Clinical Trial

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 Recruiting Phase 1
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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.
Protocol Amendment History 4 amendments
This ClinicalTrials.gov record has been amended 4 times since 2023-05-17; most recent amendment 2026-02-17.
Status change: Enrolling by Invitation → Active, Not Recruiting 2023-09-18
Trial Details
NCT Number NCT05876780
Lead Sponsor Sarepta Therapeutics, Inc.
Conditions Limb Girdle Muscular Dystrophy
Enrollment 6 participants
Start Date 2022-12-19
Primary Completion 2028-08-28 (estimated)
Study Completion 2028-08-28 (estimated)
Updated on ClinicalTrials.gov 2026-02-19