Trials / Terminated
TerminatedNCT05906251
A Gene Transfer Study to Evaluate the Safety, Tolerability and Efficacy of SRP-6004 in Ambulatory Participants With Limb Girdle Muscular Dystrophy, Type 2B/R2 (LGMD2B/R2, Dysferlin [DYSF] Related)
An Open-Label, Systemic Gene Transfer Study to Evaluate the Safety, Tolerability, and Efficacy of SRP 6004 Administered by Systemic Infusion in Ambulatory Subjects With Limb Girdle Muscular Dystrophy Type 2B/R2 (LGMD2B/R2, Dysferlin Related)
- Status
- Terminated
- Phase
- Phase 1
- Study type
- Interventional
- Enrollment
- 2 (actual)
- Sponsor
- Sarepta Therapeutics, Inc. · Industry
- Sex
- All
- Age
- 18 Years – 50 Years
- Healthy volunteers
- Not accepted
Summary
The primary purpose of this study is to evaluate the safety of SRP-6004 administered by intravenous (IV) infusion in ambulatory participants with LGMD2B/R2 (DYSF related).
Conditions
Interventions
| Type | Name | Description |
|---|---|---|
| GENETIC | SRP-6004 | Single IV infusion of SRP-6004 |
Timeline
- Start date
- 2023-05-22
- Primary completion
- 2025-06-13
- Completion
- 2025-06-13
- First posted
- 2023-06-15
- Last updated
- 2025-09-04
Locations
1 site across 1 country: United States
Regulatory
- FDA-regulated drug study
Source: ClinicalTrials.gov record NCT05906251. Inclusion in this directory is not an endorsement.