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
- Conditions
- Limb-girdle Muscular Dystrophy
- Interventions
- Registration Number
- NCT06246513
- Lead Sponsor
- Sarepta Therapeutics, Inc.
- Brief Summary
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).
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ACTIVE_NOT_RECRUITING
- Sex
- All
- Target Recruitment
- 17
-
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.
- 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.
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SINGLE_GROUP
- Arm && Interventions
Group Intervention Description SRP-9003 SRP-9003 Participants will receive a single intravenous (IV) infusion of SRP-9003. SRP-9003 Glucocorticoid Participants will receive a single intravenous (IV) infusion of SRP-9003.
- Primary Outcome Measures
Name Time Method Cohort 1: 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
- Secondary Outcome Measures
Name Time Method Cohort 1 and Cohort 2: Change From Baseline in β-SG Expression at Day 60 Post-dose as Measured by Western Blot Baseline, Day 60 Cohort 1 and Cohort 2: Change From Baseline Through Month 60 in Performance of Upper Limb Version 2.0 (PUL 2.0) Total Score Baseline through Month 60 Cohort 1: Change From Baseline Through Month 60 in the Time to Rise from the Floor Test Baseline through Month 60 Cohort 1: Change From Baseline Through Month 60 in the Time to Complete the 10-meter Walk/Run (10MWR) Test Baseline through Month 60 Cohort 1: Change From Baseline Through Month 60 in the Time to Ascend 4 Steps Test Baseline through Month 60 Cohort 1: Change From Baseline Through Month 60 in the Time to Complete the 100-meter Walk/Run (100MWR) Test Baseline through Month 60 Cohort 1: Change From Baseline Through Month 60 in the Timed Up and Go Test Baseline through Month 60 Number of Participants Experiencing Treatment-emergent Adverse Events (TEAEs) Baseline through Month 60 Time to Change of Loss of Ambulation Baseline through Month 60 Cohort 1 and Cohort 2: Change From Baseline in β-SG Expression at Day 60 Post-dose as Measured by IF Percent Fluorescent Intensity (PFI) Baseline, Day 60 Cohort 2: Change From Baseline in β-SG Expression at Day 60 Post-dose as Measured by IF Percent β-SG Positive Fibers (PβSGPF) Baseline, Day 60 Cohort 1 and Cohort 2: Change From Baseline Through Month 60 in North Star Assessment for Dysferlinopathy (NSAD) Total Score Baseline through Month 60 Change From Baseline Through Month 60 in Creatine Kinase Level Baseline through Month 60
Trial Locations
- Locations (5)
University of California, San Diego-Altman Clinical and Translational Research Institute
🇺🇸La Jolla, California, United States
Nationwide Childrens Hospital
🇺🇸Columbus, Ohio, United States
The Children's Hospital of Philadelphia
🇺🇸Philadelphia, Pennsylvania, United States
Children's Hospital of The King's Daughter
🇺🇸Norfolk, Virginia, United States
Newcastle University
🇬🇧Newcastle upon Tyne, United Kingdom