A Long-term Follow-up Study of Participants Who Received Delandistrogene Moxeparvovec (SRP-9001) in a Previous Clinical Study

Study Overview
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Brief Summary
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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.

Official Title
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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

Conditions
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Duchenne Muscular Dystrophy

Intervention / Treatment
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* Genetic: delandistrogene moxeparvovec


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Received delandistrogene moxeparvovec for Duchenne muscular dystrophy in a previous clinical study. Has (a) parent(s) or legal caregiver(s) or is ≥18 years of age and able to understand and comply with the study visit schedule and all other protocol requirements. Exclusion Criteria: Participant or family does not want to disclose participation with general practitioner/primary care physician and other medical providers. Other inclusion/exclusion criteria may apply.
Sexes Eligible for Study
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Male

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Other
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Delandistrogene Moxeparvovec<br>Participant received delandistrogene moxeparvovec in a previous clinical study. | Genetic: delandistrogene moxeparvovec<br>* 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.<br>* Other names: ELEVIDYS;|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Number of Participants with a Treatment-emergent Adverse Event (TEAE), Serious Adverse Event (SAE), and Adverse Event of Special Interest (AESI) |  | Up to 5 years after dosing | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Change in the North Star Ambulatory Assessment (NSAA) Total Score From Pre-infusion Baseline to 5 Years Post-infusion of Delandistrogene Moxeparvovec |  | Baseline, 5 years | 
| Change in Time to Rise From Floor From Pre-infusion Baseline to 5 Years Post-infusion of Delandistrogene Moxeparvovec |  | Baseline, 5 years | 
| Change in the Time of 10-meter Walk/Run (10MWR) From Pre-infusion Baseline to 5 Years Post-infusion of Delandistrogene Moxeparvovec |  | Baseline, 5 years | 
| Change in Performance of Upper Limb (PUL) (Version 2.0) Total Scores From Pre-infusion Baseline to 5 Years Post-infusion of Delandistrogene Moxeparvovec |  | Baseline, 5 years | 
| Change in PUL (Version 2.0) Domain Specific Scores From Pre-infusion Baseline to 5 Years Post-infusion of Delandistrogene Moxeparvovec |  | Baseline, 5 years | 
| Change in Forced Vital Capacity Percent (FVC%) Predicted From Pre-infusion Baseline to 5 Years Post-infusion of Delandistrogene Moxeparvovec |  | Baseline, 5 years | 
| Change in Peak Expiratory Flow Percent (PEF%) Predicted From Pre-infusion Baseline to 5 Years Post-infusion of Delandistrogene Moxeparvovec |  | Baseline, 5 years | 
| Change in Cardiac Magnetic Resonance Imaging (MRI) Findings From Pre-infusion Baseline to 5 Years Post-infusion of Delandistrogene Moxeparvovec |  | Baseline, 5 years | 
| Change in Musculoskeletal MRI Findings From Pre-infusion Baseline to 5 Years Post-infusion of Delandistrogene Moxeparvovec |  | Baseline, 5 years | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Duchenne Muscular Dystrophy, DMD, Pediatric, Long-term, Follow-up, Safety, Efficacy