A Trial to Learn More About an Experimental Gene Therapy Called Bidridistrogene Xeboparvovec (SRP-9003) as a Possible Treatment for Limb Girdle Muscular Dystrophy 2E/R4

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

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).

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.