ClinicaliQ Trial Snapshot
- A Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of Delandistrogene Moxeparvovec (SRP-9001) in Non-Ambulatory and Ambulatory Participants With Duchenne Muscular Dystrophy (DMD) — Active Not Recruiting • Phase III • NCT05881408.
- A gene therapy trial testing whether a single infusion can slow muscle weakness in boys with Duchenne muscular dystrophy.
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What This Trial Is Studying
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. Conditions: Duchenne Muscular Dystrophy Interventions: delandistrogene moxeparvovec, placebo Lead Sponsor: Sarepta Therapeutics, Inc. Planned Enrollment: 148 participants