Gene Delivery Clinical Trial of SRP-9003 (Bidridistrogene Xeboparvovec) for Participants With Limb-Girdle Muscular Dystrophy, Type 2E (LGMD2E) (Beta-Sarcoglycan Deficiency)

Stopped early · Phase 1/Phase 2

Conditions studied: Limb-Girdle Muscular Dystrophy, Type 2E

In brief

The proposed clinical trial is the first-in-human, single-center, open-label, gene delivery study of SRP-9003 (bidridistrogene xeboparvovec) in participants with LGMD2E.

Key facts

Study ID

NCT03652259

Run by

Sarepta Therapeutics, Inc.

People needed

6

Starts

2018-10-27

Expected to finish

2025-01-14

Last updated by the study team

2026-01-20

Who can join

Age: 4 and older, up to 15. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• Males or females of any ethnic group
• β-SG deoxyribonucleic acid (DNA) gene mutations at both alleles
• Weakness demonstrated based on history of difficulty in running, jumping and climbing stairs
• A 100 meter walk/run (MWR) test result: ≥40 % of predicted for age-, height-, gender-, and weight-matched healthy controls at the screening visit

You may not qualify if…

• Active viral infection based on clinical observations
• Cardiac magnetic resonance imaging (MRI) determined left ventricular ejection fraction (LVEF) <40%
• Serological evidence of human immunodeficiency virus (HIV), hepatitis B, or hepatitis C infection
• Diagnosis of (or ongoing treatment for) an autoimmune disease
• Abnormal laboratory values considered clinically significant
• Concomitant illness or requirement for chronic drug treatment that, in the opinion of the Principal Investigator, creates unnecessary risks for gene transfer.
• Other inclusion/exclusion criteria apply.

Where it is running

• Nationwide Children's Hospital — Columbus, Ohio, United States

Full record on ClinicalTrials.gov

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