Title
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)
Phase
Phase 1/Phase 2Lead Sponsor
Sarepta TherapeuticsStudy Type
InterventionalStatus
Active, not recruitingIndication/Condition
Limb-Girdle Muscular Dystrophy, Type 2E [, ]Intervention/Treatment
SRP-9003Study Participants
6The proposed clinical trial is the first-in-human, single-center, open-label, gene delivery study of SRP-9003 (bidridistrogene xeboparvovec) in participants with LGMD2E.
SRP-9003 will be administered through a single systemic injection.
Participants will receive a single intravenous (IV) infusion of SRP-9003 at a prespecified dose.
Participants will receive a single IV infusion of SRP-9003. Dose will be determined based on the findings from Cohort 1.
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.
