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
- 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
- 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.
Related Research Topics
Explore scientific publications, clinical data analysis, treatment approaches, and expert-compiled information related to the mechanisms and outcomes of this trial. Click any topic for comprehensive research insights.
What molecular mechanisms underlie SRP-9003 gene therapy for β-sarcoglycan deficiency in LGMD2E?
How does systemic gene delivery of SRP-9003 compare to standard-of-care treatments for LGMD2E in clinical trials?
Which biomarkers are used for patient selection and monitoring response to SRP-9003 in LGMD2E trials?
What adverse events have been reported in gene therapy trials for limb-girdle muscular dystrophy subtypes?
Are there combination therapies or competitor drugs targeting β-sarcoglycan deficiency in muscular dystrophy?
Trial Locations
- Locations (1)
Nationwide Children's Hospital
🇺🇸Columbus, Ohio, United States
Nationwide Children's Hospital🇺🇸Columbus, Ohio, United States