WITHDRAWN

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

Conditions

Muscular Dystrophies, Limb-Girdle SRP-9005 Type 2C/R5 LGMD2C/R5 Pediatric Non-ambulatory Ambulatory Phase 1 Phase 3

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Study Overview

This clinical trial focuses on testing the efficacy of different digital interventions to promote re-engagement in cancer-related long-term follow-up care for adolescent and young adult (AYA) survivors of childhood cancer.

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.

Official Title

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)

Quick Facts

Study Start:2025-06-30

Study Completion:2032-03-30

Study Type:Not specified

Phase:Not Applicable

Enrollment:Not specified

Status:WITHDRAWN

Study ID

NCT06952686

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.

Ages Eligible for Study:4 Years

Sexes Eligible for Study:ALL

Accepts Healthy Volunteers:No

Standard Ages:CHILD, ADULT, OLDER_ADULT

Inclusion Criteria	Exclusion 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

Inclusion Criteria

Exclusion 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

Contacts and Locations

Principal Investigator

Medical Director

STUDY_DIRECTOR

Sarepta Therapeutics, Inc.

Study Locations (Sites)

Children's Hospital of the King's Daughters

Norfolk, Virginia, 23510

United States

Collaborators and Investigators

Sponsor: Sarepta Therapeutics, Inc.

Medical Director, STUDY_DIRECTOR, Sarepta Therapeutics, Inc.

Study Record Dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

Study Start Date2025-06-30

Study Completion Date2032-03-30

Study Record Updates

Study Start Date2025-06-30

Study Completion Date2032-03-30

Terms related to this study

Keywords Provided by Researchers

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

Additional Relevant MeSH Terms

Muscular Dystrophies, Limb-Girdle