A Multicenter, Open-label, Single-dose, Systemic Gene Transfer Study to Evaluate the Safety, Tolerability and Efficacy of SRP-9003 on Subjects with Limb Girdle Muscular Dystrophy, Type 2E/R4 (β-Sarcoglycan Deficiency)
Phase: Phase 1 - Active, Not Recruiting
Condition(s): Limb Gridle Muscular Dystrophy 2E/R4
NCT Number: NCT05876780 Other Study ID Number(s): SRP-9003-102
Study Overview
The primary purpose of this study is to evaluate the safety of SRP-9003 and to quantify expression of β-SG in the skeletal muscle of participants with limb-girdle muscular dystrophy, type 2E/R4 (LGMD2E/R4).
Sponsor: Sarepta Therapeutics
Participation Criteria
- Cohort 1 only: Able to walk per protocol specified criteria. - Cohort 2 only: Unable to walk per protocol specified criteria and 4 to 50 years of age.
- Possesses 1 homozygous or 2 heterozygous pathogenic and/or likely pathogenic β-sarcoglycan gene Deoxyribonucleic acid (DNA) (SGCB) gene mutations based on documented clinical findings.
- Ability to cooperate with muscle testing.
- Negative for antibodies to AAVrh74
Study Plan
Participants will receive single IV infusion of SRP-9003 and be monitored at Nationwide Children's Hosptial for 5 years.
Principal Investigator
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