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

Sarepta Therapeutics

Status and phase: Terminated; Phase 2; Phase 1

Conditions: Limb-Girdle Muscular Dystrophy, Type 2E

Treatments: Genetic: SRP-9003

Study type: Interventional

Funder types: Industry

Identifiers

NCT03652259

IRB17-00253 (Other Identifier)

SRP-9003-101

Details and patient eligibility

About

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.

Enrollment: 6 patients

Sex: All

Ages: 4 to 15 years old

Volunteers: No Healthy Volunteers

Inclusion and exclusion criteria

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.

Trial design

Primary purpose: Treatment

Allocation: Non-Randomized

Interventional model: Sequential Assignment

Masking: None (Open label)

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6 participants in 2 patient groups

Cohort 1: SRP-9003

Experimental group

Description:

Participants will receive a single intravenous (IV) infusion of SRP-9003 at a prespecified dose.

Treatment:

Genetic: SRP-9003

Cohort 2: SRP-9003

Experimental group

Description:

Participants will receive a single IV infusion of SRP-9003. Dose will be determined based on the findings from Cohort 1.

Treatment:

Genetic: SRP-9003