A Long-term Follow-up Study of Participants Who Received Delandistrogene Moxeparvovec (SRP-9001) in a Previous Clinical Study
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
Sponsor: Sarepta Therapeutics, Inc. + Hoffmann-La Roche
No open prediction endpoints
Endpoints are classified and published by ProgramSignal analysts.
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Primary Endpoints (CT.gov)
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 10 years
Secondary Endpoints
Change in the North Star Ambulatory Assessment (NSAA) Total Score From Pre-infusion Baseline of Delandistrogene Moxeparvovec to the End of the Study Participation
Change in Time to Rise From Floor From Pre-infusion Baseline of Delandistrogene Moxeparvovec to the End of the Study Participation
Change in the Time of 10-meter Walk/Run (10MWR) From Pre-infusion Baseline of Delandistrogene Moxeparvovec to the End of the Study Participation
Eligibility Criteria
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: * Not applicable
✦ Analyst Commentary
Expert commentary on why this trial matters and what to watch for.
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