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ACTIVE NOT RECRUITING
NCT05876780
PHASE1

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.

View on ClinicalTrials.gov

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

Interventions

GENETIC

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