<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/NCT03779334</url>
  </required_header>
  <id_info>
    <org_study_id>BN40703</org_study_id>
    <secondary_id>2018-002087-12</secondary_id>
    <nct_id>NCT03779334</nct_id>
  </id_info>
  <brief_title>A Study of Risdiplam in Infants With Genetically Diagnosed and Presymptomatic Spinal Muscular Atrophy</brief_title>
  <acronym>Rainbowfish</acronym>
  <official_title>An Open-Label Study of Risdiplam in Infants With Genetically Diagnosed and Presymptomatic Spinal Muscular Atrophy</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>
      A global study of oral risdiplam in pre-symptomatic participants with spinal muscular atrophy&#xD;
      (SMA).&#xD;
    </textblock>
  </brief_summary>
  <detailed_description>
    <textblock>
      The study is an open-label, single-arm, multicenter clinical study to investigate the&#xD;
      efficacy, safety, pharmacokinetics, and pharmacodynamics of risdiplam in infants aged from&#xD;
      birth to 6 weeks who have been genetically diagnosed with SMA but are not yet presenting with&#xD;
      symptoms. There will be a screening, treatment, open-label extension (OLE) and a follow-up.&#xD;
      All participants will receive risdiplam orally once daily for 2 years followed by an OLE&#xD;
      phase of at least 3 years and a follow-up, for a total treatment duration of at least 5 years&#xD;
      for each participant enrolled.&#xD;
    </textblock>
  </detailed_description>
  <overall_status>Active, not recruiting</overall_status>
  <start_date type="Actual">August 7, 2019</start_date>
  <completion_date type="Anticipated">January 21, 2029</completion_date>
  <primary_completion_date type="Actual">February 20, 2023</primary_completion_date>
  <phase>Phase 2</phase>
  <study_type>Interventional</study_type>
  <has_expanded_access>No</has_expanded_access>
  <study_design_info>
    <allocation>N/A</allocation>
    <intervention_model>Single Group Assignment</intervention_model>
    <intervention_model_description>Open-label, single arm</intervention_model_description>
    <primary_purpose>Treatment</primary_purpose>
    <masking>None (Open Label)</masking>
  </study_design_info>
  <primary_outcome>
    <measure>Percentage of participants with two copies of the survival motor neuron (SMN) 2 gene (excluding the known SMN2 gene modifier mutation c.859G&gt;C) and baseline compound muscle action potential (CMAP) &gt;=1.5 millivolt (mV) who are sitting without support</measure>
    <time_frame>At Month 12</time_frame>
    <description>Sitting is defined as &quot;sits without support for 5 seconds&quot; as assessed in Item 22 of the Bayley Scales of Infant and Toddler Development, Third Edition (BSID-III) Gross Motor Scale</description>
  </primary_outcome>
  <secondary_outcome>
    <measure>Percentage of participants developing clinically manifested SMA</measure>
    <time_frame>At Month 12 and 24</time_frame>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Time to permanent ventilation and/or death</measure>
    <time_frame>Up to 7 years</time_frame>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Percentage of participants who are alive without permanent ventilation</measure>
    <time_frame>At Month 12 and 24</time_frame>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Percentage of participants alive</measure>
    <time_frame>At Month 12 and 24</time_frame>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Percentage of participants who achieve the attainment level of the motor milestones as assessed in the Hammersmith Infant Neurological Examination-2 (HINE-2)</measure>
    <time_frame>At Month 12 and 24</time_frame>
    <description>HINE-2 assessment includes head control, sitting, voluntary grasp, ability to kick, rolling, crawling, standing, and walking</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Percentage of participants with two copies of the SMN2 gene sitting without support for 5 seconds (independent of the CMAP value at baseline).</measure>
    <time_frame>At Month 12</time_frame>
    <description>Assessed with BSID-III Gross Motor Scale.</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Percentage of participants sitting without support for 5 seconds</measure>
    <time_frame>At Month 24</time_frame>
    <description>Assessed with BSID-III Gross Motor Scale</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Percentage of participants sitting without support for 30 seconds</measure>
    <time_frame>At Month 12 and 24</time_frame>
    <description>Assessed with BSID-III Gross Motor Scale</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Percentage of participants standing for at least 3 seconds</measure>
    <time_frame>At Month 24</time_frame>
    <description>Assessed with BSID-III Gross Motor Scale</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Percentage of participants walking (takes at least 3 steps)</measure>
    <time_frame>At Month 24</time_frame>
    <description>Assessed with BSID-III Gross Motor Scale</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Percentage of participants demonstrating the ability to achieve a scaled score on BSID-III Gross Motor Subtests within 1.5 standard deviations of chronological reference standard</measure>
    <time_frame>At Month 24 and 42</time_frame>
    <description>Assessed through BSID-III Gross Motor Scale</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Change from baseline score in the Children's Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP INTEND) motor function scale</measure>
    <time_frame>At Month 12</time_frame>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Percentage of participants who achieve a score of 40 or higher, 50 or higher, and 60 or higher in the CHOP INTEND motor function scale</measure>
    <time_frame>At Month 12</time_frame>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Percentage of participants who meet CHOP INTEND stopping criteria at any point</measure>
    <time_frame>Up to Month 24</time_frame>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Change from baseline in the Hammersmith Functional Motor Scale Expanded (HFMSE) score</measure>
    <time_frame>At Month 60</time_frame>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Number and percentage of participants within 3rd percentile of normal range for weight-for-age, length/height-for-age and weight-for-length/height</measure>
    <time_frame>At Month 12, 24, 36, 48 and 60</time_frame>
    <description>Based on the WHO Child Growth Standards (WHO 2019)</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Number and percentage of participants within 3rd percentile of normal range for head circumference-for-age</measure>
    <time_frame>At Month 12 and 24</time_frame>
    <description>Based on the WHO Child Growth Standards (WHO 2019)</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Change from baseline percentiles for weight-for-age, length/height-for-age, and weight-for- length/height</measure>
    <time_frame>At Month 12, 24, 36, 48 and 60</time_frame>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Change from baseline percentiles for head circumference- for-age</measure>
    <time_frame>At Month 12 and 24</time_frame>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Change from baseline in chest circumference</measure>
    <time_frame>At Month 12 and 24</time_frame>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Ratio between chest and head circumferences</measure>
    <time_frame>At Month 12 and 24</time_frame>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Percentage of participants with the ability to swallow and to feed orally</measure>
    <time_frame>At Month 12, 24, 36, 48 and 60</time_frame>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Change from baseline in compound muscle action potential (CMAP) amplitude</measure>
    <time_frame>At Month 12 and 24</time_frame>
    <description>Measured by CMAP</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Measurement of pharmacodynamic marker levels in blood</measure>
    <time_frame>Day 1, 56, 196, 364, 728 and at early withdrawal</time_frame>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Percentage of participants with adverse events</measure>
    <time_frame>Up to 7 years</time_frame>
    <description>Adverse event severity is determined according to the National Cancer Institute Common Terminology Criteria for Adverse Events, Version 5 (NCI CTCAE) v5</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Ophthalmological examination as appropriate for age</measure>
    <time_frame>Up to 7 years</time_frame>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Plasma concentration of risdiplam and its metabolites to characterize the PK profile</measure>
    <time_frame>Up to 7 years</time_frame>
  </secondary_outcome>
  <number_of_arms>1</number_of_arms>
  <enrollment type="Actual">25</enrollment>
  <condition>Muscular Atrophy, Spinal</condition>
  <arm_group>
    <arm_group_label>Open-label Arm</arm_group_label>
    <arm_group_type>Experimental</arm_group_type>
    <description>Participants will be enrolled to receive risdiplam orally once daily at a dose selected to achieve the targeted exposure range.</description>
  </arm_group>
  <intervention>
    <intervention_type>Drug</intervention_type>
    <intervention_name>Risdiplam</intervention_name>
    <description>Risdiplam will be administered orally.</description>
    <arm_group_label>Open-label Arm</arm_group_label>
  </intervention>
  <eligibility>
    <criteria>
      <textblock>
        Inclusion Criteria:&#xD;
&#xD;
          -  Males and females aged from birth (1 day) to 6 weeks (42 days) of age at the time of&#xD;
             first dose (Day 1); a minimum age of 7 days at first dose is required for the first&#xD;
             infant to be enrolled&#xD;
&#xD;
          -  Gestational age of 37-42 weeks for singleton births; gestational age of 34-42 weeks&#xD;
             for twins&#xD;
&#xD;
          -  Body weight &gt;= 3rd percentile for age, using appropriate country-specific guidelines&#xD;
&#xD;
          -  Genetic diagnosis of 5q-autosomal recessive SMA, including confirmation of homozygous&#xD;
             deletion or compound heterozygosity predictive of loss of function of the SMN1 gene&#xD;
&#xD;
          -  Absence of clinical signs or symptoms at screening (Day -42 to Day -2) or at baseline&#xD;
             (Day -1) that are, in the opinion of the investigator, strongly suggestive of SMA&#xD;
&#xD;
          -  Receiving adequate nutrition and hydration at the time of screening, in the opinion of&#xD;
             the investigator&#xD;
&#xD;
          -  Adequately recovered from any acute illness at baseline and considered well enough to&#xD;
             participate in the study, in the opinion of the investigator&#xD;
&#xD;
          -  Able and expected to be able to safely travel to the study site for the entire&#xD;
             duration of the study and in accordance to the frequency of required study visits, in&#xD;
             the opinion of the investigator&#xD;
&#xD;
          -  Able to complete all study procedures, measurements, and visits, and the parent (or&#xD;
             caregiver), in the opinion of the investigator, has adequately supportive psychosocial&#xD;
             circumstances&#xD;
&#xD;
          -  Parent (or caregiver) is willing to consider nasogastric, naso-jejunal, or gastrostomy&#xD;
             tube placement during the study to maintain safe hydration, nutrition, and treatment&#xD;
             delivery, if recommended by the investigator&#xD;
&#xD;
          -  Parent (or caregiver) is willing to consider the use of non-invasive ventilation&#xD;
             during the study, if recommended by the investigator&#xD;
&#xD;
        Exclusion Criteria:&#xD;
&#xD;
          -  Concomitant or previous participation in any investigational drug or device study at&#xD;
             any time&#xD;
&#xD;
          -  Concomitant or previous administration of an SMN2-targeting antisense oligonucleotide,&#xD;
             SMN2-splicing modifier, or gene therapy either in a clinical study or as part of&#xD;
             medical care&#xD;
&#xD;
          -  Presence of significant concurrent syndromes or diseases&#xD;
&#xD;
          -  In the opinion of the investigator, inadequate venous or capillary blood access for&#xD;
             the study procedures&#xD;
&#xD;
          -  Requiring invasive ventilation, tracheostomy or awake non-invasive ventilation&#xD;
&#xD;
          -  Awake hypoxemia (SaO2 &lt; 95%) with or without ventilator support&#xD;
&#xD;
          -  Multiple or fixed contractures and/or hip subluxation or dislocation at birth&#xD;
&#xD;
          -  Systolic blood pressure or diastolic blood pressure or heart rate considered to be&#xD;
             clinically significant by the investigator&#xD;
&#xD;
          -  Presence of clinically relevant ECG abnormalities before study drug administration;&#xD;
             corrected QT interval using Bazett's method &gt; 460 ms; personal or family history&#xD;
             (first degree relatives) of congenital long QT syndrome indicating a safety risk for&#xD;
             patients as determined by the investigator. First-degree atrioventricular block or&#xD;
             isolated right bundle branch block are allowed&#xD;
&#xD;
          -  The infant (and the mother, if breastfeeding the infant) taking any inhibitor of&#xD;
             CYP3A4 taken within 2 weeks, any inducer of CYP3A4 taken within 4 weeks, any OCT 2 and&#xD;
             MATE substrates within 2 weeks and known FMO1 or FMO3 inhibitors or substrates&#xD;
&#xD;
          -  Clinically significant abnormalities in laboratory test results&#xD;
&#xD;
          -  Ascertained or presumptive hypersensitivity to risdiplam or to the constituents of its&#xD;
             formulation&#xD;
&#xD;
          -  Treatment with oral salbutamol or another beta-2 adrenergic agonist taken orally for&#xD;
             SMA is not allowed. Use of inhaled beta-2 adrenergic agonists is allowed&#xD;
&#xD;
          -  Infants exposed to drugs with known retinal toxicity given to mothers during pregnancy&#xD;
             (and lactation) should not be enrolled. Anticipated need for drugs known to cause&#xD;
             retinal toxicity during the study.&#xD;
&#xD;
          -  Diagnosis of ophthalmic diseases&#xD;
      </textblock>
    </criteria>
    <gender>All</gender>
    <minimum_age>1 Day</minimum_age>
    <maximum_age>6 Weeks</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>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>Sydney Children's Hospital; CENTRE FOR CHILD HEALTH RESEARCH &amp; INNOVATION (CHeRI)</name>
      <address>
        <city>Randwick</city>
        <state>New South Wales</state>
        <zip>2031</zip>
        <country>Australia</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Chr de La Citadelle</name>
      <address>
        <city>Liège</city>
        <zip>4000</zip>
        <country>Belgium</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Hospital das Clinicas - FMUSP_X; Neurologia</name>
      <address>
        <city>Sao Paulo</city>
        <state>SP</state>
        <zip>05403-000</zip>
        <country>Brazil</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Szpital Gdanskiego Uniwersytetu Medycznego; Clinic of developmental neurology</name>
      <address>
        <city>Gda?sk</city>
        <zip>80-952</zip>
        <country>Poland</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Russian Children Neuromuscular Center of Veltischev</name>
      <address>
        <city>Moscow</city>
        <state>Moskovskaja Oblast</state>
        <zip>125412</zip>
        <country>Russian Federation</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Kaohsiung Medical University Chung-Ho Hospital; Pediatric Neurology</name>
      <address>
        <city>Kaohsiung</city>
        <zip>807</zip>
        <country>Taiwan</country>
      </address>
    </facility>
  </location>
  <location_countries>
    <country>Australia</country>
    <country>Belgium</country>
    <country>Brazil</country>
    <country>Poland</country>
    <country>Russian Federation</country>
    <country>Taiwan</country>
    <country>United States</country>
  </location_countries>
  <removed_countries>
    <country>Canada</country>
    <country>China</country>
    <country>Italy</country>
    <country>Saudi Arabia</country>
  </removed_countries>
  <verification_date>August 2023</verification_date>
  <study_first_submitted>December 17, 2018</study_first_submitted>
  <study_first_submitted_qc>December 17, 2018</study_first_submitted_qc>
  <study_first_posted type="Actual">December 19, 2018</study_first_posted>
  <last_update_submitted>August 16, 2023</last_update_submitted>
  <last_update_submitted_qc>August 16, 2023</last_update_submitted_qc>
  <last_update_posted type="Actual">August 21, 2023</last_update_posted>
  <responsible_party>
    <responsible_party_type>Sponsor</responsible_party_type>
  </responsible_party>
  <condition_browse>
    <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
    <mesh_term>Muscular Atrophy</mesh_term>
    <mesh_term>Muscular Atrophy, Spinal</mesh_term>
    <mesh_term>Atrophy</mesh_term>
  </condition_browse>
  <intervention_browse>
    <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
    <mesh_term>Risdiplam</mesh_term>
  </intervention_browse>
  <patient_data>
    <sharing_ipd>Yes</sharing_ipd>
    <ipd_description>Qualified researchers may request access to individual patient level data through the clinical study data request platform (www.vivli.org). Further details on Roche's criteria for eligible studies are available here (https://vivli.org/ourmember/roche/). For further details on Roche's Global Policy on the Sharing of Clinical Information and how to request access to related clinical study documents, see here (https://www.roche.com/research_and_development/who_we_are_how_we_work/clinical_trials/our_commitment_to_data_sharing.htm).</ipd_description>
  </patient_data>
  <!-- Results have not yet been posted for this study                                          -->
</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/NCT03779334
org study id: BN40703
secondary id: 2018-002087-12
nct id: NCT03779334
lead sponsor: 
has dmc: Yes
is fda regulated drug: Yes
is fda regulated device: No
A global study of oral risdiplam in pre-symptomatic participants with spinal muscular atrophy
      (SMA).
The study is an open-label, single-arm, multicenter clinical study to investigate the
      efficacy, safety, pharmacokinetics, and pharmacodynamics of risdiplam in infants aged from
      birth to 6 weeks who have been genetically diagnosed with SMA but are not yet presenting with
      symptoms. There will be a screening, treatment, open-label extension (OLE) and a follow-up.
      All participants will receive risdiplam orally once daily for 2 years followed by an OLE
      phase of at least 3 years and a follow-up, for a total treatment duration of at least 5 years
      for each participant enrolled.
allocation: N/A
intervention model: Single Group Assignment
intervention model description: Open-label, single arm
primary purpose: Treatment
masking: None (Open Label)
measure: Percentage of participants with two copies of the survival motor neuron (SMN) 2 gene (excluding the known SMN2 gene modifier mutation c.859G>C) and baseline compound muscle action potential (CMAP) >=1.5 millivolt (mV) who are sitting without support
time frame: At Month 12
description: Sitting is defined as "sits without support for 5 seconds" as assessed in Item 22 of the Bayley Scales of Infant and Toddler Development, Third Edition (BSID-III) Gross Motor Scale
measure: Percentage of participants developing clinically manifested SMA
time frame: At Month 12 and 24
measure: Time to permanent ventilation and/or death
time frame: Up to 7 years
measure: Percentage of participants who are alive without permanent ventilation
time frame: At Month 12 and 24
measure: Percentage of participants alive
time frame: At Month 12 and 24
measure: Percentage of participants who achieve the attainment level of the motor milestones as assessed in the Hammersmith Infant Neurological Examination-2 (HINE-2)
time frame: At Month 12 and 24
description: HINE-2 assessment includes head control, sitting, voluntary grasp, ability to kick, rolling, crawling, standing, and walking
measure: Percentage of participants with two copies of the SMN2 gene sitting without support for 5 seconds (independent of the CMAP value at baseline).
time frame: At Month 12
description: Assessed with BSID-III Gross Motor Scale.
measure: Percentage of participants sitting without support for 5 seconds
time frame: At Month 24
description: Assessed with BSID-III Gross Motor Scale
measure: Percentage of participants sitting without support for 30 seconds
time frame: At Month 12 and 24
description: Assessed with BSID-III Gross Motor Scale
measure: Percentage of participants standing for at least 3 seconds
time frame: At Month 24
description: Assessed with BSID-III Gross Motor Scale
measure: Percentage of participants walking (takes at least 3 steps)
time frame: At Month 24
description: Assessed with BSID-III Gross Motor Scale
measure: Percentage of participants demonstrating the ability to achieve a scaled score on BSID-III Gross Motor Subtests within 1.5 standard deviations of chronological reference standard
time frame: At Month 24 and 42
description: Assessed through BSID-III Gross Motor Scale
measure: Change from baseline score in the Children's Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP INTEND) motor function scale
time frame: At Month 12
measure: Percentage of participants who achieve a score of 40 or higher, 50 or higher, and 60 or higher in the CHOP INTEND motor function scale
time frame: At Month 12
measure: Percentage of participants who meet CHOP INTEND stopping criteria at any point
time frame: Up to Month 24
measure: Change from baseline in the Hammersmith Functional Motor Scale Expanded (HFMSE) score
time frame: At Month 60
measure: Number and percentage of participants within 3rd percentile of normal range for weight-for-age, length/height-for-age and weight-for-length/height
time frame: At Month 12, 24, 36, 48 and 60
description: Based on the WHO Child Growth Standards (WHO 2019)
measure: Number and percentage of participants within 3rd percentile of normal range for head circumference-for-age
time frame: At Month 12 and 24
description: Based on the WHO Child Growth Standards (WHO 2019)
measure: Change from baseline percentiles for weight-for-age, length/height-for-age, and weight-for- length/height
time frame: At Month 12, 24, 36, 48 and 60
measure: Change from baseline percentiles for head circumference- for-age
time frame: At Month 12 and 24
measure: Change from baseline in chest circumference
time frame: At Month 12 and 24
measure: Ratio between chest and head circumferences
time frame: At Month 12 and 24
measure: Percentage of participants with the ability to swallow and to feed orally
time frame: At Month 12, 24, 36, 48 and 60
measure: Change from baseline in compound muscle action potential (CMAP) amplitude
time frame: At Month 12 and 24
description: Measured by CMAP
measure: Measurement of pharmacodynamic marker levels in blood
time frame: Day 1, 56, 196, 364, 728 and at early withdrawal
measure: Percentage of participants with adverse events
time frame: Up to 7 years
description: Adverse event severity is determined according to the National Cancer Institute Common Terminology Criteria for Adverse Events, Version 5 (NCI CTCAE) v5
measure: Ophthalmological examination as appropriate for age
time frame: Up to 7 years
measure: Plasma concentration of risdiplam and its metabolites to characterize the PK profile
time frame: Up to 7 years
arm group label: Open-label Arm
arm group type: Experimental
description: Participants will be enrolled to receive risdiplam orally once daily at a dose selected to achieve the targeted exposure range.
intervention type: Drug
intervention name: Risdiplam
description: Risdiplam will be administered orally.
arm group label: Open-label Arm
criteria: 
gender: All
minimum age: 1 Day
maximum age: 6 Weeks
healthy volunteers: No
last name: Clinical Trials
role: Study Director
affiliation: Hoffmann-La Roche
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
country: Australia
country: Belgium
country: Brazil
country: Poland
country: Russian Federation
country: Taiwan
country: United States
country: Canada
country: China
country: Italy
country: Saudi Arabia
responsible party type: Sponsor
mesh term: Muscular Atrophy
mesh term: Muscular Atrophy, Spinal
mesh term: Atrophy
mesh term: Risdiplam
sharing ipd: Yes
ipd description: Qualified researchers may request access to individual patient level data through the clinical study data request platform (www.vivli.org). Further details on Roche's criteria for eligible studies are available here (https://vivli.org/ourmember/roche/). For further details on Roche's Global Policy on the Sharing of Clinical Information and how to request access to related clinical study documents, see here (https://www.roche.com/research_and_development/who_we_are_how_we_work/clinical_trials/our_commitment_to_data_sharing.htm).