Clinical Trial

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

Study acronym: EMERGENE
Active, Not Recruiting Phase 3
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Summary
This is a multicenter, global study of the effects of a single systemic dose of SRP-9003 on beta-sarcoglycan (β-SG) gene expression in participants with limb-girdle muscular dystrophy, type 2E/R4 (LGMD2E/R4). This study will consist of both ambulatory participants (Cohort 1) and non-ambulatory participants (Cohort 2).
Protocol Amendment History 7 amendments
This ClinicalTrials.gov record has been amended 7 times since 2024-01-30; most recent amendment 2026-02-27.
Status change: Recruiting → Active, Not Recruiting 2025-02-28
Trial Details
NCT Number NCT06246513
Lead Sponsor Sarepta Therapeutics, Inc.
Conditions Limb-girdle Muscular Dystrophy
Enrollment 17 participants
Start Date 2024-01-15
Primary Completion 2025-03-04 (estimated)
Study Completion 2029-11-30 (estimated)
Updated on ClinicalTrials.gov 2026-03-02