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

Description

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 Overview

Study Details

Study overview

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.

A Seamless Phase 1/3, Multicenter, Single Dose Systemic Gene Transfer Study to Evaluate the Safety, Tolerability, and Efficacy of SRP-9005 in Limb Girdle Muscular Dystrophy Type 2C/R5 Subjects (COMPASS)

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

Condition
Muscular Dystrophies, Limb-Girdle
Intervention / Treatment

-

Contacts and Locations

Norfolk

Children's Hospital of the King's Daughters, Norfolk, Virginia, United States, 23510

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

For general information about clinical research, read Learn About Studies.

Eligibility 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
  • * 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

Ages Eligible for Study

4 Years to

Sexes Eligible for Study

ALL

Accepts Healthy Volunteers

No

Collaborators and Investigators

Sarepta Therapeutics, Inc.,

Medical Director, STUDY_DIRECTOR, Sarepta Therapeutics, Inc.

Study Record Dates

2032-03-30