Status:
WITHDRAWN
A Study of SRP-9005 in Limb Girdle Muscular Dystrophy Type 2C/R5 Pediatric and Adult Participants
Lead Sponsor:
Sarepta Therapeutics, Inc.
Conditions:
Muscular Dystrophies, Limb-Girdle
Eligibility:
All Genders
4+ years
Phase:
PHASE3
Brief Summary
This is a study of a single systemic dose of SRP-9005 in pediatric and adult participants with limb girdle muscular dystrophy type 2C/R5 (LGMD2C/R5). It is comprised of 2 parts (Part A, Part B) that w...
Eligibility Criteria
Inclusion
- Key
- Ambulatory, defined as able to walk without assistive aid, 10MWR \<30 seconds, and NSAD total score ≥20
- Non-ambulatory, defined as 10MWR ≥30 seconds or unable to perform, and PUL 2.0 entry scale score ≥3
- Possess 1 homozygous or 2 heterozygous pathogenic and/or likely pathogenic gamma-SG deoxyribonucleic acid (DNA) gene mutations as documented prior to pre-Infusion screening. Results to be confirmed by sponsor at a CLIA/CAP/ISO15189 certified laboratory prior to dosing.
- Have AAVrh74 antibody titers \<1:400 (that is, not elevated) as determined by an enzyme-linked immunoassay
- Key
Exclusion
- Has a symptomatic infection (for example, upper respiratory tract infection, pneumonia, pyelonephritis, meningitis) within 4 weeks before study treatment infusion
- Part A: Has left ventricular ejection fraction (LVEF) \<50% on the screening echocardiogram (ECHO) (without use of cardiac medication) or clinical signs and/or symptoms of cardiomyopathy or any history of cardiac disease
- Part B: Has LVEF \<40% on the screening ECHO or clinical signs and/or symptoms of cardiomyopathy
- Has FVC \<40% of predicted value at screening and/or requirement for nocturnal ventilation
- Serological evidence of current, chronic, or active human immunodeficiency virus infection, or hepatitis B or C infection or active viral or bacterial infection based on clinical observations
- Any prior treatment with gene therapy, cell-based therapy (for example, stem cell transplantation), clustered regularly interspaced short palindromic repeats/CRISPR-associated protein 9 (CRISPR/Cas9), or any other form of gene editing
- Treatment with human growth factor within 3 months of Day 1
- Treatment with any investigational medication within 6 months of the screening visit
- Is unable to undergo or tolerate a cardiac MRI procedure for any reason
- Note: Other inclusion/exclusion criteria may apply.
Key Trial Info
Start Date :
June 30 2025
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
March 30 2032
Estimated Enrollment :
Patients enrolled
Trial Details
Trial ID
NCT06952686
Start Date
June 30 2025
End Date
March 30 2032
Last Update
August 6 2025
Active Locations (1)
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1
Children's Hospital of the King's Daughters
Norfolk, Virginia, United States, 23510