Trials / Completed
CompletedNCT05096221
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 Multinational, Randomized, Double-Blind, Placebo-Controlled Systemic Gene Delivery Study to Evaluate the Safety and Efficacy of SRP-9001 in Subjects With Duchenne Muscular Dystrophy (EMBARK)
- Status
- Completed
- Phase
- Phase 3
- Study type
- Interventional
- Enrollment
- 126 (actual)
- Sponsor
- Sarepta Therapeutics, Inc. · Industry
- Sex
- Male
- Age
- 4 Years – 7 Years
- Healthy volunteers
- Not accepted
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
Interventions
| Type | Name | Description |
|---|---|---|
| GENETIC | delandistrogene moxeparvovec | Single IV infusion of delandistrogene moxeparvovec. |
| GENETIC | placebo | Single IV infusion of matching placebo. |
Timeline
- Start date
- 2021-10-27
- Primary completion
- 2023-10-04
- Completion
- 2024-10-25
- First posted
- 2021-10-27
- Last updated
- 2025-07-08
- Results posted
- 2024-12-10
Locations
42 sites across 9 countries: United States, Belgium, Germany, Hong Kong, Italy, Japan, Spain, Taiwan, United Kingdom
Regulatory
- FDA-regulated drug study
Source: ClinicalTrials.gov record NCT05096221. Inclusion in this directory is not an endorsement.