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A Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of Delandistrogene Moxeparvovec (SRP-9001) in Participants With Duchenne Muscular Dystrophy (DMD)
NCT05096221 · View on ClinicalTrials.gov ↗
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.
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 |
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Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT05096221
The ClinicalTrials.gov registry entry for NCT05096221 describes a study currently listed as completed. It is categorized as Phase 3, which is the standard way researchers label where a study sits along the investigational pathway from early safety work through later efficacy and post-marketing evaluation. The registered enrollment target is 126 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is Sarepta Therapeutics, which has 10 total studies on file at ClinicalTrials.gov, and sponsors are the parties responsible for study design, oversight, and regulatory filings.
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. Interventions can include drugs, devices, procedures, behavioral programs, or observational arms, and each is tracked as a separate registry field so that downstream queries can filter accurately. When a trial lists multiple interventions, it usually reflects a multi-arm design or a comparison protocol rather than a single treatment being tested in isolation. The brief summary published in the registry is the clearest source of protocol intent and should be read before drawing conclusions from any sidebar tags.
Geographic footprint matters for practical reasons: NCT05096221 reports 20 study locations spanning 16 distinct geographic areas — top geographies include California, New York, Arkansas. A larger site network tends to correlate with broader recruitment capacity, but it does not imply anything about study quality, and site-level enrollment status can diverge from the overall registry status shown above. Every data point on this page comes from the public ClinicalTrials.gov dataset and is reproduced here for reference only; it is not a medical recommendation, an endorsement of the sponsor, or an invitation to enroll. Verify current status, eligibility criteria, and contact details directly at ClinicalTrials.gov, and discuss any participation decision with your own healthcare provider.
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. These conditions were identified from the trial registry and reflect the primary focus areas of the research.
What interventions are being tested in trial NCT05096221?
The interventions under investigation include: delandistrogene moxeparvovec (GENETIC), placebo (GENETIC). Each intervention is being evaluated for safety and efficacy as part of this clinical study.
Who is sponsoring clinical trial NCT05096221?
This trial is sponsored by Sarepta Therapeutics, which has 10 total clinical trials registered on ClinicalTrials.gov. The sponsor is responsible for the study's design, funding, and regulatory compliance.
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.
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