Trials / Withdrawn
WithdrawnNCT06952686
A Study of SRP-9005 in Limb Girdle Muscular Dystrophy Type 2C/R5 Pediatric and Adult Participants
A Seamless Phase 1/3, Multicenter, Single Dose Systemic Gene Transfer Study to Evaluate the Safety, Tolerability, and Efficacy of SRP-9005 in Limb Girdle Muscular Dystrophy Type 2C/R5 Subjects (COMPASS)
- Status
- Withdrawn
- Phase
- Phase 3
- Study type
- Interventional
- Enrollment
- 0 (actual)
- Sponsor
- Sarepta Therapeutics, Inc. · Industry
- Sex
- All
- Age
- 4 Years
- Healthy volunteers
- Not accepted
Summary
This is a study of a single systemic dose of SRP-9005 in pediatric and adult participants with limb girdle muscular dystrophy type 2C/R5 (LGMD2C/R5). It is comprised of 2 parts (Part A, Part B) that will assess safety and efficacy.
Conditions
Interventions
| Type | Name | Description |
|---|---|---|
| BIOLOGICAL | SRP-9005 | Solution for single IV infusion |
| DRUG | Corticosteroid | Oral tablet (prophylactic) |
Timeline
- Start date
- 2025-06-30
- Primary completion
- 2030-11-30
- Completion
- 2032-03-30
- First posted
- 2025-05-01
- Last updated
- 2025-08-06
Locations
1 site across 1 country: United States
Regulatory
- FDA-regulated drug study
Source: ClinicalTrials.gov record NCT06952686. Inclusion in this directory is not an endorsement.