A Study of SRP-9005 in Limb Girdle Muscular Dystrophy Type 2C/R5 Pediatric and Adult Participants

Launched by SAREPTA THERAPEUTICS, INC. · Apr 23, 2025

Keywords

ClinConnect Summary

This clinical trial is studying a treatment called SRP-9005 for people with Limb Girdle Muscular Dystrophy Type 2C/R5, a condition that affects muscle strength and movement. The trial will include both children and adults and is designed to assess how safe and effective a single dose of this treatment is. It has two parts: the first part will look at safety, while the second part will evaluate how well the treatment works.

To be eligible for this study, participants need to be able to walk independently or meet certain criteria if they cannot walk. They must also have specific genetic mutations related to the condition and low levels of certain antibodies in their blood. Participants will undergo screenings to ensure they are healthy enough for the trial, and there are some health issues that could exclude them, such as severe heart problems or recent infections. This trial is not yet recruiting participants, but it aims to provide valuable information about a potential new treatment for this type of muscular dystrophy.

Gender

ALL

Eligibility criteria

• Key Inclusion Criteria:
• • 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 Criteria:
• • 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.