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

A Single-Center, Open-Label, Systemic Gene Delivery Study to Evaluate the Safety, Tolerability, and Efficacy of SRP-9003 Administered by Systemic Infusion in Subjects With LGMD2E (β-Sarcoglycan Deficiency)

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.

INCLUSION CRITERIA

• 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

EXCLUSION CRITERIA

• 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.