<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/NCT03783923</url>
  </required_header>
  <id_info>
    <org_study_id>PTCEMF-GD-004</org_study_id>
    <nct_id>NCT03783923</nct_id>
  </id_info>
  <brief_title>A Study of Deflazacort (Emflaza®) in Participants With Limb-Girdle Muscular Dystrophy 2I (LGMD2I)</brief_title>
  <official_title>A Multicenter Open-Label Study on the Safety and Efficacy of Deflazacort (Emflaza) in Subjects With Limb-Girdle Muscular Dystrophy 2I (LGMD2I)</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>PTC Therapeutics</agency>
      <agency_class>Industry</agency_class>
    </lead_sponsor>
  </sponsors>
  <source>PTC Therapeutics</source>
  <oversight_info>
    <has_dmc>No</has_dmc>
    <is_fda_regulated_drug>Yes</is_fda_regulated_drug>
    <is_fda_regulated_device>No</is_fda_regulated_device>
    <is_us_export>Yes</is_us_export>
  </oversight_info>
  <brief_summary>
    <textblock>
      This study is designed to evaluate the safety and efficacy of deflazacort in participants&#xD;
      with LGMD2I. Most participants enrolled will have a screening visit and 3 additional visits&#xD;
      (after 1, 13, and 26 weeks of treatment).&#xD;
    </textblock>
  </brief_summary>
  <overall_status>Terminated</overall_status>
  <why_stopped>
    The study was terminated early due to low enrollment and missing efficacy assessment data due&#xD;
    to missed visits related to COVID-19.&#xD;
  </why_stopped>
  <start_date type="Actual">October 31, 2019</start_date>
  <completion_date type="Actual">January 1, 2021</completion_date>
  <primary_completion_date type="Actual">January 1, 2021</primary_completion_date>
  <phase>Phase 3</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>
    <primary_purpose>Treatment</primary_purpose>
    <masking>None (Open Label)</masking>
  </study_design_info>
  <primary_outcome>
    <measure>Change From Baseline in Time to Climb 4 Stairs After 26 Weeks of Treatment With Deflazacort</measure>
    <time_frame>Baseline, Week 26</time_frame>
  </primary_outcome>
  <secondary_outcome>
    <measure>Change From Baseline in Forced Vital Capacity (FVC) After 26 Weeks of Treatment With Deflazacort</measure>
    <time_frame>Baseline, Week 26</time_frame>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Change From Baseline in 2-Minute Walk Test After 26 Weeks of Treatment of Deflazacort</measure>
    <time_frame>Baseline, Week 26</time_frame>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Change From Baseline in Time to up and go After 26 Weeks of Treatment With Deflazacort</measure>
    <time_frame>Baseline, Week 26</time_frame>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Change From Baseline in Time to Descend 4 Stairs After 26 Weeks of Treatment With Deflazacort</measure>
    <time_frame>Baseline, Week 26</time_frame>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Change From Baseline in Time to Run/Walk 10 Meters After 26 Weeks of Treatment With Deflazacort</measure>
    <time_frame>Baseline, Week 26</time_frame>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Change From Baseline in Maximal Inspiratory Pressure (MIP) and Maximal Expiratory Pressure (MEP) After 26 Weeks of Treatment With Deflazacort</measure>
    <time_frame>Baseline, Week 26</time_frame>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Change From Baseline in Hand-Held Myometry After 26 Weeks of Treatment With Deflazacort</measure>
    <time_frame>Baseline, Week 26</time_frame>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Change From Baseline in Global T2 Relaxation Time of Selected Upper and Lower Limb Muscles After 26 Weeks of Treatment With Deflazacort</measure>
    <time_frame>Baseline, Week 26</time_frame>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Number of Participants With Adverse Events (AEs)</measure>
    <time_frame>Baseline up to Week 52</time_frame>
    <description>An AE was any untoward medical occurrence in a participant who received study drug without regard to possibility of causal relationship. Serious adverse event (SAE) was an AE resulting in any of the following outcomes or deemed significant for any other reason: death; initial or prolonged inpatient hospitalization; life-threatening experience (immediate risk of dying); persistent or significant disability/incapacity; congenital anomaly. AEs included both SAEs and non-serious AEs. A summary of other non-serious AEs and all SAEs, regardless of causality is located in the 'Reported AE section'. AEs were summarized separately for Stage 1 and for the overall ataluren experience.</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Area Under the Concentration Curve From Time Zero to t (AUC0-t) of 21-desacetyl Deflazacort and 6β-hydroxy-21-desacetyl Deflazacort</measure>
    <time_frame>Pre-dose, 0.5, 1, 2, 4, and 6 hours post-dose at Baseline and Week 13</time_frame>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Area Under the Concentration Curve From Time Zero to Infinity (AUC0-inf) of 21-desacetyl Deflazacort and 6β-hydroxy-21-desacetyl Deflazacort</measure>
    <time_frame>Pre-dose, 0.5, 1, 2, 4, and 6 hours post-dose at Baseline and Week 13</time_frame>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Maximum Observed Plasma Concentration (Cmax) of 21-desacetyl Deflazacort and 6β-hydroxy-21-desacetyl Deflazacort</measure>
    <time_frame>Pre-dose, 0.5, 1, 2, 4, and 6 hours post-dose at Baseline and Week 13</time_frame>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Time to Reach Cmax (Tmax) of 21-desacetyl Deflazacort and 6β-hydroxy-21-desacetyl Deflazacort</measure>
    <time_frame>Pre-dose, 0.5, 1, 2, 4, and 6 hours post-dose at Baseline and Week 13</time_frame>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Half-Life (t1/2) of 21-desacetyl Deflazacort and 6β-hydroxy-21-desacetyl Deflazacort</measure>
    <time_frame>Pre-dose, 0.5, 1, 2, 4, and 6 hours post-dose at Baseline and Week 13</time_frame>
  </secondary_outcome>
  <number_of_arms>1</number_of_arms>
  <enrollment type="Actual">11</enrollment>
  <condition>Limb-Girdle Muscular Dystrophy</condition>
  <arm_group>
    <arm_group_label>Deflazacort</arm_group_label>
    <arm_group_type>Experimental</arm_group_type>
    <description>Participants will receive deflazacort 0.6 milligrams per kilograms per day (mg/kg/day) orally. The dose could be reduced in case of tolerability issues. Any participant assigned to placebo prior to the Version 4.0 amendment (prior to or after 01 February 2020) will have the option to be consented under Version 4.0 and will be switched to deflazacort for 26 weeks treatment. Any participant assigned to deflazacort prior to the Version 4.0 amendment (prior to 01 February 2020) will have the option to re-consent under Protocol Version 4.0 and continue for an additional 26 weeks treatment. Any participant assigned to deflazacort prior to the Version 4.0 amendment (after 01 February 2020) will have the option to re-consent under Protocol Version 4.0 at their Week 13 Visit and continue treatment until Week 26. Any new participant enrolled until 31 May 2020 will receive deflazacort for 26 weeks.</description>
  </arm_group>
  <intervention>
    <intervention_type>Drug</intervention_type>
    <intervention_name>Deflazacort</intervention_name>
    <description>Deflazacort tablet will be administered as per the dose and schedule specified in the arm.</description>
    <arm_group_label>Deflazacort</arm_group_label>
    <other_name>Emflaza®</other_name>
  </intervention>
  <eligibility>
    <criteria>
      <textblock>
        Inclusion Criteria:&#xD;
&#xD;
          -  Genetic diagnosis of LGMD2I (confirmed mutation in the fukutin-related protein [FKRP]&#xD;
             gene).&#xD;
&#xD;
          -  Ability to ascend 4 stairs greater than or equal to (≥) 2.5 seconds and be able to&#xD;
             complete the ascent and descent both at screening and baseline.&#xD;
&#xD;
          -  Ability to understand the nature of the study and the consent form and to comply with&#xD;
             study related procedures.&#xD;
&#xD;
          -  Must weigh between 35 to 112.5 kilograms (kg).&#xD;
&#xD;
        Exclusion Criteria:&#xD;
&#xD;
          -  Received ≥4 weeks of continuous, systemic corticosteroid therapy within 3 months of&#xD;
             study screening visit.&#xD;
&#xD;
          -  Presence of significant cardiomyopathy as defined by echocardiogram (left ventricular&#xD;
             ejection fraction less than (&lt;) 30 percent [%]) at screening.&#xD;
&#xD;
          -  Requires fulltime ventilator support.&#xD;
&#xD;
          -  History of chronic systemic fungal or viral infections.&#xD;
&#xD;
          -  History of recent bacterial infection (including tuberculosis) per discretion of the&#xD;
             Investigator.&#xD;
&#xD;
          -  Diagnosis of diabetes mellitus (controlled and/or uncontrolled) defined as glycated&#xD;
             hemoglobin (HbA1c) ≥6.5% (based on historical or present diagnosis).&#xD;
&#xD;
          -  History of immunosuppression or other contraindications to glucocorticosteroid&#xD;
             therapy.&#xD;
&#xD;
          -  Requires concomitant use or greater than (&gt;) 1 week of drugs or substances that are&#xD;
             moderate to strong cytochrome P3A4 (CYP3A4) inhibitors (for example, clarithromycin,&#xD;
             fluconazole, diltiazem, verapamil, grapefruit juice) or moderate or strong CYP3A4&#xD;
             inducers (that is, rifampin, efavirenz, carbamazepine, phenytoin) at baseline.&#xD;
&#xD;
          -  Participated in an interventional clinical trial within the last 3 months prior the&#xD;
             baseline visit.&#xD;
&#xD;
          -  Unable or unwilling to comply with the contraceptive requirements of the protocol.&#xD;
&#xD;
          -  Female participants who are pregnant and/or breastfeeding.&#xD;
&#xD;
          -  Evidence or history of clinically significant hematological, renal, endocrine,&#xD;
             pulmonary, gastrointestinal, cardiovascular, hepatic, neurologic, psychiatric, or&#xD;
             allergic disease.&#xD;
      </textblock>
    </criteria>
    <gender>All</gender>
    <minimum_age>18 Years</minimum_age>
    <maximum_age>N/A</maximum_age>
    <healthy_volunteers>No</healthy_volunteers>
  </eligibility>
  <overall_official>
    <last_name>Cristobal Passalacqua, MD</last_name>
    <role>Study Director</role>
    <affiliation>PTC Therapeutics</affiliation>
  </overall_official>
  <location>
    <facility>
      <name>Rare Disease Research, LLC</name>
      <address>
        <city>Atlanta</city>
        <state>Georgia</state>
        <zip>30324</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>University of Iowa Hospitals and Clinics</name>
      <address>
        <city>Iowa City</city>
        <state>Iowa</state>
        <zip>52242</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>The 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>Hugo W Moser Research Institute at 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 Minnesota</name>
      <address>
        <city>Minneapolis</city>
        <state>Minnesota</state>
        <zip>55455</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Washington University School of Medicine</name>
      <address>
        <city>Saint Louis</city>
        <state>Missouri</state>
        <zip>63110</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>University of Pennsylvania</name>
      <address>
        <city>Philadelphia</city>
        <state>Pennsylvania</state>
        <zip>19104</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>University of Washington</name>
      <address>
        <city>Seattle</city>
        <state>Washington</state>
        <zip>98195</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>University of Alberta</name>
      <address>
        <city>Edmonton</city>
        <state>Alberta</state>
        <zip>T6G 2G3</zip>
        <country>Canada</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Ottawa Hospital</name>
      <address>
        <city>Ottawa</city>
        <zip>K1Y 4E9</zip>
        <country>Canada</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Rigshospitalet, University of Copenhagen</name>
      <address>
        <city>Copenhagen</city>
        <zip>2200</zip>
        <country>Denmark</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>CHRU de NANCY Service de Neurologie</name>
      <address>
        <city>France</city>
        <zip>54035</zip>
        <country>France</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>University Hospital La Timone</name>
      <address>
        <city>Marseille</city>
        <zip>13385</zip>
        <country>France</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Ludwig-Maximilians University Munich, Friedrich-Baur-Institute</name>
      <address>
        <city>Munich</city>
        <zip>80801</zip>
        <country>Germany</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Oslo University Hospital</name>
      <address>
        <city>Oslo</city>
        <zip>0424</zip>
        <country>Norway</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Pirogov Russian National Research Medical University</name>
      <address>
        <city>Moscow</city>
        <zip>125412</zip>
        <country>Russian Federation</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Saint-Petersburg State Pediatric Medical University</name>
      <address>
        <city>Saint Petersburg</city>
        <zip>194100</zip>
        <country>Russian Federation</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Sahlgrenska University Hospital</name>
      <address>
        <city>Gothenburg</city>
        <zip>41345</zip>
        <country>Sweden</country>
      </address>
    </facility>
  </location>
  <location_countries>
    <country>Canada</country>
    <country>Denmark</country>
    <country>France</country>
    <country>Germany</country>
    <country>Norway</country>
    <country>Russian Federation</country>
    <country>Sweden</country>
    <country>United States</country>
  </location_countries>
  <verification_date>May 2022</verification_date>
  <study_first_submitted>December 19, 2018</study_first_submitted>
  <study_first_submitted_qc>December 19, 2018</study_first_submitted_qc>
  <study_first_posted type="Actual">December 21, 2018</study_first_posted>
  <results_first_submitted>March 1, 2022</results_first_submitted>
  <results_first_submitted_qc>May 31, 2022</results_first_submitted_qc>
  <results_first_posted type="Actual">June 27, 2022</results_first_posted>
  <disposition_first_submitted>November 9, 2021</disposition_first_submitted>
  <disposition_first_submitted_qc>May 31, 2022</disposition_first_submitted_qc>
  <disposition_first_posted type="Actual">June 27, 2022</disposition_first_posted>
  <last_update_submitted>May 31, 2022</last_update_submitted>
  <last_update_submitted_qc>May 31, 2022</last_update_submitted_qc>
  <last_update_posted type="Actual">June 27, 2022</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 Dystrophies</mesh_term>
    <mesh_term>Muscular Dystrophies, Limb-Girdle</mesh_term>
  </condition_browse>
  <intervention_browse>
    <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
    <mesh_term>Deflazacort</mesh_term>
  </intervention_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>March 25, 2020</document_date>
      <document_url>https://ClinicalTrials.gov/ProvidedDocs/23/NCT03783923/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 28, 2020</document_date>
      <document_url>https://ClinicalTrials.gov/ProvidedDocs/23/NCT03783923/SAP_001.pdf</document_url>
    </provided_document>
  </provided_document_section>

  <clinical_results>

    <participant_flow>
      <pre_assignment_details>Participants enrolled under protocol v3.0 were 1:1 randomized to get placebo or deflazacort. After protocol v4.0, the study became an open-label study and all participants received deflazacort.&#xD;
Per planned analysis, participants who took at least 1 dose of deflazacort were pooled in the safety and efficacy summaries.</pre_assignment_details>
      <group_list>
        <group group_id="P1">
          <title>Deflazacort</title>
          <description>Participants received deflazacort tablets, administered orally once daily at a target dose of 0.6 milligrams (mg)/kilograms (kg)/day for 26 weeks in placebo-controlled period and for 26 weeks in open-label extension period.</description>
        </group>
        <group group_id="P2">
          <title>Placebo</title>
          <description>Participants received placebo matched to deflazacort tablets, administered orally once daily for 26 weeks in placebo-controlled period. Participants were then transitioned to receive deflazacort tablets, administered orally once daily at a target dose of 0.6 mg/kg/day for 26 weeks in open-label extension period.</description>
        </group>
      </group_list>
      <period_list>
        <period>
          <title>Placebo-Controlled Period (26 Weeks)</title>
          <milestone_list>
            <milestone>
              <title>STARTED</title>
              <participants_list>
                <participants group_id="P1" count="5"/>
                <participants group_id="P2" count="6"/>
              </participants_list>
            </milestone>
            <milestone>
              <title>Safety Population</title>
              <participants_list>
                <participants group_id="P1" count="5"/>
                <participants group_id="P2" count="2">2 participants who received placebo in placebo-controlled period transitioned to receive deflazacort in open-label extension.</participants>
              </participants_list>
            </milestone>
            <milestone>
              <title>COMPLETED</title>
              <participants_list>
                <participants group_id="P1" count="1"/>
                <participants group_id="P2" count="2"/>
              </participants_list>
            </milestone>
            <milestone>
              <title>NOT COMPLETED</title>
              <participants_list>
                <participants group_id="P1" count="4"/>
                <participants group_id="P2" count="4"/>
              </participants_list>
            </milestone>
          </milestone_list>
          <drop_withdraw_reason_list>
            <drop_withdraw_reason>
              <title>Study Terminated</title>
              <participants_list>
                <participants group_id="P1" count="3"/>
                <participants group_id="P2" count="2"/>
              </participants_list>
            </drop_withdraw_reason>
            <drop_withdraw_reason>
              <title>Adverse Event</title>
              <participants_list>
                <participants group_id="P1" count="1"/>
                <participants group_id="P2" 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="2"/>
              </participants_list>
            </drop_withdraw_reason>
          </drop_withdraw_reason_list>
        </period>
        <period>
          <title>Open-Label Extension (26 Weeks)</title>
          <milestone_list>
            <milestone>
              <title>STARTED</title>
              <participants_list>
                <participants group_id="P1" count="1"/>
                <participants group_id="P2" count="2"/>
              </participants_list>
            </milestone>
            <milestone>
              <title>COMPLETED</title>
              <participants_list>
                <participants group_id="P1" count="0"/>
                <participants group_id="P2" count="0"/>
              </participants_list>
            </milestone>
            <milestone>
              <title>NOT COMPLETED</title>
              <participants_list>
                <participants group_id="P1" count="1"/>
                <participants group_id="P2" count="2"/>
              </participants_list>
            </milestone>
          </milestone_list>
          <drop_withdraw_reason_list>
            <drop_withdraw_reason>
              <title>Withdrawal by Subject</title>
              <participants_list>
                <participants group_id="P1" count="1"/>
                <participants group_id="P2" count="1"/>
              </participants_list>
            </drop_withdraw_reason>
            <drop_withdraw_reason>
              <title>Study Terminated</title>
              <participants_list>
                <participants group_id="P1" count="0"/>
                <participants group_id="P2" count="1"/>
              </participants_list>
            </drop_withdraw_reason>
          </drop_withdraw_reason_list>
        </period>
      </period_list>
    </participant_flow>

    <baseline>
      <population>Enrolled population included all informed-consented participants who met all inclusion and exclusion criteria.</population>
      <group_list>
        <group group_id="B1">
          <title>Deflazacort</title>
          <description>Participants received deflazacort tablets, administered orally once daily at a target dose of 0.6 mg/kg/day.</description>
        </group>
        <group group_id="B2">
          <title>Placebo</title>
          <description>Participants received placebo matched to deflazacort tablets, administered orally once daily for 26 weeks in placebo-controlled period. Participants were then transitioned to receive deflazacort tablets, administered orally once daily at a target dose of 0.6 mg/kg/day for 26 weeks in open-label extension period.</description>
        </group>
        <group group_id="B3">
          <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="5"/>
            <count group_id="B2" value="6"/>
            <count group_id="B3" value="11"/>
          </count_list>
        </analyzed>
      </analyzed_list>
      <measure_list>
        <measure>
          <title>Age</title>
          <units>Participants</units>
          <param>Count of Participants</param>
          <class_list>
            <class>
              <category_list>
                <category>
                  <title>&lt;=18 years</title>
                  <measurement_list>
                    <measurement group_id="B1" value="0"/>
                    <measurement group_id="B2" value="0"/>
                    <measurement group_id="B3" value="0"/>
                  </measurement_list>
                </category>
                <category>
                  <title>Between 18 and 65 years</title>
                  <measurement_list>
                    <measurement group_id="B1" value="5"/>
                    <measurement group_id="B2" value="5"/>
                    <measurement group_id="B3" value="10"/>
                  </measurement_list>
                </category>
                <category>
                  <title>&gt;=65 years</title>
                  <measurement_list>
                    <measurement group_id="B1" value="0"/>
                    <measurement group_id="B2" value="1"/>
                    <measurement group_id="B3" value="1"/>
                  </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="4"/>
                    <measurement group_id="B2" value="4"/>
                    <measurement group_id="B3" value="8"/>
                  </measurement_list>
                </category>
                <category>
                  <title>Male</title>
                  <measurement_list>
                    <measurement group_id="B1" value="1"/>
                    <measurement group_id="B2" value="2"/>
                    <measurement group_id="B3" value="3"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
        <measure>
          <title>Race (NIH/OMB)</title>
          <units>Participants</units>
          <param>Count of Participants</param>
          <class_list>
            <class>
              <category_list>
                <category>
                  <title>American Indian or Alaska Native</title>
                  <measurement_list>
                    <measurement group_id="B1" value="0"/>
                    <measurement group_id="B2" value="0"/>
                    <measurement group_id="B3" value="0"/>
                  </measurement_list>
                </category>
                <category>
                  <title>Asian</title>
                  <measurement_list>
                    <measurement group_id="B1" value="0"/>
                    <measurement group_id="B2" value="0"/>
                    <measurement group_id="B3" value="0"/>
                  </measurement_list>
                </category>
                <category>
                  <title>Native Hawaiian or Other Pacific Islander</title>
                  <measurement_list>
                    <measurement group_id="B1" value="0"/>
                    <measurement group_id="B2" value="0"/>
                    <measurement group_id="B3" value="0"/>
                  </measurement_list>
                </category>
                <category>
                  <title>Black or African American</title>
                  <measurement_list>
                    <measurement group_id="B1" value="0"/>
                    <measurement group_id="B2" value="0"/>
                    <measurement group_id="B3" value="0"/>
                  </measurement_list>
                </category>
                <category>
                  <title>White</title>
                  <measurement_list>
                    <measurement group_id="B1" value="5"/>
                    <measurement group_id="B2" value="6"/>
                    <measurement group_id="B3" value="11"/>
                  </measurement_list>
                </category>
                <category>
                  <title>More than one race</title>
                  <measurement_list>
                    <measurement group_id="B1" value="0"/>
                    <measurement group_id="B2" value="0"/>
                    <measurement group_id="B3" value="0"/>
                  </measurement_list>
                </category>
                <category>
                  <title>Unknown or Not Reported</title>
                  <measurement_list>
                    <measurement group_id="B1" value="0"/>
                    <measurement group_id="B2" value="0"/>
                    <measurement group_id="B3" value="0"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
      </measure_list>
    </baseline>

    <outcome_list>
      <outcome>
        <type>Primary</type>
        <title>Change From Baseline in Time to Climb 4 Stairs After 26 Weeks of Treatment With Deflazacort</title>
        <time_frame>Baseline, Week 26</time_frame>
        <population>Safety population included all enrolled participants who received at least 1 dose of deflazacort. Here, 'Number analyzed' = participants evaluable at specified timepoint.</population>
        <group_list>
          <group group_id="O1">
            <title>Deflazacort</title>
            <description>Participants received deflazacort tablets, administered orally once daily at a target dose of 0.6 mg/kg/day.</description>
          </group>
        </group_list>
        <measure>
          <title>Change From Baseline in Time to Climb 4 Stairs After 26 Weeks of Treatment With Deflazacort</title>
          <population>Safety population included all enrolled participants who received at least 1 dose of deflazacort. Here, 'Number analyzed' = participants evaluable at specified timepoint.</population>
          <units>seconds</units>
          <param>Mean</param>
          <dispersion>Standard Deviation</dispersion>
          <analyzed_list>
            <analyzed>
              <units>Participants</units>
              <scope>Measure</scope>
              <count_list>
                <count group_id="O1" value="7"/>
              </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="7"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="5.476" spread="2.0178"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Change at Week 26</title>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="O1" value="1"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="-0.200"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
      </outcome>
      <outcome>
        <type>Secondary</type>
        <title>Change From Baseline in Forced Vital Capacity (FVC) After 26 Weeks of Treatment With Deflazacort</title>
        <time_frame>Baseline, Week 26</time_frame>
        <population>Due to early termination of the study and missing efficacy assessment data due to missed visits related to COVID-19, data was not collected or analyzed for this secondary efficacy endpoint.</population>
        <group_list>
          <group group_id="O1">
            <title>Deflazacort</title>
            <description>Participants received deflazacort tablets, administered orally once daily at a target dose of 0.6 mg/kg/day.</description>
          </group>
        </group_list>
        <measure>
          <title>Change From Baseline in Forced Vital Capacity (FVC) After 26 Weeks of Treatment With Deflazacort</title>
          <population>Due to early termination of the study and missing efficacy assessment data due to missed visits related to COVID-19, data was not collected or analyzed for this secondary efficacy endpoint.</population>
          <analyzed_list>
            <analyzed>
              <units>Participants</units>
              <scope>Measure</scope>
              <count_list>
                <count group_id="O1" value="0"/>
              </count_list>
            </analyzed>
          </analyzed_list>
        </measure>
      </outcome>
      <outcome>
        <type>Secondary</type>
        <title>Change From Baseline in 2-Minute Walk Test After 26 Weeks of Treatment of Deflazacort</title>
        <time_frame>Baseline, Week 26</time_frame>
        <population>Safety population included all enrolled participants who received at least 1 dose of deflazacort. Here, 'Number analyzed' = participants evaluable at specified timepoint.</population>
        <group_list>
          <group group_id="O1">
            <title>Deflazacort</title>
            <description>Participants received deflazacort tablets, administered orally once daily at a target dose of 0.6 mg/kg/day.</description>
          </group>
        </group_list>
        <measure>
          <title>Change From Baseline in 2-Minute Walk Test After 26 Weeks of Treatment of Deflazacort</title>
          <population>Safety population included all enrolled participants who received at least 1 dose of deflazacort. Here, 'Number analyzed' = participants evaluable at specified timepoint.</population>
          <units>meters</units>
          <param>Mean</param>
          <dispersion>Standard Deviation</dispersion>
          <analyzed_list>
            <analyzed>
              <units>Participants</units>
              <scope>Measure</scope>
              <count_list>
                <count group_id="O1" value="7"/>
              </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="7"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="135.4" spread="26.75"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Change at Week 26</title>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="O1" value="1"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="2.0"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
      </outcome>
      <outcome>
        <type>Secondary</type>
        <title>Change From Baseline in Time to up and go After 26 Weeks of Treatment With Deflazacort</title>
        <time_frame>Baseline, Week 26</time_frame>
        <population>Safety population included all enrolled participants who received at least 1 dose of deflazacort. Here, 'Number analyzed' = participants evaluable at specified timepoint.</population>
        <group_list>
          <group group_id="O1">
            <title>Deflazacort</title>
            <description>Participants received deflazacort tablets, administered orally once daily at a target dose of 0.6 mg/kg/day.</description>
          </group>
        </group_list>
        <measure>
          <title>Change From Baseline in Time to up and go After 26 Weeks of Treatment With Deflazacort</title>
          <population>Safety population included all enrolled participants who received at least 1 dose of deflazacort. Here, 'Number analyzed' = participants evaluable at specified timepoint.</population>
          <units>seconds</units>
          <param>Mean</param>
          <dispersion>Standard Deviation</dispersion>
          <analyzed_list>
            <analyzed>
              <units>Participants</units>
              <scope>Measure</scope>
              <count_list>
                <count group_id="O1" value="7"/>
              </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="7"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="11.93" spread="4.743"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Change at Week 26</title>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="O1" value="1"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="9.70"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
      </outcome>
      <outcome>
        <type>Secondary</type>
        <title>Change From Baseline in Time to Descend 4 Stairs After 26 Weeks of Treatment With Deflazacort</title>
        <time_frame>Baseline, Week 26</time_frame>
        <population>Safety population included all enrolled participants who received at least 1 dose of deflazacort. Here, 'Number analyzed' = participants evaluable at specified timepoint.</population>
        <group_list>
          <group group_id="O1">
            <title>Deflazacort</title>
            <description>Participants received deflazacort tablets, administered orally once daily at a target dose of 0.6 mg/kg/day.</description>
          </group>
        </group_list>
        <measure>
          <title>Change From Baseline in Time to Descend 4 Stairs After 26 Weeks of Treatment With Deflazacort</title>
          <population>Safety population included all enrolled participants who received at least 1 dose of deflazacort. Here, 'Number analyzed' = participants evaluable at specified timepoint.</population>
          <units>seconds</units>
          <param>Mean</param>
          <dispersion>Standard Deviation</dispersion>
          <analyzed_list>
            <analyzed>
              <units>Participants</units>
              <scope>Measure</scope>
              <count_list>
                <count group_id="O1" value="7"/>
              </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="7"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="3.66" spread="1.707"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Change at Week 26</title>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="O1" value="1"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="0.10"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
      </outcome>
      <outcome>
        <type>Secondary</type>
        <title>Change From Baseline in Time to Run/Walk 10 Meters After 26 Weeks of Treatment With Deflazacort</title>
        <time_frame>Baseline, Week 26</time_frame>
        <population>Safety population included all enrolled participants who received at least 1 dose of deflazacort. Here, 'Number analyzed' = participants evaluable at specified timepoint.</population>
        <group_list>
          <group group_id="O1">
            <title>Deflazacort</title>
            <description>Participants received deflazacort tablets, administered orally once daily at a target dose of 0.6 mg/kg/day.</description>
          </group>
        </group_list>
        <measure>
          <title>Change From Baseline in Time to Run/Walk 10 Meters After 26 Weeks of Treatment With Deflazacort</title>
          <population>Safety population included all enrolled participants who received at least 1 dose of deflazacort. Here, 'Number analyzed' = participants evaluable at specified timepoint.</population>
          <units>seconds</units>
          <param>Mean</param>
          <dispersion>Standard Deviation</dispersion>
          <analyzed_list>
            <analyzed>
              <units>Participants</units>
              <scope>Measure</scope>
              <count_list>
                <count group_id="O1" value="7"/>
              </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="7"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="8.53" spread="1.897"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Change at Week 26</title>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="O1" value="1"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="-0.40"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
      </outcome>
      <outcome>
        <type>Secondary</type>
        <title>Change From Baseline in Maximal Inspiratory Pressure (MIP) and Maximal Expiratory Pressure (MEP) After 26 Weeks of Treatment With Deflazacort</title>
        <time_frame>Baseline, Week 26</time_frame>
        <population>Due to early termination of the study and missing efficacy assessment data due to missed visits related to COVID-19, data was not collected or analyzed for this secondary efficacy endpoint.</population>
        <group_list>
          <group group_id="O1">
            <title>Deflazacort</title>
            <description>Participants received deflazacort tablets, administered orally once daily at a target dose of 0.6 mg/kg/day.</description>
          </group>
        </group_list>
        <measure>
          <title>Change From Baseline in Maximal Inspiratory Pressure (MIP) and Maximal Expiratory Pressure (MEP) After 26 Weeks of Treatment With Deflazacort</title>
          <population>Due to early termination of the study and missing efficacy assessment data due to missed visits related to COVID-19, data was not collected or analyzed for this secondary efficacy endpoint.</population>
          <analyzed_list>
            <analyzed>
              <units>Participants</units>
              <scope>Measure</scope>
              <count_list>
                <count group_id="O1" value="0"/>
              </count_list>
            </analyzed>
          </analyzed_list>
        </measure>
      </outcome>
      <outcome>
        <type>Secondary</type>
        <title>Change From Baseline in Hand-Held Myometry After 26 Weeks of Treatment With Deflazacort</title>
        <time_frame>Baseline, Week 26</time_frame>
        <population>Due to early termination of the study and missing efficacy assessment data due to missed visits related to COVID-19, data was not collected or analyzed for this secondary efficacy endpoint.</population>
        <group_list>
          <group group_id="O1">
            <title>Deflazacort</title>
            <description>Participants received deflazacort tablets, administered orally once daily at a target dose of 0.6 mg/kg/day.</description>
          </group>
        </group_list>
        <measure>
          <title>Change From Baseline in Hand-Held Myometry After 26 Weeks of Treatment With Deflazacort</title>
          <population>Due to early termination of the study and missing efficacy assessment data due to missed visits related to COVID-19, data was not collected or analyzed for this secondary efficacy endpoint.</population>
          <analyzed_list>
            <analyzed>
              <units>Participants</units>
              <scope>Measure</scope>
              <count_list>
                <count group_id="O1" value="0"/>
              </count_list>
            </analyzed>
          </analyzed_list>
        </measure>
      </outcome>
      <outcome>
        <type>Secondary</type>
        <title>Change From Baseline in Global T2 Relaxation Time of Selected Upper and Lower Limb Muscles After 26 Weeks of Treatment With Deflazacort</title>
        <time_frame>Baseline, Week 26</time_frame>
        <population>Due to early termination of the study and missing efficacy assessment data due to missed visits related to COVID-19, data was not collected or analyzed for this secondary efficacy endpoint.</population>
        <group_list>
          <group group_id="O1">
            <title>Deflazacort</title>
            <description>Participants received deflazacort tablets, administered orally once daily at a target dose of 0.6 mg/kg/day.</description>
          </group>
        </group_list>
        <measure>
          <title>Change From Baseline in Global T2 Relaxation Time of Selected Upper and Lower Limb Muscles After 26 Weeks of Treatment With Deflazacort</title>
          <population>Due to early termination of the study and missing efficacy assessment data due to missed visits related to COVID-19, data was not collected or analyzed for this secondary efficacy endpoint.</population>
          <analyzed_list>
            <analyzed>
              <units>Participants</units>
              <scope>Measure</scope>
              <count_list>
                <count group_id="O1" value="0"/>
              </count_list>
            </analyzed>
          </analyzed_list>
        </measure>
      </outcome>
      <outcome>
        <type>Secondary</type>
        <title>Number of Participants With Adverse Events (AEs)</title>
        <description>An AE was any untoward medical occurrence in a participant who received study drug without regard to possibility of causal relationship. Serious adverse event (SAE) was an AE resulting in any of the following outcomes or deemed significant for any other reason: death; initial or prolonged inpatient hospitalization; life-threatening experience (immediate risk of dying); persistent or significant disability/incapacity; congenital anomaly. AEs included both SAEs and non-serious AEs. A summary of other non-serious AEs and all SAEs, regardless of causality is located in the 'Reported AE section'. AEs were summarized separately for Stage 1 and for the overall ataluren experience.</description>
        <time_frame>Baseline up to Week 52</time_frame>
        <population>Safety population included all enrolled participants who received at least 1 dose of deflazacort. Per planned analysis, participants who took at least 1 dose of deflazacort were pooled in the safety summaries. The participants who received only placebo during the study were not included for safety analysis.</population>
        <group_list>
          <group group_id="O1">
            <title>Deflazacort</title>
            <description>Participants received deflazacort tablets, administered orally once daily at a target dose of 0.6 mg/kg/day.</description>
          </group>
        </group_list>
        <measure>
          <title>Number of Participants With Adverse Events (AEs)</title>
          <description>An AE was any untoward medical occurrence in a participant who received study drug without regard to possibility of causal relationship. Serious adverse event (SAE) was an AE resulting in any of the following outcomes or deemed significant for any other reason: death; initial or prolonged inpatient hospitalization; life-threatening experience (immediate risk of dying); persistent or significant disability/incapacity; congenital anomaly. AEs included both SAEs and non-serious AEs. A summary of other non-serious AEs and all SAEs, regardless of causality is located in the 'Reported AE section'. AEs were summarized separately for Stage 1 and for the overall ataluren experience.</description>
          <population>Safety population included all enrolled participants who received at least 1 dose of deflazacort. Per planned analysis, participants who took at least 1 dose of deflazacort were pooled in the safety summaries. The participants who received only placebo during the study were not included for safety analysis.</population>
          <units>Participants</units>
          <param>Count of Participants</param>
          <analyzed_list>
            <analyzed>
              <units>Participants</units>
              <scope>Measure</scope>
              <count_list>
                <count group_id="O1" value="7"/>
              </count_list>
            </analyzed>
          </analyzed_list>
          <class_list>
            <class>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="5"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
      </outcome>
      <outcome>
        <type>Secondary</type>
        <title>Area Under the Concentration Curve From Time Zero to t (AUC0-t) of 21-desacetyl Deflazacort and 6β-hydroxy-21-desacetyl Deflazacort</title>
        <time_frame>Pre-dose, 0.5, 1, 2, 4, and 6 hours post-dose at Baseline and Week 13</time_frame>
        <population>The pharmacokinetic (PK) population included all enrolled participants who received at least 1 dose of deflazacort and had at least one PK profile. Here, 'Number analyzed' = participants evaluable for specified category.</population>
        <group_list>
          <group group_id="O1">
            <title>Deflazacort</title>
            <description>Participants received deflazacort tablets, administered orally once daily at a target dose of 0.6 mg/kg/day.</description>
          </group>
        </group_list>
        <measure>
          <title>Area Under the Concentration Curve From Time Zero to t (AUC0-t) of 21-desacetyl Deflazacort and 6β-hydroxy-21-desacetyl Deflazacort</title>
          <population>The pharmacokinetic (PK) population included all enrolled participants who received at least 1 dose of deflazacort and had at least one PK profile. Here, 'Number analyzed' = participants evaluable for specified category.</population>
          <units>nanograms (ng)*hour (hr)/milliliter (mL)</units>
          <param>Mean</param>
          <dispersion>Standard Deviation</dispersion>
          <analyzed_list>
            <analyzed>
              <units>Participants</units>
              <scope>Measure</scope>
              <count_list>
                <count group_id="O1" value="4"/>
              </count_list>
            </analyzed>
          </analyzed_list>
          <class_list>
            <class>
              <title>21-desacetyl deflazacort: Baseline</title>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="O1" value="4"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="401.1" spread="139.62"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>21-desacetyl deflazacort: Week 13</title>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="O1" value="2"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="365.6" spread="NA">Due to concentration below the limit of quantitation (BLQ) of 0.50 ng/mL, data not calculated.</measurement>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>6β-hydroxy-21-desacetyl deflazacort: Week 1</title>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="O1" value="4"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="418.2" spread="58.569"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>6β-hydroxy-21-desacetyl deflazacort: Week 13</title>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="O1" value="2"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="515.3" spread="NA">Due to concentration BLQ of 0.50 ng/mL, data not calculated.</measurement>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
      </outcome>
      <outcome>
        <type>Secondary</type>
        <title>Area Under the Concentration Curve From Time Zero to Infinity (AUC0-inf) of 21-desacetyl Deflazacort and 6β-hydroxy-21-desacetyl Deflazacort</title>
        <time_frame>Pre-dose, 0.5, 1, 2, 4, and 6 hours post-dose at Baseline and Week 13</time_frame>
        <population>The PK population included all enrolled participants who received at least 1 dose of deflazacort and had at least one PK profile. Here, 'Number analyzed' = participants evaluable for specified category.</population>
        <group_list>
          <group group_id="O1">
            <title>Deflazacort</title>
            <description>Participants received deflazacort tablets, administered orally once daily at a target dose of 0.6 mg/kg/day.</description>
          </group>
        </group_list>
        <measure>
          <title>Area Under the Concentration Curve From Time Zero to Infinity (AUC0-inf) of 21-desacetyl Deflazacort and 6β-hydroxy-21-desacetyl Deflazacort</title>
          <population>The PK population included all enrolled participants who received at least 1 dose of deflazacort and had at least one PK profile. Here, 'Number analyzed' = participants evaluable for specified category.</population>
          <units>ng*hr/mL</units>
          <param>Mean</param>
          <dispersion>Standard Deviation</dispersion>
          <analyzed_list>
            <analyzed>
              <units>Participants</units>
              <scope>Measure</scope>
              <count_list>
                <count group_id="O1" value="4"/>
              </count_list>
            </analyzed>
          </analyzed_list>
          <class_list>
            <class>
              <title>21-desacetyl deflazacort: Baseline</title>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="O1" value="4"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="423.6" spread="150.16"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>21-desacetyl deflazacort: Week 13</title>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="O1" value="0"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
            </class>
            <class>
              <title>6β-hydroxy-21-desacetyl deflazacort: Week 1</title>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="O1" value="3"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="520.3" spread="31.508"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>6β-hydroxy-21-desacetyl deflazacort: Week 13</title>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="O1" value="0"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
            </class>
          </class_list>
        </measure>
      </outcome>
      <outcome>
        <type>Secondary</type>
        <title>Maximum Observed Plasma Concentration (Cmax) of 21-desacetyl Deflazacort and 6β-hydroxy-21-desacetyl Deflazacort</title>
        <time_frame>Pre-dose, 0.5, 1, 2, 4, and 6 hours post-dose at Baseline and Week 13</time_frame>
        <population>The PK population included all enrolled participants who received at least 1 dose of deflazacort and had at least one PK profile. Here, 'Number analyzed' = participants evaluable for specified category.</population>
        <group_list>
          <group group_id="O1">
            <title>Deflazacort</title>
            <description>Participants received deflazacort tablets, administered orally once daily at a target dose of 0.6 mg/kg/day.</description>
          </group>
        </group_list>
        <measure>
          <title>Maximum Observed Plasma Concentration (Cmax) of 21-desacetyl Deflazacort and 6β-hydroxy-21-desacetyl Deflazacort</title>
          <population>The PK population included all enrolled participants who received at least 1 dose of deflazacort and had at least one PK profile. Here, 'Number analyzed' = participants evaluable for specified category.</population>
          <units>ng/mL</units>
          <param>Mean</param>
          <dispersion>Standard Deviation</dispersion>
          <analyzed_list>
            <analyzed>
              <units>Participants</units>
              <scope>Measure</scope>
              <count_list>
                <count group_id="O1" value="4"/>
              </count_list>
            </analyzed>
          </analyzed_list>
          <class_list>
            <class>
              <title>21-desacetyl deflazacort: Baseline</title>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="O1" value="4"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="184.8" spread="49.054"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>21-desacetyl deflazacort: Week 13</title>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="O1" value="2"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="171.0" spread="NA">Due to concentration BLQ of 0.50 ng/mL, data not calculated.</measurement>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>6β-hydroxy-21-desacetyl deflazacort: Week 1</title>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="O1" value="4"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="135.8" spread="29.205"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>6β-hydroxy-21-desacetyl deflazacort: Week 13</title>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="O1" value="2"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="162.0" spread="NA">Due to concentration BLQ of 0.50 ng/mL, data not calculated.</measurement>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
      </outcome>
      <outcome>
        <type>Secondary</type>
        <title>Time to Reach Cmax (Tmax) of 21-desacetyl Deflazacort and 6β-hydroxy-21-desacetyl Deflazacort</title>
        <time_frame>Pre-dose, 0.5, 1, 2, 4, and 6 hours post-dose at Baseline and Week 13</time_frame>
        <population>The PK population included all enrolled participants who received at least 1 dose of deflazacort and had at least one PK profile. Here, 'Number analyzed' = participants evaluable for specified category.</population>
        <group_list>
          <group group_id="O1">
            <title>Deflazacort</title>
            <description>Participants received deflazacort tablets, administered orally once daily at a target dose of 0.6 mg/kg/day.</description>
          </group>
        </group_list>
        <measure>
          <title>Time to Reach Cmax (Tmax) of 21-desacetyl Deflazacort and 6β-hydroxy-21-desacetyl Deflazacort</title>
          <population>The PK population included all enrolled participants who received at least 1 dose of deflazacort and had at least one PK profile. Here, 'Number analyzed' = participants evaluable for specified category.</population>
          <units>hr</units>
          <param>Median</param>
          <dispersion>Full Range</dispersion>
          <analyzed_list>
            <analyzed>
              <units>Participants</units>
              <scope>Measure</scope>
              <count_list>
                <count group_id="O1" value="4"/>
              </count_list>
            </analyzed>
          </analyzed_list>
          <class_list>
            <class>
              <title>21-desacetyl deflazacort: Baseline</title>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="O1" value="4"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="0.992" lower_limit="0.50" upper_limit="1.00"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>21-desacetyl deflazacort: Week 13</title>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="O1" value="2"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="0.525" lower_limit="0.50" upper_limit="0.55"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>6β-hydroxy-21-desacetyl deflazacort: Week 1</title>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="O1" value="4"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="1.000" lower_limit="1.00" upper_limit="1.90"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>6β-hydroxy-21-desacetyl deflazacort: Week 13</title>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="O1" value="2"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="1.550" lower_limit="1.10" upper_limit="2.00"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
      </outcome>
      <outcome>
        <type>Secondary</type>
        <title>Half-Life (t1/2) of 21-desacetyl Deflazacort and 6β-hydroxy-21-desacetyl Deflazacort</title>
        <time_frame>Pre-dose, 0.5, 1, 2, 4, and 6 hours post-dose at Baseline and Week 13</time_frame>
        <population>The PK population included all enrolled participants who received at least 1 dose of deflazacort and had at least one PK profile. Here, 'Number analyzed' = participants evaluable for specified category.</population>
        <group_list>
          <group group_id="O1">
            <title>Deflazacort</title>
            <description>Participants received deflazacort tablets, administered orally once daily at a target dose of 0.6 mg/kg/day.</description>
          </group>
        </group_list>
        <measure>
          <title>Half-Life (t1/2) of 21-desacetyl Deflazacort and 6β-hydroxy-21-desacetyl Deflazacort</title>
          <population>The PK population included all enrolled participants who received at least 1 dose of deflazacort and had at least one PK profile. Here, 'Number analyzed' = participants evaluable for specified category.</population>
          <units>hr</units>
          <param>Mean</param>
          <dispersion>Standard Deviation</dispersion>
          <analyzed_list>
            <analyzed>
              <units>Participants</units>
              <scope>Measure</scope>
              <count_list>
                <count group_id="O1" value="4"/>
              </count_list>
            </analyzed>
          </analyzed_list>
          <class_list>
            <class>
              <title>21-desacetyl deflazacort: Baseline</title>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="O1" value="4"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="1.174" spread="0.0849"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>21-desacetyl deflazacort: Week 13</title>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="O1" value="2"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="1.235" spread="NA">Due to concentration BLQ of 0.50 ng/mL, data not calculated.</measurement>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>6β-hydroxy-21-desacetyl deflazacort: Week 1</title>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="O1" value="3"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="2.358" spread="0.5825"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>6β-hydroxy-21-desacetyl deflazacort: Week 13</title>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="O1" value="1"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="1.95"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
      </outcome>
    </outcome_list>

    <reported_events>
      <time_frame>Baseline up to Week 52</time_frame>
      <desc>Safety population included all enrolled participants who received at least 1 dose of deflazacort. Per planned analysis, participants who took at least 1 dose of deflazacort were pooled in the safety summaries. The participants who received only placebo (not deflazacort) during the study were not monitored for safety assessments (including AE monitoring) per planned analysis.</desc>
      <group_list>
        <group group_id="E1">
          <title>Deflazacort</title>
          <description>Participants received deflazacort tablets, administered orally once daily at a target dose of 0.6 mg/kg/day.</description>
        </group>
      </group_list>
      <serious_events>
        <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="7"/>
              </event>
              <event>
                <sub_title>Total, serious adverse events</sub_title>
                <counts group_id="E1" subjects_affected="0" subjects_at_risk="7"/>
              </event>
            </event_list>
          </category>
        </category_list>
      </serious_events>
      <other_events>
        <frequency_threshold>5</frequency_threshold>
        <default_vocab>MedDRA 23.1</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="5" subjects_at_risk="7"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Endocrine disorders</title>
            <event_list>
              <event>
                <sub_title>Cushingoid</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="7"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Gastrointestinal disorders</title>
            <event_list>
              <event>
                <sub_title>Abdominal discomfort</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="7"/>
              </event>
              <event>
                <sub_title>Dyspepsia</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="7"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>General disorders</title>
            <event_list>
              <event>
                <sub_title>Fatigue</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="7"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Infections and infestations</title>
            <event_list>
              <event>
                <sub_title>Influenza</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="7"/>
              </event>
              <event>
                <sub_title>Upper respiratory tract infection</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="7"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Injury, poisoning and procedural complications</title>
            <event_list>
              <event>
                <sub_title>Contusion</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="7"/>
              </event>
              <event>
                <sub_title>Post-traumatic pain</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="7"/>
              </event>
              <event>
                <sub_title>Skin laceration</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="7"/>
              </event>
              <event>
                <sub_title>Spinal compression fracture</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="7"/>
              </event>
              <event>
                <sub_title>Fall</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="7"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Investigations</title>
            <event_list>
              <event>
                <sub_title>Weight increased</sub_title>
                <counts group_id="E1" subjects_affected="2" subjects_at_risk="7"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Metabolism and nutrition disorders</title>
            <event_list>
              <event>
                <sub_title>Increased appetite</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="7"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Musculoskeletal and connective tissue disorders</title>
            <event_list>
              <event>
                <sub_title>Arthralgia</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="7"/>
              </event>
              <event>
                <sub_title>Back pain</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="7"/>
              </event>
              <event>
                <sub_title>Muscle tightness</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="7"/>
              </event>
              <event>
                <sub_title>Myalgia</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="7"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Nervous system disorders</title>
            <event_list>
              <event>
                <sub_title>Headache</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="7"/>
              </event>
              <event>
                <sub_title>Hypoaesthesia</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="7"/>
              </event>
              <event>
                <sub_title>Tension headache</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="7"/>
              </event>
              <event>
                <sub_title>Visual field defect</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="7"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Psychiatric disorders</title>
            <event_list>
              <event>
                <sub_title>Apathy</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="7"/>
              </event>
              <event>
                <sub_title>Depressed mood</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="7"/>
              </event>
              <event>
                <sub_title>Sleep disorder</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="7"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Renal and urinary disorders</title>
            <event_list>
              <event>
                <sub_title>Pollakiuria</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="7"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Reproductive system and breast disorders</title>
            <event_list>
              <event>
                <sub_title>Amenorrhoea</sub_title>
                <description>This is a gender-specific AE. Only female participants were at risk.</description>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="5"/>
              </event>
              <event>
                <sub_title>Menstruation irregular</sub_title>
                <description>This is a gender-specific AE. Only female participants were at risk.</description>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="5"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Skin and subcutaneous tissue disorders</title>
            <event_list>
              <event>
                <sub_title>Acne</sub_title>
                <counts group_id="E1" subjects_affected="2" subjects_at_risk="7"/>
              </event>
              <event>
                <sub_title>Abnormal hair growth</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="7"/>
              </event>
              <event>
                <sub_title>Hirsutism</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="7"/>
              </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 Sponsor can review results and/or communications prior to public release and can embargo communications regarding trial results for a period that is up to 180 days from the time submitted to the sponsor for review. The sponsor may consult with the PI to require changes to the communication or extend the embargo.</restrictive_agreement>
    </certain_agreements>
    <limitations_and_caveats>The study was terminated early due to low enrollment and missing efficacy assessment data due to missed visits related to COVID-19.</limitations_and_caveats>
    <point_of_contact>
      <name_or_title>Medical Information</name_or_title>
      <organization>PTC Therapeutics, Inc.</organization>
      <phone>1-866-562-4620</phone>
      <email>medinfo@ptcbio.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/NCT03783923
org study id: PTCEMF-GD-004
nct id: NCT03783923
lead sponsor: 
has dmc: No
is fda regulated drug: Yes
is fda regulated device: No
is us export: Yes
This study is designed to evaluate the safety and efficacy of deflazacort in participants
      with LGMD2I. Most participants enrolled will have a screening visit and 3 additional visits
      (after 1, 13, and 26 weeks of treatment).
allocation: N/A
intervention model: Single Group Assignment
primary purpose: Treatment
masking: None (Open Label)
measure: Change From Baseline in Time to Climb 4 Stairs After 26 Weeks of Treatment With Deflazacort
time frame: Baseline, Week 26
measure: Change From Baseline in Forced Vital Capacity (FVC) After 26 Weeks of Treatment With Deflazacort
time frame: Baseline, Week 26
measure: Change From Baseline in 2-Minute Walk Test After 26 Weeks of Treatment of Deflazacort
time frame: Baseline, Week 26
measure: Change From Baseline in Time to up and go After 26 Weeks of Treatment With Deflazacort
time frame: Baseline, Week 26
measure: Change From Baseline in Time to Descend 4 Stairs After 26 Weeks of Treatment With Deflazacort
time frame: Baseline, Week 26
measure: Change From Baseline in Time to Run/Walk 10 Meters After 26 Weeks of Treatment With Deflazacort
time frame: Baseline, Week 26
measure: Change From Baseline in Maximal Inspiratory Pressure (MIP) and Maximal Expiratory Pressure (MEP) After 26 Weeks of Treatment With Deflazacort
time frame: Baseline, Week 26
measure: Change From Baseline in Hand-Held Myometry After 26 Weeks of Treatment With Deflazacort
time frame: Baseline, Week 26
measure: Change From Baseline in Global T2 Relaxation Time of Selected Upper and Lower Limb Muscles After 26 Weeks of Treatment With Deflazacort
time frame: Baseline, Week 26
measure: Number of Participants With Adverse Events (AEs)
time frame: Baseline up to Week 52
description: An AE was any untoward medical occurrence in a participant who received study drug without regard to possibility of causal relationship. Serious adverse event (SAE) was an AE resulting in any of the following outcomes or deemed significant for any other reason: death; initial or prolonged inpatient hospitalization; life-threatening experience (immediate risk of dying); persistent or significant disability/incapacity; congenital anomaly. AEs included both SAEs and non-serious AEs. A summary of other non-serious AEs and all SAEs, regardless of causality is located in the 'Reported AE section'. AEs were summarized separately for Stage 1 and for the overall ataluren experience.
measure: Area Under the Concentration Curve From Time Zero to t (AUC0-t) of 21-desacetyl Deflazacort and 6β-hydroxy-21-desacetyl Deflazacort
time frame: Pre-dose, 0.5, 1, 2, 4, and 6 hours post-dose at Baseline and Week 13
measure: Area Under the Concentration Curve From Time Zero to Infinity (AUC0-inf) of 21-desacetyl Deflazacort and 6β-hydroxy-21-desacetyl Deflazacort
time frame: Pre-dose, 0.5, 1, 2, 4, and 6 hours post-dose at Baseline and Week 13
measure: Maximum Observed Plasma Concentration (Cmax) of 21-desacetyl Deflazacort and 6β-hydroxy-21-desacetyl Deflazacort
time frame: Pre-dose, 0.5, 1, 2, 4, and 6 hours post-dose at Baseline and Week 13
measure: Time to Reach Cmax (Tmax) of 21-desacetyl Deflazacort and 6β-hydroxy-21-desacetyl Deflazacort
time frame: Pre-dose, 0.5, 1, 2, 4, and 6 hours post-dose at Baseline and Week 13
measure: Half-Life (t1/2) of 21-desacetyl Deflazacort and 6β-hydroxy-21-desacetyl Deflazacort
time frame: Pre-dose, 0.5, 1, 2, 4, and 6 hours post-dose at Baseline and Week 13
arm group label: Deflazacort
arm group type: Experimental
description: Participants will receive deflazacort 0.6 milligrams per kilograms per day (mg/kg/day) orally. The dose could be reduced in case of tolerability issues. Any participant assigned to placebo prior to the Version 4.0 amendment (prior to or after 01 February 2020) will have the option to be consented under Version 4.0 and will be switched to deflazacort for 26 weeks treatment. Any participant assigned to deflazacort prior to the Version 4.0 amendment (prior to 01 February 2020) will have the option to re-consent under Protocol Version 4.0 and continue for an additional 26 weeks treatment. Any participant assigned to deflazacort prior to the Version 4.0 amendment (after 01 February 2020) will have the option to re-consent under Protocol Version 4.0 at their Week 13 Visit and continue treatment until Week 26. Any new participant enrolled until 31 May 2020 will receive deflazacort for 26 weeks.
intervention type: Drug
intervention name: Deflazacort
description: Deflazacort tablet will be administered as per the dose and schedule specified in the arm.
arm group label: Deflazacort
other name: Emflaza®
criteria: 
gender: All
minimum age: 18 Years
maximum age: N/A
healthy volunteers: No
last name: Cristobal Passalacqua, MD
role: Study Director
affiliation: PTC Therapeutics
facility: 
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country: Canada
country: Denmark
country: France
country: Germany
country: Norway
country: Russian Federation
country: Sweden
country: United States
responsible party type: Sponsor
mesh term: Muscular Dystrophies
mesh term: Muscular Dystrophies, Limb-Girdle
mesh term: Deflazacort
provided document: 
provided document: 
participant flow: 
baseline: 
outcome list: 
reported events: 
certain agreements: 
limitations and caveats: The study was terminated early due to low enrollment and missing efficacy assessment data due to missed visits related to COVID-19.
point of contact: 