A Phase 3 Multinational, Open-label, Systemic Gene Delivery Study to Evaluate the Safety and Efficacy of SRP-9003 in Subjects with Limb Girdle Muscular Dystrophy 2E/R4 (EMERGENE)
Phase: Phase 3 - Active, Not Recruiting
Condition(s): Limb Girdle Muscular Dystrophy 2E/R4
Keyword(s): 2022-503112-17-00 ( Other Identifier ) (OTHER: Clinical Trials Information System (CTIS)
NCT Number: NCT06246513 Other Study ID Number(s): SRP-9003-301
Study Overview
This is a multicenter, global study of the effects of a single systemic dose of SRP-9003 on beta-sarcoglycan (β-SG) gene expression in participants with limb-girdle muscular dystrophy, type 2E/R4 (LGMD2E/R4).
Participation Criteria
- Cohort 1, only ambulatory participants:
- Able to walk without assistive aid
- 10-meter walk test (10MWT) <30 seconds
- NSAD ≥25
- Cohort 2, only non-ambulatory participants:
- 10MWT ≥30 seconds or unable to perform
- PUL 2.0 entry scale score ≥3
- Aged 4 years and older
- Participants must possess 1 homozygous or 2 heterozygous pathogenic and/or likely pathogenic β-SG DNA gene mutations
- Able to cooperate with muscle testing
- Participants must have adeno-associated virus serotype rh74 (AAVrh74) antibody titers <1:400 (that is, not elevated) as determined by AAVrh74 antibody enzyme-linked immunosorbent assay.
Study Plan
Participants will receive a single intravenous (IV) infusion of SRP-9003. The participants will be monitored for 5 years after the infusion.
Principal Investigator
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