Trials / Active Not Recruiting
Active Not RecruitingNCT05876780
A Gene Transfer Single Dose Study to Evaluate the Safety, Tolerability and Efficacy of SRP-9003 in Non-Ambulatory and Ambulatory Participants With Limb Girdle Muscular Dystrophy, Type 2E/R4 (Beta-Sarcoglycan [β-SG] Deficiency)
A Multicenter, Open-label, Single-dose, Systemic Gene Transfer Study to Evaluate the Safety, Tolerability, and Efficacy of SRP-9003 on Subjects With Limb Girdle Muscular Dystrophy, Type 2E/R4 (β-Sarcoglycan Deficiency)
- Status
- Active Not Recruiting
- Phase
- Phase 1
- Study type
- Interventional
- Enrollment
- 6 (actual)
- Sponsor
- Sarepta Therapeutics, Inc. · Industry
- Sex
- All
- Age
- 4 Years – 50 Years
- Healthy volunteers
- Not accepted
Summary
The primary purpose of this study is to evaluate the safety of SRP-9003 and to quantify expression of β-SG in the skeletal muscle of participants with limb-girdle muscular dystrophy, type 2E/R4 (LGMD2E/R4). The study will include both ambulatory (Cohort 1) and non-ambulatory (Cohort 2) participants.
Conditions
Interventions
| Type | Name | Description |
|---|---|---|
| GENETIC | SRP-9003 | Single IV infusion of SRP-9003 |
Timeline
- Start date
- 2022-12-19
- Primary completion
- 2028-08-28
- Completion
- 2028-08-28
- First posted
- 2023-05-25
- Last updated
- 2026-02-19
Locations
2 sites across 1 country: United States
Regulatory
- FDA-regulated drug study
Source: ClinicalTrials.gov record NCT05876780. Inclusion in this directory is not an endorsement.