Trials / Active Not Recruiting
Active Not RecruitingNCT06246513
A Trial to Learn More About an Experimental Gene Therapy Called Bidridistrogene Xeboparvovec (SRP-9003) as a Possible Treatment for Limb Girdle Muscular Dystrophy 2E/R4
A Phase 3 Multinational, Open-label, Systemic Gene Delivery Study to Evaluate the Safety and Efficacy of SRP-9003 in Subjects With Limb Girdle Muscular Dystrophy 2E/R4
- Status
- Active Not Recruiting
- Phase
- Phase 3
- Study type
- Interventional
- Enrollment
- 17 (actual)
- Sponsor
- Sarepta Therapeutics, Inc. · Industry
- Sex
- All
- Age
- 4 Years
- Healthy volunteers
- Not accepted
Summary
This is a multicenter, global study of the effects of a single systemic dose of SRP-9003 on beta-sarcoglycan (β-SG) gene expression in participants with limb-girdle muscular dystrophy, type 2E/R4 (LGMD2E/R4). This study will consist of both ambulatory participants (Cohort 1) and non-ambulatory participants (Cohort 2).
Conditions
Interventions
| Type | Name | Description |
|---|---|---|
| BIOLOGICAL | SRP-9003 | Solution for single IV infusion |
| DRUG | Glucocorticoid | Oral tablet (prophylactic) |
Timeline
- Start date
- 2024-01-15
- Primary completion
- 2025-03-04
- Completion
- 2029-11-30
- First posted
- 2024-02-07
- Last updated
- 2026-03-02
Locations
10 sites across 6 countries: United States, Belgium, Germany, Italy, Spain, United Kingdom
Regulatory
- FDA-regulated drug study
Source: ClinicalTrials.gov record NCT06246513. Inclusion in this directory is not an endorsement.