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

NCT06952686 | Withdrawn Phase 3 DMC FDA Drug

• 1 other identifier: SRP-9005-101
• Study Type: interventional
• Target: N/A
• Locations: 1 country
• Sites: 1

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

Keywords

SRP-9005; Type 2C/R5; LGMD2C/R5; Pediatric; Non-ambulatory; Ambulatory; Phase 1; Phase 3

Outcome Measures

Primary Outcomes (2)

• Part A : Number of Participants with Treatment-emergent Adverse Events (TEAEs)

  Baseline through Month 60

• Part A and B: Change from Baseline in gamma-Sarcoglycan (SG) Expression at Day 60 Post-dose in Ambulatory Participants as Measured by Immunofluorescence (IF) Percent Positive Fibers (PPF)

  Baseline, Day 60

Secondary Outcomes (29)

• Part A and B: Change from Baseline Through Month 60 in North Star Assessment for Dysferlinopathy (NSAD) Total Score

  Baseline through Month 60

• Part A and B: Change from Baseline Through Month 60 in Performance of the Upper Limb (PUL) Version 2.0 Total Score

  Baseline through Month 60

• Part A and B: Change from Baseline through Month 60 in Time to Rise From Floor

  Baseline through Month 60

• Part A and B: Change from Baseline Through Month 60 in Time to Ascend 4 Steps

  Baseline through Month 60

• Part A and B: Change from Baseline Through Month 60 in Time to Complete the 10-meter Walk/Run (10MWR)

  Baseline through Month 60

Study Arms (1)

SRP-9005

EXPERIMENTAL

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

Biological: SRP-9005; Drug: Corticosteroid

Interventions

SRP-9005 BIOLOGICAL

Solution for single IV infusion

Also known as: rAAVrh74.MHCK7.hSGCG

SRP-9005

Corticosteroid DRUG

Oral tablet (prophylactic)

Also known as: Prednisone or equivalent

SRP-9005

Eligibility Criteria

• Age: 4 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Sarepta Therapeutics, Inc.: lead

Study Sites (1)

Children's Hospital of the King's Daughters

Norfolk, Virginia, 23510, United States

Location

MeSH Terms

Conditions

Muscular Dystrophies, Limb-Girdle

Interventions

Adrenal Cortex Hormones; Prednisone

Condition Hierarchy (Ancestors)

Muscular Dystrophies; Muscular Disorders, Atrophic; Muscular Diseases; Musculoskeletal Diseases; Neuromuscular Diseases; Nervous System Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Intervention Hierarchy (Ancestors)

Hormones; Hormones, Hormone Substitutes, and Hormone Antagonists; Pregnadienediols; Pregnadienes; Pregnanes; Steroids; Fused-Ring Compounds; Polycyclic Compounds

Study Officials

• Medical Director

  Sarepta Therapeutics, Inc.

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: This study comprises 2 parts: Part A is the Phase 1 portion, Part B is the Phase 3 expansion portion.

Study Record Dates

• First Submitted: April 23, 2025
• First Posted: May 1, 2025
• Study Start: June 30, 2025
• Primary Completion (Estimated): November 30, 2030
• Study Completion (Estimated): March 30, 2032
• Last Updated: August 6, 2025

Record last verified: 2025-07

Locations

US (1)