xml: <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/NCT05967351</url>
    </required_header>
    <id_info>
      <org_study_id>SRP-9001-305</org_study_id>
      <secondary_id>2023-505043-39-00</secondary_id>
      <nct_id>NCT05967351</nct_id>
    </id_info>
    <brief_title>A Long-term Follow-up Study of Participants Who Received Delandistrogene Moxeparvovec (SRP-9001) in a Previous Clinical Study</brief_title>
    <acronym>EXPEDITION</acronym>
    <official_title>A Phase 3, Multinational, Long-term Follow-up Study to Evaluate Safety and Efficacy in Subjects Who Have Previously Received SRP-9001 in a Clinical Study</official_title>
    <sponsors>
      <lead_sponsor>
        <agency>Sarepta Therapeutics, Inc.</agency>
        <agency_class>Industry</agency_class>
      </lead_sponsor>
      <collaborator>
        <agency>Hoffmann-La Roche</agency>
        <agency_class>Industry</agency_class>
      </collaborator>
    </sponsors>
    <source>Sarepta Therapeutics, Inc.</source>
    <oversight_info>
      <has_dmc>Yes</has_dmc>
      <is_fda_regulated_drug>Yes</is_fda_regulated_drug>
      <is_fda_regulated_device>No</is_fda_regulated_device>
    </oversight_info>
    <brief_summary>
      <textblock>
        The purpose of this study is to provide a single clinical study with a uniform approach to&#xD;
        monitoring long-term safety and efficacy in participants who received delandistrogene&#xD;
        moxeparvovec in a previous clinical study. No study drug will be administered as part of this&#xD;
        study. Pre-infusion baseline will be defined as the timepoint just prior to infusion of&#xD;
        delandistrogene moxeparvovec from a previous clinical study. Each participant will be&#xD;
        followed for a minimum of 5 years post-infusion of delandistrogene moxeparvovec from a&#xD;
        previous clinical study. The duration of participation in this study is dependent on the&#xD;
        length of follow-up the participant completed post-infusion of delandistrogene moxeparvovec&#xD;
        from a previous clinical study.&#xD;
      </textblock>
    </brief_summary>
    <overall_status>Not yet recruiting</overall_status>
    <start_date type="Anticipated">August 31, 2023</start_date>
    <completion_date type="Anticipated">November 30, 2030</completion_date>
    <primary_completion_date type="Anticipated">November 30, 2030</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>Other</primary_purpose>
      <masking>None (Open Label)</masking>
    </study_design_info>
    <primary_outcome>
      <measure>Number of Participants with a Treatment-emergent Adverse Event (TEAE), Serious Adverse Event (SAE), and Adverse Event of Special Interest (AESI)</measure>
      <time_frame>Up to 5 years after dosing</time_frame>
    </primary_outcome>
    <secondary_outcome>
      <measure>Change in the North Star Ambulatory Assessment (NSAA) Total Score From Pre-infusion Baseline to 5 Years Post-infusion of Delandistrogene Moxeparvovec</measure>
      <time_frame>Baseline, 5 years</time_frame>
    </secondary_outcome>
    <secondary_outcome>
      <measure>Change in Time to Rise From Floor From Pre-infusion Baseline to 5 Years Post-infusion of Delandistrogene Moxeparvovec</measure>
      <time_frame>Baseline, 5 years</time_frame>
    </secondary_outcome>
    <secondary_outcome>
      <measure>Change in the Time of 10-meter Walk/Run (10MWR) From Pre-infusion Baseline to 5 Years Post-infusion of Delandistrogene Moxeparvovec</measure>
      <time_frame>Baseline, 5 years</time_frame>
    </secondary_outcome>
    <secondary_outcome>
      <measure>Change in Performance of Upper Limb (PUL) (Version 2.0) Total Scores From Pre-infusion Baseline to 5 Years Post-infusion of Delandistrogene Moxeparvovec</measure>
      <time_frame>Baseline, 5 years</time_frame>
    </secondary_outcome>
    <secondary_outcome>
      <measure>Change in PUL (Version 2.0) Domain Specific Scores From Pre-infusion Baseline to 5 Years Post-infusion of Delandistrogene Moxeparvovec</measure>
      <time_frame>Baseline, 5 years</time_frame>
    </secondary_outcome>
    <secondary_outcome>
      <measure>Change in Forced Vital Capacity Percent (FVC%) Predicted From Pre-infusion Baseline to 5 Years Post-infusion of Delandistrogene Moxeparvovec</measure>
      <time_frame>Baseline, 5 years</time_frame>
    </secondary_outcome>
    <secondary_outcome>
      <measure>Change in Peak Expiratory Flow Percent (PEF%) Predicted From Pre-infusion Baseline to 5 Years Post-infusion of Delandistrogene Moxeparvovec</measure>
      <time_frame>Baseline, 5 years</time_frame>
    </secondary_outcome>
    <secondary_outcome>
      <measure>Change in Cardiac Magnetic Resonance Imaging (MRI) Findings From Pre-infusion Baseline to 5 Years Post-infusion of Delandistrogene Moxeparvovec</measure>
      <time_frame>Baseline, 5 years</time_frame>
    </secondary_outcome>
    <secondary_outcome>
      <measure>Change in Musculoskeletal MRI Findings From Pre-infusion Baseline to 5 Years Post-infusion of Delandistrogene Moxeparvovec</measure>
      <time_frame>Baseline, 5 years</time_frame>
    </secondary_outcome>
    <number_of_arms>1</number_of_arms>
    <enrollment type="Anticipated">400</enrollment>
    <condition>Duchenne Muscular Dystrophy</condition>
    <arm_group>
      <arm_group_label>Delandistrogene Moxeparvovec</arm_group_label>
      <arm_group_type>Experimental</arm_group_type>
      <description>Participant received delandistrogene moxeparvovec in a previous clinical study.</description>
    </arm_group>
    <intervention>
      <intervention_type>Genetic</intervention_type>
      <intervention_name>delandistrogene moxeparvovec</intervention_name>
      <description>No study drug will be administered as part of this study. Eligible participants who received treatment with delandistrogene moxeparvovec during a previous clinical study will be included.</description>
      <arm_group_label>Delandistrogene Moxeparvovec</arm_group_label>
      <other_name>SRP-9001</other_name>
      <other_name>delandistrogene moxeparvovec-rokl</other_name>
      <other_name>ELEVIDYS</other_name>
    </intervention>
    <eligibility>
      <criteria>
        <textblock>
          Inclusion Criteria:&#xD;
  &#xD;
            -  Received delandistrogene moxeparvovec for Duchenne muscular dystrophy in a previous&#xD;
               clinical study.&#xD;
  &#xD;
            -  Has (a) parent(s) or legal caregiver(s) or is ≥18 years of age and able to understand&#xD;
               and comply with the study visit schedule and all other protocol requirements.&#xD;
  &#xD;
          Exclusion Criteria:&#xD;
  &#xD;
            -  Participant or family does not want to disclose participation with general&#xD;
               practitioner/primary care physician and other medical providers.&#xD;
  &#xD;
          Other inclusion/exclusion criteria may apply.&#xD;
        </textblock>
      </criteria>
      <gender>Male</gender>
      <minimum_age>N/A</minimum_age>
      <maximum_age>N/A</maximum_age>
      <healthy_volunteers>No</healthy_volunteers>
    </eligibility>
    <overall_contact>
      <last_name>Sarepta Therapeutics Inc. For Clinical Trial Information, Select Option 4</last_name>
      <phone>1-888-SAREPTA (1-888-727-3782)</phone>
      <email>SareptAlly@sarepta.com</email>
    </overall_contact>
    <location>
      <facility>
        <name>David Geffen School of Medicine at UCLA</name>
        <address>
          <city>Los Angeles</city>
          <state>California</state>
          <zip>90095</zip>
          <country>United States</country>
        </address>
      </facility>
      <investigator>
        <last_name>Perry Shieh, MD</last_name>
        <role>Principal Investigator</role>
      </investigator>
    </location>
    <location>
      <facility>
        <name>Nationwide Children's Hospital</name>
        <address>
          <city>Columbus</city>
          <state>Ohio</state>
          <zip>43205</zip>
          <country>United States</country>
        </address>
      </facility>
      <investigator>
        <last_name>Jerry Mendell, MD</last_name>
        <role>Principal Investigator</role>
      </investigator>
    </location>
    <location_countries>
      <country>United States</country>
    </location_countries>
    <verification_date>July 2023</verification_date>
    <study_first_submitted>July 21, 2023</study_first_submitted>
    <study_first_submitted_qc>July 21, 2023</study_first_submitted_qc>
    <study_first_posted type="Actual">August 1, 2023</study_first_posted>
    <last_update_submitted>July 21, 2023</last_update_submitted>
    <last_update_submitted_qc>July 21, 2023</last_update_submitted_qc>
    <last_update_posted type="Actual">August 1, 2023</last_update_posted>
    <responsible_party>
      <responsible_party_type>Sponsor</responsible_party_type>
    </responsible_party>
    <keyword>Duchenne Muscular Dystrophy</keyword>
    <keyword>DMD</keyword>
    <keyword>Pediatric</keyword>
    <keyword>Long-term</keyword>
    <keyword>Follow-up</keyword>
    <keyword>Safety</keyword>
    <keyword>Efficacy</keyword>
    <condition_browse>
      <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
      <mesh_term>Muscular Dystrophies</mesh_term>
      <mesh_term>Muscular Dystrophy, Duchenne</mesh_term>
    </condition_browse>
    <!-- Results have not yet been posted for this study                                          -->
  </clinical_study>
  
  
text: 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/NCT05967351
  org study id: SRP-9001-305
  secondary id: 2023-505043-39-00
  nct id: NCT05967351
  lead sponsor: 
  collaborator: 
  has dmc: Yes
  is fda regulated drug: Yes
  is fda regulated device: No
  The purpose of this study is to provide a single clinical study with a uniform approach to
        monitoring long-term safety and efficacy in participants who received delandistrogene
        moxeparvovec in a previous clinical study. No study drug will be administered as part of this
        study. Pre-infusion baseline will be defined as the timepoint just prior to infusion of
        delandistrogene moxeparvovec from a previous clinical study. Each participant will be
        followed for a minimum of 5 years post-infusion of delandistrogene moxeparvovec from a
        previous clinical study. The duration of participation in this study is dependent on the
        length of follow-up the participant completed post-infusion of delandistrogene moxeparvovec
        from a previous clinical study.
  allocation: N/A
  intervention model: Single Group Assignment
  primary purpose: Other
  masking: None (Open Label)
  measure: Number of Participants with a Treatment-emergent Adverse Event (TEAE), Serious Adverse Event (SAE), and Adverse Event of Special Interest (AESI)
  time frame: Up to 5 years after dosing
  measure: Change in the North Star Ambulatory Assessment (NSAA) Total Score From Pre-infusion Baseline to 5 Years Post-infusion of Delandistrogene Moxeparvovec
  time frame: Baseline, 5 years
  measure: Change in Time to Rise From Floor From Pre-infusion Baseline to 5 Years Post-infusion of Delandistrogene Moxeparvovec
  time frame: Baseline, 5 years
  measure: Change in the Time of 10-meter Walk/Run (10MWR) From Pre-infusion Baseline to 5 Years Post-infusion of Delandistrogene Moxeparvovec
  time frame: Baseline, 5 years
  measure: Change in Performance of Upper Limb (PUL) (Version 2.0) Total Scores From Pre-infusion Baseline to 5 Years Post-infusion of Delandistrogene Moxeparvovec
  time frame: Baseline, 5 years
  measure: Change in PUL (Version 2.0) Domain Specific Scores From Pre-infusion Baseline to 5 Years Post-infusion of Delandistrogene Moxeparvovec
  time frame: Baseline, 5 years
  measure: Change in Forced Vital Capacity Percent (FVC%) Predicted From Pre-infusion Baseline to 5 Years Post-infusion of Delandistrogene Moxeparvovec
  time frame: Baseline, 5 years
  measure: Change in Peak Expiratory Flow Percent (PEF%) Predicted From Pre-infusion Baseline to 5 Years Post-infusion of Delandistrogene Moxeparvovec
  time frame: Baseline, 5 years
  measure: Change in Cardiac Magnetic Resonance Imaging (MRI) Findings From Pre-infusion Baseline to 5 Years Post-infusion of Delandistrogene Moxeparvovec
  time frame: Baseline, 5 years
  measure: Change in Musculoskeletal MRI Findings From Pre-infusion Baseline to 5 Years Post-infusion of Delandistrogene Moxeparvovec
  time frame: Baseline, 5 years
  arm group label: Delandistrogene Moxeparvovec
  arm group type: Experimental
  description: Participant received delandistrogene moxeparvovec in a previous clinical study.
  intervention type: Genetic
  intervention name: delandistrogene moxeparvovec
  description: No study drug will be administered as part of this study. Eligible participants who received treatment with delandistrogene moxeparvovec during a previous clinical study will be included.
  arm group label: Delandistrogene Moxeparvovec
  other name: SRP-9001
  other name: delandistrogene moxeparvovec-rokl
  other name: ELEVIDYS
  criteria: 
  gender: Male
  minimum age: N/A
  maximum age: N/A
  healthy volunteers: No
  last name: Sarepta Therapeutics Inc. For Clinical Trial Information, Select Option 4
  phone: 1-888-SAREPTA (1-888-727-3782)
  email: SareptAlly@sarepta.com
  facility: 
  investigator: 
  facility: 
  investigator: 
  country: United States
  responsible party type: Sponsor
  mesh term: Muscular Dystrophies
  mesh term: Muscular Dystrophy, Duchenne