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NCT05096221 · ClinicalTrials.gov registry record · Phase 3
A Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of Delandistrogene Moxeparvovec (SRP-9001) in Participants With Duchenne Muscular Dystrophy (DMD)
A Phase 3 study of Duchenne Muscular Dystrophy, sponsored by Sarepta Therapeutics.
- Completed
- Registry status
- Phase 3
- Development phase
- 126
- Enrollment target
- 20
- Study locations
NCT05096221: Completed Phase 3 study of Duchenne Muscular Dystrophy, sponsored by Sarepta Therapeutics.
NCT05096221 is a Phase 3 study of Duchenne Muscular Dystrophy that has completed, run by Sarepta Therapeutics. The registered enrollment target is 126 participants, below the 410-participant average among 48 other Duchenne Muscular Dystrophy trials with a reported enrollment target (69% lower). The trial reports 20 study locations across 16 states. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT05096221, a Phase 3 study of Duchenne Muscular Dystrophy, has completed, sponsored by Sarepta Therapeutics.
- COMPLETED
- Registry status
- Phase 3
- Development phase
- 126 participants
- Enrollment target
- 20
- Study locations
Study Summary
The study will evaluate the safety and efficacy of gene transfer therapy in boys with DMD. It is a randomized, double-blind, placebo-controlled study. The participants who are randomized to the placebo arm will have an opportunity for treatment with gene transfer therapy at the beginning of the second year.
Primary Outcome
The NSAA is a healthcare provider administered scale that rates performance of various motor abilities in ambulant children with Duchenne Muscular Dystrophy and is used to monitor disease progression and treatment effects. During assessment, participants are asked to perform 17 different functional activities that are graded as: 2 - "Normal" - no obvious modification of activity; 1 - Modified method but achieves goal independent of assistance; 0 - Unable to achieve independently. The NSAA total
Conditions Studied
Interventions
- GENETIC delandistrogene moxeparvovec
- GENETIC placebo
Study Locations (20)
California
- UC San Diego Altman Clinical and Translational Research Institute - La Jolla
- UCLA Medical Center - Los Angeles
- Lucile Packard Children's Hospital at Stanford - Palo Alto
- University of California, Davis - Sacramento
New York
- Columbia University/NYPH - New York
- University of Rochester - Rochester
Arkansas
- Arkansas Children's - Little Rock
Colorado
- Children's Hospital Colorado - Aurora
Florida
- University of Florida - Gainesville
Illinois
- Lurie Children's Hospital of Chicago - Chicago
Iowa
- University of Iowa Stead Family Children's Hospital - Iowa City
Maryland
- The Johns Hopkins Hospital - Baltimore
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 126 participants |
| Start Date | 2021-10-27 |
| Est. Completion | 2024-10-25 |
| Phase | Phase 3 |
What the finished NCT05096221 record still lists
NCT05096221 is an interventional study that assigns participants to a tested intervention. The registered 126 participants enrollment target is mid-sized for trials with a published cap, below the 410-participant average among 48 other Duchenne Muscular Dystrophy trials with a reported enrollment target (69% lower).
The record links to 1 condition, with Duchenne Muscular Dystrophy appearing as the primary indexed condition, and to 2 interventions - of which delandistrogene moxeparvovec is the first listed.
NCT05096221 names 20 study sites across 16 states, led by California, New York, Arkansas.
Frequently Asked Questions
What is clinical trial NCT05096221 about?
NCT05096221 is a clinical study titled "A Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of Delandistrogene Moxeparvovec (SRP-9001) in Participants With Duchenne Muscular Dystrophy (DMD)". The study will evaluate the safety and efficacy of gene transfer therapy in boys with DMD. It is a randomized, double-blind, placebo-controlled study. The participants who are randomized to the placebo arm will have an opportunity for treatment with gene transfer therapy at the beginning of the seco...
What is the current status of trial NCT05096221?
This trial is currently completed. It is a Phase 3 study. The enrollment target is 126 participants. The study started on 2021-10-27. Estimated completion is 2024-10-25.
What conditions does trial NCT05096221 study?
This clinical trial studies the following conditions: Duchenne Muscular Dystrophy.
What interventions are being tested in trial NCT05096221?
The interventions under investigation include: delandistrogene moxeparvovec (GENETIC), placebo (GENETIC).
Who is sponsoring clinical trial NCT05096221?
This trial is sponsored by Sarepta Therapeutics, which has 33 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT05096221 being conducted?
This trial has 20 study locations across Arkansas, California, Colorado, Florida, Illinois. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
Learn More About Clinical Trials
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Where NCT05096221's enrollment target sits among peer trials
126 12th of 48 higher than 37 of 48 other Duchenne Muscular Dystrophy trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Duchenne Muscular Dystrophy trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.
Source ClinicalTrials.gov registry export · 2026-08-08
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