SRP-9003
Sponsors
Sarepta Therapeutics Inc., Sarepta Therapeutics, Inc.
Conditions
Limb Girdle Muscular DystrophyLimb-Girdle Muscular Dystrophy, Type 2ELimb-girdle Muscular DystrophyLimb-girdle muscular dystrophytype 2E/R4 (LGMD2E/R4) or β-sarcoglycanopathy
Phase 1
Gene Delivery Clinical Trial of SRP-9003 (Bidridistrogene Xeboparvovec) for Participants With Limb-Girdle Muscular Dystrophy, Type 2E (LGMD2E) (Beta-Sarcoglycan Deficiency)
TerminatedNCT03652259
Start: 2018-10-27End: 2025-01-14Updated: 2026-01-20
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)
Active, not recruitingNCT05876780
Start: 2022-12-19End: 2028-08-28Updated: 2026-02-19
Phase 3
A Trial to Learn More About an Experimental Gene Therapy Called Bidridistrogene Xeboparvovec (SRP-9003) as a Possible Treatment for Limb Girdle Muscular Dystrophy 2E/R4
Active, not recruitingNCT06246513
Start: 2024-01-15End: 2029-11-30Updated: 2026-03-02
A Phase 3 Multinational, Open-label, Systemic Gene Delivery Study to Evaluate the Safety and Efficacy of SRP-9003 in Subjects with Limb Girdle Muscular Dystrophy 2E/R4 (EMERGENE)
RecruitingCTIS2022-503112-17-00
Start: 2024-10-16Target: 8Updated: 2025-10-21