Gene Delivery Clinical Trial of SRP-9003 (Bidridistrogene Xeboparvovec) for Participants With Limb-Girdle Muscular Dystrophy, Type 2E (LGMD2E) (Beta-Sarcoglycan Deficiency)
Phase 1
Active, not recruiting
- Conditions
- Limb-Girdle Muscular Dystrophy, Type 2E
- Interventions
- Genetic: SRP-9003
- Registration Number
- NCT03652259
- Lead Sponsor
- Sarepta Therapeutics, Inc.
- Brief Summary
The proposed clinical trial is the first-in-human, single-center, open-label, gene delivery study of SRP-9003 (bidridistrogene xeboparvovec) in participants with LGMD2E.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ACTIVE_NOT_RECRUITING
- Sex
- All
- Target Recruitment
- 6
Inclusion Criteria
Not provided
Exclusion Criteria
Not provided
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SEQUENTIAL
- Arm && Interventions
Group Intervention Description Cohort 1: SRP-9003 SRP-9003 Participants will receive a single intravenous (IV) infusion of SRP-9003 at a prespecified dose. Cohort 2: SRP-9003 SRP-9003 Participants will receive a single IV infusion of SRP-9003. Dose will be determined based on the findings from Cohort 1.
- Primary Outcome Measures
Name Time Method Number of Treatment-Emergent Adverse Events (AEs) and Treatment-Emergent Serious Adverse Events (SAEs) Baseline up to 7 years
- Secondary Outcome Measures
Name Time Method Change From Baseline in Quantity of β-SG Protein Expression at Day 60, as Measured by Immunohistochemistry Percent B-SG Positive Fibers Baseline, Day 60 Change From Baseline in Quantity of β-SG Protein Expression at Day 60, as Measured by Immunofluorescence Baseline, Day 60 β-SG gene expression levels will be quantified by immunofluorescence and compared between pre and post muscle biopsies.
Change From Baseline in Quantity of Beta-Sarcoglycan (β-SG) Protein Expression at Day 60, as Measured by Western Blot Baseline, Day 60 β-SG gene expression levels will be quantified by Western Blot and compared between pre and post muscle biopsies.
Trial Locations
- Locations (1)
Nationwide Children's Hospital
🇺🇸Columbus, Ohio, United States