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

Trial ID: NCT06246513

Interventional - Phase 3 - Active, not recruiting

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). This study will consist of both ambulatory participants (Cohort 1) and non-ambulatory participants (Cohort 2).

Conditions

• Limb-girdle Muscular Dystrophy

Interventions

• SRP-9003
• Glucocorticoid

Eligibility

Inclusion criteria

• Cohort 1, only ambulatory participants:
• Able to walk without assistive aid
• 10MWR \<30 seconds
• NSAD ≥25
• Cohort 2, only non-ambulatory participants:
• 10MWR ≥30 seconds or unable to perform
• PUL 2.0 entry scale score ≥3
• 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.

Exclusion criteria

• Left ventricular ejection fraction \< 40% or clinical signs and/or symptoms of cardiomyopathy
• Forced vital capacity ≤40% of predicted value and/or requirement for nocturnal ventilation
• Diagnosis of (or ongoing treatment for) an autoimmune disease and on active immunosuppressant treatment
• Presence of any other clinically significant illness or medical condition (other than LGMD2E/R4)
• Other inclusion/exclusion criteria apply.

Sponsor

Sarepta Therapeutics, Inc.

Source: ClinicalTrials.gov

Search more clinical trials on myTomorrows