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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)
Sponsor: Sarepta Therapeutics, Inc.
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.
Official title: A Multicenter, Open-label, Single-dose, Systemic Gene Transfer Study to Evaluate the Safety, Tolerability, and Efficacy of SRP-9003 on Subjects With Limb Girdle Muscular Dystrophy, Type 2E/R4 (β-Sarcoglycan Deficiency)
Key Details
Gender
All
Age Range
4 Years - 50 Years
Study Type
INTERVENTIONAL
Enrollment
6
Start Date
2022-12-19
Completion Date
2028-08-28
Last Updated
2026-02-19
Healthy Volunteers
No
Conditions
Interventions
SRP-9003
Single IV infusion of SRP-9003
Locations (2)
Nationwide Children's Hospital
Columbus, Ohio, United States
St. Jude Children's Research Hospital
Memphis, Tennessee, United States