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

NCT ID: NCT06952686

Last Updated: 2025-08-06

Basic Information

• Recruitment Status: WITHDRAWN
• Clinical Phase: PHASE3
• Study Classification: INTERVENTIONAL
• Study Start Date: 2025-06-30
• Study Completion Date: 2032-03-30

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 will assess safety and efficacy.

Conditions

Muscular Dystrophies, Limb-Girdle

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

SRP-9005

Participants will receive a single intravenous (IV) infusion of SRP-9005.

Group Type: EXPERIMENTAL

SRP-9005

Intervention Type: BIOLOGICAL

Solution for single IV infusion

Corticosteroid

Intervention Type: DRUG

Oral tablet (prophylactic)

Interventions

SRP-9005

Solution for single IV infusion

Intervention Type: BIOLOGICAL

Corticosteroid

Oral tablet (prophylactic)

Intervention Type: DRUG

Other Intervention Names

rAAVrh74.MHCK7.hSGCG; Prednisone or equivalent

Eligibility Criteria

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

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

• Minimum Eligible Age: 4 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Sarepta Therapeutics, Inc.

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Medical Director

Role: STUDY_DIRECTOR

Sarepta Therapeutics, Inc.

Locations

Children's Hospital of the King's Daughters

Norfolk, Virginia, United States

Countries

United States

Other Identifiers

SRP-9005-101

Identifier Type: -

Identifier Source: org_study_id