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 - Active, Not Recruiting
Condition(s): Limb Girdle Muscular Dystrophy 2E/R4
NCT Number: NCT03652259 Other Study ID Number(s): SRP-9003-101
Study Overview
First in human, single center, open-label gene delivery study of SRP-9003 (bidridistrogene xeboparvovec) in participants with LGMD2E.
Sponsor: Sarepta Therapeutics
Participation 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 and gender
Study Plan
All participants received a single intravenous (IV) infusion of bidridistrogene xeboparvovec (SRP-9003) at a prespecified dose. Participants will be monitored after the infusion for 7 years.
Principal Investigator
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