Trials / Terminated
TerminatedNCT03652259
Gene Delivery Clinical Trial of SRP-9003 (Bidridistrogene Xeboparvovec) for Participants With Limb-Girdle Muscular Dystrophy, Type 2E (LGMD2E) (Beta-Sarcoglycan Deficiency)
A Single-Center, Open-Label, Systemic Gene Delivery Study to Evaluate the Safety, Tolerability, and Efficacy of SRP-9003 Administered by Systemic Infusion in Subjects With LGMD2E (β-Sarcoglycan Deficiency)
- Status
- Terminated
- Phase
- Phase 1 / Phase 2
- Study type
- Interventional
- Enrollment
- 6 (actual)
- Sponsor
- Sarepta Therapeutics, Inc. · Industry
- Sex
- All
- Age
- 4 Years – 15 Years
- Healthy volunteers
- Not accepted
Summary
The proposed clinical trial is the first-in-human, single-center, open-label, gene delivery study of SRP-9003 (bidridistrogene xeboparvovec) in participants with LGMD2E.
Conditions
Interventions
| Type | Name | Description |
|---|---|---|
| GENETIC | SRP-9003 | SRP-9003 will be administered through a single systemic injection. |
Timeline
- Start date
- 2018-10-27
- Primary completion
- 2025-01-14
- Completion
- 2025-01-14
- First posted
- 2018-08-29
- Last updated
- 2026-01-20
Locations
1 site across 1 country: United States
Regulatory
- FDA-regulated drug study
Source: ClinicalTrials.gov record NCT03652259. Inclusion in this directory is not an endorsement.