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SRP-9004 in LGMD2D/R3
NCT06747273Phase 1Terminated
- Sponsor
- Sarepta Therapeutics
- Start
- Jan 2025
- Primary completion
- Jun 2025
- Modality
- Gene Editing, Gene Silencing, Gene Addition/Replacement
- Countries
- 1
01
Overview
Phase 1, open-label, single-arm trial of patidistrogene bexoparvovec in limb-girdle muscular dystrophy type 2D (LGMD2D), sponsored by Sarepta Therapeutics. Primary endpoint: Number of Participants with Treatment-emergent Adverse Events (TEAEs), Adverse Events of Special Interest (AESI) and Treatment-emergent Serious Adverse Events (SAEs) up to 60 months. Started Jan 2025; status: terminated.
02
Drugs
patidistrogene bexoparvovec
03
Conditions
limb-girdle muscular dystrophy type 2D (LGMD2D)
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