This multicenter, global Phase 3 study by Sarepta Therapeutics examines 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). It includes both ambulatory participants (Cohort 1) and non-ambulatory participants (Cohort 2), with eligibility based on cohort-specific mobility test thresholds and the presence of 1 homozygous or 2 heterozygous changes. Participants also receive a glucocorticoid (prednisone or equivalent); the trial is active but no longer recruiting.
The summary above is a plain-language rendering of the official record. The original English title is shown for reference.
Criteria are reproduced from the ClinicalTrials.gov record in the original English. Only the trial team can determine 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.