A Phase 3 interventional study of delandistrogene moxeparvovec and placebo in Duchenne Muscular Dystrophy, sponsored by Sarepta Therapeutics, Inc.. Active, not recruiting at 46 sites in 14 countries. Open to male participants. Per ClinicalTrials.gov, last updated 2026-05-22.
Sponsored by Sarepta Therapeutics, Inc. · Phase 3, Interventional, and Treatment
The study will evaluate the safety and efficacy of delandistrogene moxeparvovec gene transfer therapy in non-ambulatory and ambulatory males with DMD. This is a randomized, double-blind, placebo-controlled 2-part study. Participants will be in the study for approximately 128 weeks. All participants will have the opportunity to receive intravenous (IV) delandistrogene moxeparvovec in either Part 1 or Part 2.
473 studies on the registry are indexed under Muscular Dystrophy, Duchenne; 107 are open to participants now.
This study's planned enrollment of 148 is above the median of 26 across 326 interventional studies indexed under Muscular Dystrophy, Duchenne.
Browse Muscular Dystrophy, Duchenne studies →Sarepta Therapeutics, Inc. is the lead sponsor of 52 studies on the registry; 8 are open to participants now.
Of its 20 completed or terminated interventional studies of FDA-regulated products, 12 (60%) have results posted.
Counted across the registry records on this site, refreshed daily.
Exclusion Criteria:
Other inclusion or exclusion criteria could apply.
Participants will receive single IV infusion of delandistrogene moxeparvovec on Day 1. Then, participants will receive a single IV infusion of matching placebo at approximately 72 weeks.
Genetic: delandistrogene moxeparvovec · Genetic: placebo
Participants will receive matching placebo IV infusion on Day 1. Then, participants will have the opportunity to receive a single IV infusion of delandistrogene moxeparvovec at approximately 72 weeks.
Genetic: delandistrogene moxeparvovec · Genetic: placebo
Single IV infusion of delandistrogene moxeparvovec
Also known as: SRP-9001, delandistrogene moxeparvovec-rokl, ELEVIDYS
Single IV infusion of matching placebo
Part 1: Change From Baseline in the Total Score of Performance of Upper Limb (PUL) (Version 2.0) at Week 72
Time frame: Baseline, Week 72
Part 1: Change From Baseline in Percent Predicted Forced Vital Capacity (FVC) at Week 72
Time frame: Baseline, Week 72
Part 1: Change From Baseline in Percent Predicted Peak Expiratory Flow (PEF) at Week 72
Time frame: Baseline, Week 72
Part 1: Quantity of Delandistrogene Moxeparvovec Dystrophin Expression at Week 12 as Measured by Western Blot
Time frame: Week 12
Part 1: Change From Baseline in Patient-Reported Outcomes Measurement Information (PROMIS) Score in Upper Extremity Function to Week 72
Time frame: Baseline, Week 72
Number of Participants with a Treatment Emergent Adverse Event (TEAE), Adverse Event of Special Interest (AESI), and Serious Adverse Event (SAE)
Time frame: Baseline up to Week 124
Part 1 (For Cohort 2 Only): Change From Baseline in the North Star Ambulatory Assessment (NSAA) Total Score at Week 72
Time frame: Baseline, Week 72
Part 1: Change From Baseline in Global Circumferential Strain as Measured by Cardiac MRI at Week 72
Time frame: Baseline, Week 72
Part 1: Change From Baseline in the Middle Domain Score of PUL (Version 2.0) at Week 72
Time frame: Baseline, Week 72
This study is active, not recruiting, as verified in May 2026. You cannot join it, but the record below documents what was studied.
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Sarepta Therapeutics, Inc.