="E7" events="0" subjects_affected="0" subjects_at_risk="29"/>
              </event>
            </event_list>
          </category>
        </category_list>
      </serious_events>
      <other_events>
        <frequency_threshold>5</frequency_threshold>
        <default_vocab>MedDRA 23.0</default_vocab>
        <default_assessment>Systematic Assessment</default_assessment>
        <category_list>
          <category>
            <title>Total</title>
            <event_list>
              <event>
                <sub_title>Total, other adverse events</sub_title>
                <counts group_id="E1" subjects_affected="43" subjects_at_risk="56"/>
                <counts group_id="E2" subjects_affected="43" subjects_at_risk="55"/>
                <counts group_id="E3" subjects_affected="47" subjects_at_risk="55"/>
                <counts group_id="E4" subjects_affected="8" subjects_at_risk="14"/>
                <counts group_id="E5" subjects_affected="7" subjects_at_risk="13"/>
                <counts group_id="E6" subjects_affected="13" subjects_at_risk="24"/>
                <counts group_id="E7" subjects_affected="12" subjects_at_risk="29"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Ear and labyrinth disorders</title>
            <event_list>
              <event>
                <sub_title>Ear pain</sub_title>
                <counts group_id="E1" events="1" subjects_affected="1" subjects_at_risk="56"/>
                <counts group_id="E2" events="1" subjects_affected="1" subjects_at_risk="55"/>
                <counts group_id="E3" events="7" subjects_affected="4" subjects_at_risk="55"/>
                <counts group_id="E4" events="0" subjects_affected="0" subjects_at_risk="14"/>
                <counts group_id="E5" events="0" subjects_affected="0" subjects_at_risk="13"/>
                <counts group_id="E6" events="0" subjects_affected="0" subjects_at_risk="24"/>
                <counts group_id="E7" events="0" subjects_affected="0" subjects_at_risk="29"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Gastrointestinal disorders</title>
            <event_list>
              <event>
                <sub_title>Abdominal discomfort</sub_title>
                <counts group_id="E1" events="2" subjects_affected="1" subjects_at_risk="56"/>
                <counts group_id="E2" events="1" subjects_affected="1" subjects_at_risk="55"/>
                <counts group_id="E3" events="1" subjects_affected="1" subjects_at_risk="55"/>
                <counts group_id="E4" events="0" subjects_affected="0" subjects_at_risk="14"/>
                <counts group_id="E5" events="1" subjects_affected="1" subjects_at_risk="13"/>
                <counts group_id="E6" events="0" subjects_affected="0" subjects_at_risk="24"/>
                <counts group_id="E7" events="0" subjects_affected="0" subjects_at_risk="29"/>
              </event>
              <event>
                <sub_title>Abdominal pain</sub_title>
                <counts group_id="E1" events="1" subjects_affected="1" subjects_at_risk="56"/>
                <counts group_id="E2" events="2" subjects_affected="2" subjects_at_risk="55"/>
                <counts group_id="E3" events="5" subjects_affected="3" subjects_at_risk="55"/>
                <counts group_id="E4" events="0" subjects_affected="0" subjects_at_risk="14"/>
                <counts group_id="E5" events="0" subjects_affected="0" subjects_at_risk="13"/>
                <counts group_id="E6" events="0" subjects_affected="0" subjects_at_risk="24"/>
                <counts group_id="E7" events="0" subjects_affected="0" subjects_at_risk="29"/>
              </event>
              <event>
                <sub_title>Abdominal pain upper</sub_title>
                <counts group_id="E1" events="4" subjects_affected="3" subjects_at_risk="56"/>
                <counts group_id="E2" events="5" subjects_affected="4" subjects_at_risk="55"/>
                <counts group_id="E3" events="12" subjects_affected="7" subjects_at_risk="55"/>
                <counts group_id="E4" events="0" subjects_affected="0" subjects_at_risk="14"/>
                <counts group_id="E5" events="0" subjects_affected="0" subjects_at_risk="13"/>
                <counts group_id="E6" events="1" subjects_affected="1" subjects_at_risk="24"/>
                <counts group_id="E7" events="2" subjects_affected="1" subjects_at_risk="29"/>
              </event>
              <event>
                <sub_title>Constipation</sub_title>
                <counts group_id="E1" events="5" subjects_affected="4" subjects_at_risk="56"/>
                <counts group_id="E2" events="4" subjects_affected="4" subjects_at_risk="55"/>
                <counts group_id="E3" events="1" subjects_affected="1" subjects_at_risk="55"/>
                <counts group_id="E4" events="0" subjects_affected="0" subjects_at_risk="14"/>
                <counts group_id="E5" events="0" subjects_affected="0" subjects_at_risk="13"/>
                <counts group_id="E6" events="1" subjects_affected="1" subjects_at_risk="24"/>
                <counts group_id="E7" events="0" subjects_affected="0" subjects_at_risk="29"/>
              </event>
              <event>
                <sub_title>Diarrhoea</sub_title>
                <counts group_id="E1" events="3" subjects_affected="3" subjects_at_risk="56"/>
                <counts group_id="E2" events="13" subjects_affected="10" subjects_at_risk="55"/>
                <counts group_id="E3" events="8" subjects_affected="4" subjects_at_risk="55"/>
                <counts group_id="E4" events="0" subjects_affected="0" subjects_at_risk="14"/>
                <counts group_id="E5" events="0" subjects_affected="0" subjects_at_risk="13"/>
                <counts group_id="E6" events="1" subjects_affected="1" subjects_at_risk="24"/>
                <counts group_id="E7" events="2" subjects_affected="2" subjects_at_risk="29"/>
              </event>
              <event>
                <sub_title>Dyspepsia</sub_title>
                <counts group_id="E1" events="0" subjects_affected="0" subjects_at_risk="56"/>
                <counts group_id="E2" events="1" subjects_affected="1" subjects_at_risk="55"/>
                <counts group_id="E3" events="4" subjects_affected="3" subjects_at_risk="55"/>
                <counts group_id="E4" events="0" subjects_affected="0" subjects_at_risk="14"/>
                <counts group_id="E5" events="0" subjects_affected="0" subjects_at_risk="13"/>
                <counts group_id="E6" events="0" subjects_affected="0" subjects_at_risk="24"/>
                <counts group_id="E7" events="0" subjects_affected="0" subjects_at_risk="29"/>
              </event>
              <event>
                <sub_title>Nausea</sub_title>
                <counts group_id="E1" events="1" subjects_affected="1" subjects_at_risk="56"/>
                <counts group_id="E2" events="2" subjects_affected="2" subjects_at_risk="55"/>
                <counts group_id="E3" events="9" subjects_affected="5" subjects_at_risk="55"/>
                <counts group_id="E4" events="0" subjects_affected="0" subjects_at_risk="14"/>
                <counts group_id="E5" events="0" subjects_affected="0" subjects_at_risk="13"/>
                <counts group_id="E6" events="0" subjects_affected="0" subjects_at_risk="24"/>
                <counts group_id="E7" events="4" subjects_affected="4" subjects_at_risk="29"/>
              </event>
              <event>
                <sub_title>Vomiting</sub_title>
                <counts group_id="E1" events="6" subjects_affected="6" subjects_at_risk="56"/>
                <counts group_id="E2" events="14" subjects_affected="8" subjects_at_risk="55"/>
                <counts group_id="E3" events="11" subjects_affected="6" subjects_at_risk="55"/>
                <counts group_id="E4" events="0" subjects_affected="0" subjects_at_risk="14"/>
                <counts group_id="E5" events="0" subjects_affected="0" subjects_at_risk="13"/>
                <counts group_id="E6" events="4" subjects_affected="3" subjects_at_risk="24"/>
                <counts group_id="E7" events="2" subjects_affected="2" subjects_at_risk="29"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>General disorders</title>
            <event_list>
              <event>
                <sub_title>Fatigue</sub_title>
                <counts group_id="E1" events="0" subjects_affected="0" subjects_at_risk="56"/>
                <counts group_id="E2" events="4" subjects_affected="4" subjects_at_risk="55"/>
                <counts group_id="E3" events="0" subjects_affected="0" subjects_at_risk="55"/>
                <counts group_id="E4" events="0" subjects_affected="0" subjects_at_risk="14"/>
                <counts group_id="E5" events="0" subjects_affected="0" subjects_at_risk="13"/>
                <counts group_id="E6" events="1" subjects_affected="1" subjects_at_risk="24"/>
                <counts group_id="E7" events="1" subjects_affected="1" subjects_at_risk="29"/>
              </event>
              <event>
                <sub_title>Gait inability</sub_title>
                <counts group_id="E1" events="0" subjects_affected="0" subjects_at_risk="56"/>
                <counts group_id="E2" events="0" subjects_affected="0" subjects_at_risk="55"/>
                <counts group_id="E3" events="0" subjects_affected="0" subjects_at_risk="55"/>
                <counts group_id="E4" events="0" subjects_affected="0" subjects_at_risk="14"/>
                <counts group_id="E5" events="1" subjects_affected="1" subjects_at_risk="13"/>
                <counts group_id="E6" events="0" subjects_affected="0" subjects_at_risk="24"/>
                <counts group_id="E7" events="0" subjects_affected="0" subjects_at_risk="29"/>
              </event>
              <event>
                <sub_title>Injection site bruising</sub_title>
                <counts group_id="E1" events="3" subjects_affected="2" subjects_at_risk="56"/>
                <counts group_id="E2" events="4" subjects_affected="4" subjects_at_risk="55"/>
                <counts group_id="E3" events="4" subjects_affected="4" subjects_at_risk="55"/>
                <counts group_id="E4" events="0" subjects_affected="0" subjects_at_risk="14"/>
                <counts group_id="E5" events="0" subjects_affected="0" subjects_at_risk="13"/>
                <counts group_id="E6" events="0" subjects_affected="0" subjects_at_risk="24"/>
                <counts group_id="E7" events="0" subjects_affected="0" subjects_at_risk="29"/>
              </event>
              <event>
                <sub_title>Injection site erythema</sub_title>
                <counts group_id="E1" events="25" subjects_affected="8" subjects_at_risk="56"/>
                <counts group_id="E2" events="43" subjects_affected="11" subjects_at_risk="55"/>
                <counts group_id="E3" events="38" subjects_affected="12" subjects_at_risk="55"/>
                <counts group_id="E4" events="0" subjects_affected="0" subjects_at_risk="14"/>
                <counts group_id="E5" events="17" subjects_affected="3" subjects_at_risk="13"/>
                <counts group_id="E6" events="6" subjects_affected="2" subjects_at_risk="24"/>
                <counts group_id="E7" events="1" subjects_affected="1" subjects_at_risk="29"/>
              </event>
              <event>
                <sub_title>Injection site induration</sub_title>
                <counts group_id="E1" events="0" subjects_affected="0" subjects_at_risk="56"/>
                <counts group_id="E2" events="0" subjects_affected="0" subjects_at_risk="55"/>
                <counts group_id="E3" events="0" subjects_affected="0" subjects_at_risk="55"/>
                <counts group_id="E4" events="1" subjects_affected="1" subjects_at_risk="14"/>
                <counts group_id="E5" events="0" subjects_affected="0" subjects_at_risk="13"/>
                <counts group_id="E6" events="0" subjects_affected="0" subjects_at_risk="24"/>
                <counts group_id="E7" events="0" subjects_affected="0" subjects_at_risk="29"/>
              </event>
              <event>
                <sub_title>Injection site oedema</sub_title>
                <counts group_id="E1" events="7" subjects_affected="3" subjects_at_risk="56"/>
                <counts group_id="E2" events="9" subjects_affected="2" subjects_at_risk="55"/>
                <counts group_id="E3" events="9" subjects_affected="1" subjects_at_risk="55"/>
                <counts group_id="E4" events="0" subjects_affected="0" subjects_at_risk="14"/>
                <counts group_id="E5" events="0" subjects_affected="0" subjects_at_risk="13"/>
                <counts group_id="E6" events="0" subjects_affected="0" subjects_at_risk="24"/>
                <counts group_id="E7" events="0" subjects_affected="0" subjects_at_risk="29"/>
              </event>
              <event>
                <sub_title>Injection site pruritus</sub_title>
                <counts group_id="E1" events="0" subjects_affected="0" subjects_at_risk="56"/>
                <counts group_id="E2" events="2" subjects_affected="2" subjects_at_risk="55"/>
                <counts group_id="E3" events="6" subjects_affected="2" subjects_at_risk="55"/>
                <counts group_id="E4" events="0" subjects_affected="0" subjects_at_risk="14"/>
                <counts group_id="E5" events="12" subjects_affected="1" subjects_at_risk="13"/>
                <counts group_id="E6" events="0" subjects_affected="0" subjects_at_risk="24"/>
                <counts group_id="E7" events="0" subjects_affected="0" subjects_at_risk="29"/>
              </event>
              <event>
                <sub_title>Injection site reaction</sub_title>
                <counts group_id="E1" events="3" subjects_affected="2" subjects_at_risk="56"/>
                <counts group_id="E2" events="29" subjects_affected="4" subjects_at_risk="55"/>
                <counts group_id="E3" events="33" subjects_affected="2" subjects_at_risk="55"/>
                <counts group_id="E4" events="0" subjects_affected="0" subjects_at_risk="14"/>
                <counts group_id="E5" events="0" subjects_affected="0" subjects_at_risk="13"/>
                <counts group_id="E6" events="0" subjects_affected="0" subjects_at_risk="24"/>
                <counts group_id="E7" events="0" subjects_affected="0" subjects_at_risk="29"/>
              </event>
              <event>
                <sub_title>Injection site swelling</sub_title>
                <counts group_id="E1" events="0" subjects_affected="0" subjects_at_risk="56"/>
                <counts group_id="E2" events="31" subjects_affected="4" subjects_at_risk="55"/>
                <counts group_id="E3" events="5" subjects_affected="3" subjects_at_risk="55"/>
                <counts group_id="E4" events="0" subjects_affected="0" subjects_at_risk="14"/>
                <counts group_id="E5" events="0" subjects_affected="0" subjects_at_risk="13"/>
                <counts group_id="E6" events="1" subjects_affected="1" subjects_at_risk="24"/>
                <counts group_id="E7" events="1" subjects_affected="1" subjects_at_risk="29"/>
              </event>
              <event>
                <sub_title>Localised oedema</sub_title>
                <counts group_id="E1" events="5" subjects_affected="3" subjects_at_risk="56"/>
                <counts group_id="E2" events="0" subjects_affected="0" subjects_at_risk="55"/>
                <counts group_id="E3" events="1" subjects_affected="1" subjects_at_risk="55"/>
                <counts group_id="E4" events="0" subjects_affected="0" subjects_at_risk="14"/>
                <counts group_id="E5" events="0" subjects_affected="0" subjects_at_risk="13"/>
                <counts group_id="E6" events="0" subjects_affected="0" subjects_at_risk="24"/>
                <counts group_id="E7" events="0" subjects_affected="0" subjects_at_risk="29"/>
              </event>
              <event>
                <sub_title>Pyrexia</sub_title>
                <counts group_id="E1" events="8" subjects_affected="8" subjects_at_risk="56"/>
                <counts group_id="E2" events="13" subjects_affected="9" subjects_at_risk="55"/>
                <counts group_id="E3" events="9" subjects_affected="8" subjects_at_risk="55"/>
                <counts group_id="E4" events="2" subjects_affected="2" subjects_at_risk="14"/>
                <counts group_id="E5" events="1" subjects_affected="1" subjects_at_risk="13"/>
                <counts group_id="E6" events="3" subjects_affected="3" subjects_at_risk="24"/>
                <counts group_id="E7" events="1" subjects_affected="1" subjects_at_risk="29"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Infections and infestations</title>
            <event_list>
              <event>
                <sub_title>Ear infection</sub_title>
                <counts group_id="E1" events="0" subjects_affected="0" subjects_at_risk="56"/>
                <counts group_id="E2" events="4" subjects_affected="4" subjects_at_risk="55"/>
                <counts group_id="E3" events="2" subjects_affected="1" subjects_at_risk="55"/>
                <counts group_id="E4" events="1" subjects_affected="1" subjects_at_risk="14"/>
                <counts group_id="E5" events="0" subjects_affected="0" subjects_at_risk="13"/>
                <counts group_id="E6" events="0" subjects_affected="0" subjects_at_risk="24"/>
                <counts group_id="E7" events="0" subjects_affected="0" subjects_at_risk="29"/>
              </event>
              <event>
                <sub_title>Gastroenteritis</sub_title>
                <counts group_id="E1" events="2" subjects_affected="1" subjects_at_risk="56"/>
                <counts group_id="E2" events="1" subjects_affected="1" subjects_at_risk="55"/>
                <counts group_id="E3" events="5" subjects_affected="3" subjects_at_risk="55"/>
                <counts group_id="E4" events="0" subjects_affected="0" subjects_at_risk="14"/>
                <counts group_id="E5" events="0" subjects_affected="0" subjects_at_risk="13"/>
                <counts group_id="E6" events="0" subjects_affected="0" subjects_at_risk="24"/>
                <counts group_id="E7" events="0" subjects_affected="0" subjects_at_risk="29"/>
              </event>
              <event>
                <sub_title>Gastroenteritis viral</sub_title>
                <counts group_id="E1" events="1" subjects_affected="1" subjects_at_risk="56"/>
                <counts group_id="E2" events="1" subjects_affected="1" subjects_at_risk="55"/>
                <counts group_id="E3" events="1" subjects_affected="1" subjects_at_risk="55"/>
                <counts group_id="E4" events="1" subjects_affected="1" subjects_at_risk="14"/>
                <counts group_id="E5" events="0" subjects_affected="0" subjects_at_risk="13"/>
                <counts group_id="E6" events="1" subjects_affected="1" subjects_at_risk="24"/>
                <counts group_id="E7" events="0" subjects_affected="0" subjects_at_risk="29"/>
              </event>
              <event>
                <sub_title>Hordeolum</sub_title>
                <counts group_id="E1" events="0" subjects_affected="0" subjects_at_risk="56"/>
                <counts group_id="E2" events="2" subjects_affected="2" subjects_at_risk="55"/>
                <counts group_id="E3" events="0" subjects_affected="0" subjects_at_risk="55"/>
                <counts group_id="E4" events="0" subjects_affected="0" subjects_at_risk="14"/>
                <counts group_id="E5" events="1" subjects_affected="1" subjects_at_risk="13"/>
                <counts group_id="E6" events="0" subjects_affected="0" subjects_at_risk="24"/>
                <counts group_id="E7" events="1" subjects_affected="1" subjects_at_risk="29"/>
              </event>
              <event>
                <sub_title>Influenza</sub_title>
                <counts group_id="E1" events="3" subjects_affected="3" subjects_at_risk="56"/>
                <counts group_id="E2" events="6" subjects_affected="6" subjects_at_risk="55"/>
                <counts group_id="E3" events="1" subjects_affected="1" subjects_at_risk="55"/>
                <counts group_id="E4" events="0" subjects_affected="0" subjects_at_risk="14"/>
                <counts group_id="E5" events="0" subjects_affected="0" subjects_at_risk="13"/>
                <counts group_id="E6" events="2" subjects_affected="2" subjects_at_risk="24"/>
                <counts group_id="E7" events="0" subjects_affected="0" subjects_at_risk="29"/>
              </event>
              <event>
                <sub_title>Nasopharyngitis</sub_title>
                <counts group_id="E1" events="17" subjects_affected="13" subjects_at_risk="56"/>
                <counts group_id="E2" events="17" subjects_affected="13" subjects_at_risk="55"/>
                <counts group_id="E3" events="16" subjects_affected="13" subjects_at_risk="55"/>
                <counts group_id="E4" events="2" subjects_affected="2" subjects_at_risk="14"/>
                <counts group_id="E5" events="0" subjects_affected="0" subjects_at_risk="13"/>
                <counts group_id="E6" events="0" subjects_affected="0" subjects_at_risk="24"/>
                <counts group_id="E7" events="2" subjects_affected="2" subjects_at_risk="29"/>
              </event>
              <event>
                <sub_title>Pharyngitis</sub_title>
                <counts group_id="E1" events="2" subjects_affected="2" subjects_at_risk="56"/>
                <counts group_id="E2" events="2" subjects_affected="2" subjects_at_risk="55"/>
                <counts group_id="E3" events="3" subjects_affected="3" subjects_at_risk="55"/>
                <counts group_id="E4" events="0" subjects_affected="0" subjects_at_risk="14"/>
                <counts group_id="E5" events="1" subjects_affected="1" subjects_at_risk="13"/>
                <counts group_id="E6" events="0" subjects_affected="0" subjects_at_risk="24"/>
                <counts group_id="E7" events="0" subjects_affected="0" subjects_at_risk="29"/>
              </event>
              <event>
                <sub_title>Rhinitis</sub_title>
                <counts group_id="E1" events="1" subjects_affected="1" subjects_at_risk="56"/>
                <counts group_id="E2" events="5" subjects_affected="3" subjects_at_risk="55"/>
                <counts group_id="E3" events="7" subjects_affected="4" subjects_at_risk="55"/>
                <counts group_id="E4" events="0" subjects_affected="0" subjects_at_risk="14"/>
                <counts group_id="E5" events="0" subjects_affected="0" subjects_at_risk="13"/>
                <counts group_id="E6" events="0" subjects_affected="0" subjects_at_risk="24"/>
                <counts group_id="E7" events="0" subjects_affected="0" subjects_at_risk="29"/>
              </event>
              <event>
                <sub_title>Sinusitis</sub_title>
                <counts group_id="E1" events="1" subjects_affected="1" subjects_at_risk="56"/>
                <counts group_id="E2" events="0" subjects_affected="0" subjects_at_risk="55"/>
                <counts group_id="E3" events="4" subjects_affected="3" subjects_at_risk="55"/>
                <counts group_id="E4" events="0" subjects_affected="0" subjects_at_risk="14"/>
                <counts group_id="E5" events="0" subjects_affected="0" subjects_at_risk="13"/>
                <counts group_id="E6" events="0" subjects_affected="0" subjects_at_risk="24"/>
                <counts group_id="E7" events="0" subjects_affected="0" subjects_at_risk="29"/>
              </event>
              <event>
                <sub_title>Upper respiratory tract infection</sub_title>
                <counts group_id="E1" events="8" subjects_affected="6" subjects_at_risk="56"/>
                <counts group_id="E2" events="5" subjects_affected="4" subjects_at_risk="55"/>
                <counts group_id="E3" events="16" subjects_affected="7" subjects_at_risk="55"/>
                <counts group_id="E4" events="1" subjects_affected="1" subjects_at_risk="14"/>
                <counts group_id="E5" events="1" subjects_affected="1" subjects_at_risk="13"/>
                <counts group_id="E6" events="0" subjects_affected="0" subjects_at_risk="24"/>
                <counts group_id="E7" events="2" subjects_affected="2" subjects_at_risk="29"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Injury, poisoning and procedural complications</title>
            <event_list>
              <event>
                <sub_title>Contusion</sub_title>
                <counts group_id="E1" events="2" subjects_affected="2" subjects_at_risk="56"/>
                <counts group_id="E2" events="7" subjects_affected="5" subjects_at_risk="55"/>
                <counts group_id="E3" events="5" subjects_affected="4" subjects_at_risk="55"/>
                <counts group_id="E4" events="0" subjects_affected="0" subjects_at_risk="14"/>
                <counts group_id="E5" events="2" subjects_affected="1" subjects_at_risk="13"/>
                <counts group_id="E6" events="0" subjects_affected="0" subjects_at_risk="24"/>
                <counts group_id="E7" events="2" subjects_affected="2" subjects_at_risk="29"/>
              </event>
              <event>
                <sub_title>Fall</sub_title>
                <counts group_id="E1" events="11" subjects_affected="5" subjects_at_risk="56"/>
                <counts group_id="E2" events="1" subjects_affected="1" subjects_at_risk="55"/>
                <counts group_id="E3" events="6" subjects_affected="5" subjects_at_risk="55"/>
                <counts group_id="E4" events="0" subjects_affected="0" subjects_at_risk="14"/>
                <counts group_id="E5" events="0" subjects_affected="0" subjects_at_risk="13"/>
                <counts group_id="E6" events="0" subjects_affected="0" subjects_at_risk="24"/>
                <counts group_id="E7" events="1" subjects_affected="1" subjects_at_risk="29"/>
              </event>
              <event>
                <sub_title>Gadolinium deposition disease</sub_title>
                <counts group_id="E1" events="0" subjects_affected="0" subjects_at_risk="56"/>
                <counts group_id="E2" events="0" subjects_affected="0" subjects_at_risk="55"/>
                <counts group_id="E3" events="0" subjects_affected="0" subjects_at_risk="55"/>
                <counts group_id="E4" events="1" subjects_affected="1" subjects_at_risk="14"/>
                <counts group_id="E5" events="0" subjects_affected="0" subjects_at_risk="13"/>
                <counts group_id="E6" events="0" subjects_affected="0" subjects_at_risk="24"/>
                <counts group_id="E7" events="0" subjects_affected="0" subjects_at_risk="29"/>
              </event>
              <event>
                <sub_title>Ligament sprain</sub_title>
                <counts group_id="E1" events="4" subjects_affected="4" subjects_at_risk="56"/>
                <counts group_id="E2" events="2" subjects_affected="1" subjects_at_risk="55"/>
                <counts group_id="E3" events="1" subjects_affected="1" subjects_at_risk="55"/>
                <counts group_id="E4" events="2" subjects_affected="2" subjects_at_risk="14"/>
                <counts group_id="E5" events="0" subjects_affected="0" subjects_at_risk="13"/>
                <counts group_id="E6" events="3" subjects_affected="3" subjects_at_risk="24"/>
                <counts group_id="E7" events="1" subjects_affected="1" subjects_at_risk="29"/>
              </event>
              <event>
                <sub_title>Skin abrasion</sub_title>
                <counts group_id="E1" events="3" subjects_affected="2" subjects_at_risk="56"/>
                <counts group_id="E2" events="12" subjects_affected="3" subjects_at_risk="55"/>
                <counts group_id="E3" events="0" subjects_affected="0" subjects_at_risk="55"/>
                <counts group_id="E4" events="3" subjects_affected="1" subjects_at_risk="14"/>
                <counts group_id="E5" events="0" subjects_affected="0" subjects_at_risk="13"/>
                <counts group_id="E6" events="4" subjects_affected="1" subjects_at_risk="24"/>
                <counts group_id="E7" events="1" subjects_affected="1" subjects_at_risk="29"/>
              </event>
              <event>
                <sub_title>Thermal burn</sub_title>
                <counts group_id="E1" events="0" subjects_affected="0" subjects_at_risk="56"/>
                <counts group_id="E2" events="0" subjects_affected="0" subjects_at_risk="55"/>
                <counts group_id="E3" events="0" subjects_affected="0" subjects_at_risk="55"/>
                <counts group_id="E4" events="1" subjects_affected="1" subjects_at_risk="14"/>
                <counts group_id="E5" events="0" subjects_affected="0" subjects_at_risk="13"/>
                <counts group_id="E6" events="0" subjects_affected="0" subjects_at_risk="24"/>
                <counts group_id="E7" events="0" subjects_affected="0" subjects_at_risk="29"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Investigations</title>
            <event_list>
              <event>
                <sub_title>Bone density decreased</sub_title>
                <counts group_id="E1" events="0" subjects_affected="0" subjects_at_risk="56"/>
                <counts group_id="E2" events="0" subjects_affected="0" subjects_at_risk="55"/>
                <counts group_id="E3" events="0" subjects_affected="0" subjects_at_risk="55"/>
                <counts group_id="E4" events="0" subjects_affected="0" subjects_at_risk="14"/>
                <counts group_id="E5" events="1" subjects_affected="1" subjects_at_risk="13"/>
                <counts group_id="E6" events="0" subjects_affected="0" subjects_at_risk="24"/>
                <counts group_id="E7" events="0" subjects_affected="0" subjects_at_risk="29"/>
              </event>
              <event>
                <sub_title>Glutamate dehydrogenase increased</sub_title>
                <counts group_id="E1" events="2" subjects_affected="2" subjects_at_risk="56"/>
                <counts group_id="E2" events="1" subjects_affected="1" subjects_at_risk="55"/>
                <counts group_id="E3" events="3" subjects_affected="3" subjects_at_risk="55"/>
                <counts group_id="E4" events="0" subjects_affected="0" subjects_at_risk="14"/>
                <counts group_id="E5" events="0" subjects_affected="0" subjects_at_risk="13"/>
                <counts group_id="E6" events="0" subjects_affected="0" subjects_at_risk="24"/>
                <counts group_id="E7" events="0" subjects_affected="0" subjects_at_risk="29"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Musculoskeletal and connective tissue disorders</title>
            <event_list>
              <event>
                <sub_title>Arthralgia</sub_title>
                <counts group_id="E1" events="6" subjects_affected="6" subjects_at_risk="56"/>
                <counts group_id="E2" events="8" subjects_affected="7" subjects_at_risk="55"/>
                <counts group_id="E3" events="6" subjects_affected="5" subjects_at_risk="55"/>
                <counts group_id="E4" events="0" subjects_affected="0" subjects_at_risk="14"/>
                <counts group_id="E5" events="1" subjects_affected="1" subjects_at_risk="13"/>
                <counts group_id="E6" events="0" subjects_affected="0" subjects_at_risk="24"/>
                <counts group_id="E7" events="1" subjects_affected="1" subjects_at_risk="29"/>
              </event>
              <event>
                <sub_title>Back pain</sub_title>
                <counts group_id="E1" events="4" subjects_affected="4" subjects_at_risk="56"/>
                <counts group_id="E2" events="9" subjects_affected="7" subjects_at_risk="55"/>
                <counts group_id="E3" events="2" subjects_affected="2" subjects_at_risk="55"/>
                <counts group_id="E4" events="0" subjects_affected="0" subjects_at_risk="14"/>
                <counts group_id="E5" events="0" subjects_affected="0" subjects_at_risk="13"/>
                <counts group_id="E6" events="0" subjects_affected="0" subjects_at_risk="24"/>
                <counts group_id="E7" events="1" subjects_affected="1" subjects_at_risk="29"/>
              </event>
              <event>
                <sub_title>Mobility decreased</sub_title>
                <counts group_id="E1" events="0" subjects_affected="0" subjects_at_risk="56"/>
                <counts group_id="E2" events="0" subjects_affected="0" subjects_at_risk="55"/>
                <counts group_id="E3" events="0" subjects_affected="0" subjects_at_risk="55"/>
                <counts group_id="E4" events="0" subjects_affected="0" subjects_at_risk="14"/>
                <counts group_id="E5" events="1" subjects_affected="1" subjects_at_risk="13"/>
                <counts group_id="E6" events="0" subjects_affected="0" subjects_at_risk="24"/>
                <counts group_id="E7" events="0" subjects_affected="0" subjects_at_risk="29"/>
              </event>
              <event>
                <sub_title>Muscle spasms</sub_title>
                <counts group_id="E1" events="1" subjects_affected="1" subjects_at_risk="56"/>
                <counts group_id="E2" events="7" subjects_affected="2" subjects_at_risk="55"/>
                <counts group_id="E3" events="6" subjects_affected="2" subjects_at_risk="55"/>
                <counts group_id="E4" events="1" subjects_affected="1" subjects_at_risk="14"/>
                <counts group_id="E5" events="2" subjects_affected="2" subjects_at_risk="13"/>
                <counts group_id="E6" events="0" subjects_affected="0" subjects_at_risk="24"/>
                <counts group_id="E7" events="1" subjects_affected="1" subjects_at_risk="29"/>
              </event>
              <event>
                <sub_title>Pain in extremity</sub_title>
                <counts group_id="E1" events="3" subjects_affected="2" subjects_at_risk="56"/>
                <counts group_id="E2" events="6" subjects_affected="4" subjects_at_risk="55"/>
                <counts group_id="E3" events="11" subjects_affected="8" subjects_at_risk="55"/>
                <counts group_id="E4" events="1" subjects_affected="1" subjects_at_risk="14"/>
                <counts group_id="E5" events="0" subjects_affected="0" subjects_at_risk="13"/>
                <counts group_id="E6" events="0" subjects_affected="0" subjects_at_risk="24"/>
                <counts group_id="E7" events="0" subjects_affected="0" subjects_at_risk="29"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Nervous system disorders</title>
            <event_list>
              <event>
                <sub_title>Headache</sub_title>
                <counts group_id="E1" events="28" subjects_affected="9" subjects_at_risk="56"/>
                <counts group_id="E2" events="60" subjects_affected="14" subjects_at_risk="55"/>
                <counts group_id="E3" events="42" subjects_affected="10" subjects_at_risk="55"/>
                <counts group_id="E4" events="3" subjects_affected="1" subjects_at_risk="14"/>
                <counts group_id="E5" events="0" subjects_affected="0" subjects_at_risk="13"/>
                <counts group_id="E6" events="9" subjects_affected="2" subjects_at_risk="24"/>
                <counts group_id="E7" events="4" subjects_affected="2" subjects_at_risk="29"/>
              </event>
              <event>
                <sub_title>Migraine</sub_title>
                <counts group_id="E1" events="0" subjects_affected="0" subjects_at_risk="56"/>
                <counts group_id="E2" events="10" subjects_affected="1" subjects_at_risk="55"/>
                <counts group_id="E3" events="0" subjects_affected="0" subjects_at_risk="55"/>
                <counts group_id="E4" events="1" subjects_affected="1" subjects_at_risk="14"/>
                <counts group_id="E5" events="0" subjects_affected="0" subjects_at_risk="13"/>
                <counts group_id="E6" events="3" subjects_affected="1" subjects_at_risk="24"/>
                <counts group_id="E7" events="0" subjects_affected="0" subjects_at_risk="29"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Respiratory, thoracic and mediastinal disorders</title>
            <event_list>
              <event>
                <sub_title>Cough</sub_title>
                <counts group_id="E1" events="10" subjects_affected="10" subjects_at_risk="56"/>
                <counts group_id="E2" events="11" subjects_affected="8" subjects_at_risk="55"/>
                <counts group_id="E3" events="13" subjects_affected="7" subjects_at_risk="55"/>
                <counts group_id="E4" events="1" subjects_affected="1" subjects_at_risk="14"/>
                <counts group_id="E5" events="0" subjects_affected="0" subjects_at_risk="13"/>
                <counts group_id="E6" events="0" subjects_affected="0" subjects_at_risk="24"/>
                <counts group_id="E7" events="2" subjects_affected="1" subjects_at_risk="29"/>
              </event>
              <event>
                <sub_title>Epistaxis</sub_title>
                <counts group_id="E1" events="9" subjects_affected="3" subjects_at_risk="56"/>
                <counts group_id="E2" events="15" subjects_affected="3" subjects_at_risk="55"/>
                <counts group_id="E3" events="17" subjects_affected="6" subjects_at_risk="55"/>
                <counts group_id="E4" events="0" subjects_affected="0" subjects_at_risk="14"/>
                <counts group_id="E5" events="1" subjects_affected="1" subjects_at_risk="13"/>
                <counts group_id="E6" events="0" subjects_affected="0" subjects_at_risk="24"/>
                <counts group_id="E7" events="0" subjects_affected="0" subjects_at_risk="29"/>
              </event>
              <event>
                <sub_title>Nasal congestion</sub_title>
                <counts group_id="E1" events="1" subjects_affected="1" subjects_at_risk="56"/>
                <counts group_id="E2" events="3" subjects_affected="3" subjects_at_risk="55"/>
                <counts group_id="E3" events="3" subjects_affected="3" subjects_at_risk="55"/>
                <counts group_id="E4" events="1" subjects_affected="1" subjects_at_risk="14"/>
                <counts group_id="E5" events="0" subjects_affected="0" subjects_at_risk="13"/>
                <counts group_id="E6" events="0" subjects_affected="0" subjects_at_risk="24"/>
                <counts group_id="E7" events="2" subjects_affected="2" subjects_at_risk="29"/>
              </event>
              <event>
                <sub_title>Productive cough</sub_title>
                <counts group_id="E1" events="0" subjects_affected="0" subjects_at_risk="56"/>
                <counts group_id="E2" events="3" subjects_affected="3" subjects_at_risk="55"/>
                <counts group_id="E3" events="0" subjects_affected="0" subjects_at_risk="55"/>
                <counts group_id="E4" events="0" subjects_affected="0" subjects_at_risk="14"/>
                <counts group_id="E5" events="0" subjects_affected="0" subjects_at_risk="13"/>
                <counts group_id="E6" events="0" subjects_affected="0" subjects_at_risk="24"/>
                <counts group_id="E7" events="0" subjects_affected="0" subjects_at_risk="29"/>
              </event>
              <event>
                <sub_title>Rhinorrhoea</sub_title>
                <counts group_id="E1" events="1" subjects_affected="1" subjects_at_risk="56"/>
                <counts group_id="E2" events="7" subjects_affected="4" subjects_at_risk="55"/>
                <counts group_id="E3" events="3" subjects_affected="3" subjects_at_risk="55"/>
                <counts group_id="E4" events="2" subjects_affected="1" subjects_at_risk="14"/>
                <counts group_id="E5" events="0" subjects_affected="0" subjects_at_risk="13"/>
                <counts group_id="E6" events="0" subjects_affected="0" subjects_at_risk="24"/>
                <counts group_id="E7" events="0" subjects_affected="0" subjects_at_risk="29"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Skin and subcutaneous tissue disorders</title>
            <event_list>
              <event>
                <sub_title>Erythema</sub_title>
                <counts group_id="E1" events="4" subjects_affected="3" subjects_at_risk="56"/>
                <counts group_id="E2" events="2" subjects_affected="2" subjects_at_risk="55"/>
                <counts group_id="E3" events="1" subjects_affected="1" subjects_at_risk="55"/>
                <counts group_id="E4" events="1" subjects_affected="1" subjects_at_risk="14"/>
                <counts group_id="E5" events="0" subjects_affected="0" subjects_at_risk="13"/>
                <counts group_id="E6" events="0" subjects_affected="0" subjects_at_risk="24"/>
                <counts group_id="E7" events="0" subjects_affected="0" subjects_at_risk="29"/>
              </event>
              <event>
                <sub_title>Rash</sub_title>
                <counts group_id="E1" events="9" subjects_affected="4" subjects_at_risk="56"/>
                <counts group_id="E2" events="5" subjects_affected="4" subjects_at_risk="55"/>
                <counts group_id="E3" events="9" subjects_affected="7" subjects_at_risk="55"/>
                <counts group_id="E4" events="0" subjects_affected="0" subjects_at_risk="14"/>
                <counts group_id="E5" events="0" subjects_affected="0" subjects_at_risk="13"/>
                <counts group_id="E6" events="1" subjects_affected="1" subjects_at_risk="24"/>
                <counts group_id="E7" events="1" subjects_affected="1" subjects_at_risk="29"/>
              </event>
              <event>
                <sub_title>Rash macular</sub_title>
                <counts group_id="E1" events="0" subjects_affected="0" subjects_at_risk="56"/>
                <counts group_id="E2" events="0" subjects_affected="0" subjects_at_risk="55"/>
                <counts group_id="E3" events="0" subjects_affected="0" subjects_at_risk="55"/>
                <counts group_id="E4" events="1" subjects_affected="1" subjects_at_risk="14"/>
                <counts group_id="E5" events="0" subjects_affected="0" subjects_at_risk="13"/>
                <counts group_id="E6" events="0" subjects_affected="0" subjects_at_risk="24"/>
                <counts group_id="E7" events="0" subjects_affected="0" subjects_at_risk="29"/>
              </event>
              <event>
                <sub_title>Urticaria</sub_title>
                <counts group_id="E1" events="4" subjects_affected="3" subjects_at_risk="56"/>
                <counts group_id="E2" events="2" subjects_affected="2" subjects_at_risk="55"/>
                <counts group_id="E3" events="4" subjects_affected="3" subjects_at_risk="55"/>
                <counts group_id="E4" events="0" subjects_affected="0" subjects_at_risk="14"/>
                <counts group_id="E5" events="0" subjects_affected="0" subjects_at_risk="13"/>
                <counts group_id="E6" events="0" subjects_affected="0" subjects_at_risk="24"/>
                <counts group_id="E7" events="0" subjects_affected="0" subjects_at_risk="29"/>
              </event>
            </event_list>
          </category>
        </category_list>
      </other_events>
    </reported_events>

    <certain_agreements>
      <pi_employee>Principal Investigators are NOT employed by the organization sponsoring the study.</pi_employee>
      <restrictive_agreement>The Study being conducted under this Agreement is part of the Overall Study. Investigator is free to publish in reputable journals or to present at professional conferences the results of the Study, but only after the first publication or presentation that involves the Overall Study. The Sponsor may request that Confidential Information be deleted and/or the publication be postponed in order to protect the Sponsor's intellectual property rights.</restrictive_agreement>
    </certain_agreements>
    <point_of_contact>
      <name_or_title>Medical Communications</name_or_title>
      <organization>Hoffmann-La Roche</organization>
      <phone>800 821-8590</phone>
      <email>genentech@druginfo.com</email>
    </point_of_contact>
  </clinical_results>
</clinical_study>


download date: ClinicalTrials.gov processed this data on September 20, 2023
link text: Link to the current ClinicalTrials.gov record.
url: https://clinicaltrials.gov/ct2/show/NCT03039686
org study id: CN001-016
secondary id: 2016-001654-18
secondary id: WN40227
nct id: NCT03039686
lead sponsor: 
has dmc: Yes
is fda regulated drug: Yes
is fda regulated device: No
This is a multi-center, randomized, double-blind, placebo-controlled study to assess the
      efficacy, safety and tolerability of two different weekly doses of RO7239361 in ambulatory
      boys with Duchenne Muscular Dystrophy (DMD).
allocation: Randomized
intervention model: Parallel Assignment
primary purpose: Treatment
masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
measure: Baseline for the North Star Ambulatory Assessment (NSAA) Total Score
time frame: Baseline
description: The NSAA is a functional scale specifically designed for ambulant boys with Duchenne muscular dystrophy (DMD) that can provide information about motor function. The NSAA is a 17-item test of standing, ability to transition from lying to sitting, sitting to standing, and other mobility assessments. Each of the 17 items is evaluated on an ordinal scale of 0-2: 0 = unable to achieve independently, 1 = modified method but achieves goal independent of physical assistance from another, or 2 = normal with no obvious modification of activity. Total score range is 0 to 34. Higher scores reflect better performance.
measure: Change From Baseline in the North Star Ambulatory Assessment (NSAA) Total Score at Week 48
time frame: Baseline, Week 48
description: The NSAA is a functional scale specifically designed for ambulant boys with Duchenne muscular dystrophy (DMD) that can provide information about motor function. The NSAA is a 17-item test of standing, ability to transition from lying to sitting, sitting to standing, and other mobility assessments. Each of the 17 items is evaluated on an ordinal scale of 0-2: 0 = unable to achieve independently, 1 = modified method but achieves goal independent of physical assistance from another, or 2 = normal with no obvious modification of activity. Total score range is 0 to 34. Higher scores reflect better performance. A positive change from baseline indicates an improvement. Based on the mixed-effect model of repeated measures (MMRM).
measure: Baseline Time for 4 Stair Climb
time frame: Baseline
description: The time to complete the 4 stair climb was measured at baseline.
measure: Change From Baseline at Week 48 in 4 Stair Climb Velocity (4SCV)
time frame: Baseline, Week 48
description: 4SCV was calculated as the ratio of the number of stairs climbed (4) divided by the number of seconds taken to complete the 4-stair climb. The results were converted into velocity (distance/time). A positive change from baseline indicates an improvement. Based on the mixed-effect model of repeated measures (MMRM).
measure: Baseline for the Time to Stand From Supine
time frame: Baseline
description: The time required for a participant to stand from supine position. A longer time reflects a worse outcome.
measure: Change From Baseline at Week 48 in Stand From Supine Velocity
time frame: Baseline, Week 48
description: The time required for a participant to stand from supine position. A longer time reflects a worse outcome. A negative change from baseline indicates an improvement. Based on the mixed-effect model of repeated measures (MMRM).
measure: Baseline Time for 10 Meter Walk/Run
time frame: Baseline
description: The time required for a participant to run or walk a distance of 10 meters as quickly as possible. A longer time reflects a worse outcome.
measure: Change From Baseline at Week 48 in 10 M Walk/Run Velocity
time frame: Baseline, Week 48
description: The time required for a participant to run or walk a distance of 10 meters as quickly as possible calculated as velocity (distance/time). A positive change from baseline indicates an improvement. Based on the mixed-effect model of repeated measures (MMRM).
measure: Baseline for the Pediatric Outcome Data Collection Instrument (PODCI) Transfer and Basic Mobility Subscale
time frame: Baseline
description: The PODCI is designed to be completed by the parent/guardian of a child who has knowledge of the child's conditions. The Transfer and Basic Mobility scale is one of the subscales of the PODCI. The results are standardized into a scale of 0-100 with a higher score reflecting better performance.
measure: Change From Baseline at Week 48 in Pediatric Outcome Data Collection Instrument (PODCI) Transfer and Basic Mobility Subscale
time frame: Baseline, Week 48
description: The PODCI is designed to be completed by the parent/guardian of a child who has knowledge of the child's conditions. The Transfer and Basic Mobility scale is one of the subscales of the PODCI. The results are standardized into a scale of 0-100 with a higher score reflecting better performance. A positive change from baseline indicates an improvement. Based on the mixed-effect model of repeated measures (MMRM).
measure: Change From Baseline at Week 48 in Proximal Lower Extremity Flexor Strength
time frame: Baseline, Week 48
description: Proximal lower extremity flexor (knee extension and knee flexion) strength was measured using manual myometry. A higher score reflects a better outcome. A positive change from baseline indicates an improvement.
measure: Baseline for the 6 Minute Walk Distance (6MWD)
time frame: Baseline
description: The 6MWD measured the distance a participant was able to traverse while walking for 6 minutes. A longer distance reflects a better outcome.
measure: Change From Baseline at Week 48 in 6 Minute Walk Distance (6MWD)
time frame: Baseline, Week 48
description: The 6MWD measured the distance a participant was able to traverse while walking for 6 minutes. A longer distance reflects a better outcome. A positive change from baseline indicates an improvement. Based on the mixed-effect model of repeated measures (MMRM).
measure: Percentage of Participants for Each Clinical Global Impression of Change (CGI-C) Assessment Status at Week 48
time frame: Baseline, Week 48
description: The CGI-C was used to assess the participant's overall condition on a 7-point scale, using the status markers "very much improved, much improved, slightly improved, no change, slightly worse, much worse or very much worse" at Week 48 as compared to baseline.
measure: Change From Baseline at Week 48 in 95th Percentile Stride Velocity
time frame: Baseline, Week 48
description: Stride velocity was recorded with the ActiMyo device in a subset of the overall study population. The ActiMyo device measures the daily movement and activity levels of the participant. The device consists of two sensors worn on each ankle. A higher velocity reflects a better outcome. A positive change from baseline indicates an improvement.
measure: Number of Participants With Adverse Events (AEs)
time frame: During DB period (48 weeks) and Whole study (up to approximately 34 months)
description: An adverse event is any untoward medical occurrence in a subject administered a pharmaceutical product and which does not necessarily have to have a causal relationship with the treatment. An adverse event can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding, for example), symptom, or disease temporally associated with the use of a pharmaceutical product, whether or not considered related to the pharmaceutical product. Preexisting conditions which worsen during a study are also considered as adverse events.
measure: Number of Participants With AEs Leading to Discontinuation
time frame: During DB period (48 weeks) and Whole study (up to approximately 34 months)
description: An adverse event is any untoward medical occurrence in a subject administered a pharmaceutical product and which does not necessarily have to have a causal relationship with the treatment. An adverse event can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding, for example), symptom, or disease temporally associated with the use of a pharmaceutical product, whether or not considered related to the pharmaceutical product. Preexisting conditions which worsen during a study are also considered as adverse events. Reported here is the number of participants with AEs that led to study discontinuation.
arm group label: RO7239361 Low Dose
arm group type: Experimental
description: Participants received low dose RO7239361 SC on specified days of the 48-week DB period. Following the DB period participants received low dose RO7239361 on specified days for up to 192 weeks during the open-label period followed by 24 weeks of follow-up.
arm group label: RO7239361 High Dose
arm group type: Experimental
description: Participants received high dose RO7239361 SC on specified days of the 48-week DB period. Following the DB period participants received high dose RO7239361 on specified days for up to 192 weeks during the open-label period followed by 24 weeks of follow-up.
arm group label: Placebo
arm group type: Placebo Comparator
description: Participants received matching placebo solution subcutaneously (SC) on specified days of the 48-week double-blind (DB) period. Following the DB period participants received low dose or high dose RO7239361 on specified days for up to 192 weeks during the open-label period followed by 24 weeks of follow-up.
intervention type: Drug
intervention name: RO7239361
description: Take RO7239361 subcutaneously on specified days over a 48 week blinded period
arm group label: RO7239361 High Dose
arm group label: RO7239361 Low Dose
intervention type: Drug
intervention name: Placebo for RO7239361
description: Take placebo subcutaneously on specified days over a 48 week blinded period
arm group label: Placebo
criteria: 
gender: Male
minimum age: 6 Years
maximum age: 11 Years
healthy volunteers: No
last name: Clinical Trials
role: Study Director
affiliation: Hoffmann-La Roche
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country: Argentina
country: Australia
country: Belgium
country: Canada
country: France
country: Germany
country: Italy
country: Japan
country: Netherlands
country: Spain
country: Sweden
country: United Kingdom
country: United States
url: http://www.fda.gov/Safety/Recalls/
description: FDA Safety Alerts and Recalls
url: http://roche-duchenne-clinicaltrials.com
description: Provides information about the Roche clinical trial NCT03039686 and molecule being investigated in Duchenne
responsible party type: Sponsor
mesh term: Muscular Dystrophies
mesh term: Muscular Dystrophy, Duchenne
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