Optic IconOptic Logo
|
SearchCollectionsBenchmarks
Clinical trial

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

ID
Name
SRP-9003-301
Description
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).
Trial arms
Trial start
2024-01-15
Estimated PCD
2025-01-31
Trial end
2029-11-30
Status
Recruiting
Phase
Early phase I
Treatment
SRP-9003
Solution for single IV infusion
Arms:
SRP-9003
Other names:
scAAVrh74.MHCK7.hSGCB, bidridistrogene xeboparvovec
Glucocorticoid
Oral tablet (prophylactic)
Arms:
SRP-9003
Other names:
Prednisone or equivalent
Size
15
Primary endpoint
Change from Baseline in β-SG Expression at Day 60 Post-dose as Measured by Immunofluorescence (IF) Percent β-SG Positive Fibers
Baseline, Day 60
Eligibility criteria
Inclusion 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 * 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.
Protocol
{'studyType': 'INTERVENTIONAL', 'phases': ['PHASE3'], 'designInfo': {'allocation': 'NA', 'interventionModel': 'SINGLE_GROUP', 'primaryPurpose': 'TREATMENT', 'maskingInfo': {'masking': 'NONE'}}, 'enrollmentInfo': {'count': 15, 'type': 'ESTIMATED'}}
Updated at
2024-02-07

1 organization

2 products

1 indication

Organization
Sarepta Therapeutics
Product
Glucocorticoid
Indication
Limb-girdle Muscular Dystrophy
Product
SRP-9003