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

August 1, 2025 updated by: Sarepta Therapeutics, Inc.

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)

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.

Study Overview

Status

Withdrawn

Conditions

Intervention / Treatment

Study Type

Interventional

Phase

  • Phase 3

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

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

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.

Eligibility Criteria

Ages Eligible for Study

  • Child
  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Description

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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: SRP-9005
Participants will receive a single intravenous (IV) infusion of SRP-9005.
Biological: SRP-9005
Solution for single IV infusion
Other Names:
  • rAAVrh74.MHCK7.hSGCG
Drug: Corticosteroid
Oral tablet (prophylactic)
Other Names:
  • Prednisone or equivalent

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
Part A : Number of Participants with Treatment-emergent Adverse Events (TEAEs)
Time Frame: Baseline through Month 60
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)
Time Frame: Baseline, Day 60
Baseline, Day 60

Secondary Outcome Measures

Outcome Measure
Time Frame
Part A and B: Change from Baseline Through Month 60 in North Star Assessment for Dysferlinopathy (NSAD) Total Score
Time Frame: Baseline through Month 60
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
Time Frame: Baseline through Month 60
Baseline through Month 60
Part A and B: Change from Baseline through Month 60 in Time to Rise From Floor
Time Frame: Baseline through Month 60
Baseline through Month 60
Part A and B: Change from Baseline Through Month 60 in Time to Ascend 4 Steps
Time Frame: Baseline through Month 60
Baseline through Month 60
Part A and B: Change from Baseline Through Month 60 in Time to Complete the 10-meter Walk/Run (10MWR)
Time Frame: Baseline through Month 60
Baseline through Month 60
Part A and B: Change from Baseline Through Month 60 in Time to Complete 100-meter Walk/Run (100MWR)
Time Frame: Baseline through Month 60
Baseline through Month 60
Part A and B: Change from Baseline Through Month 60 in Timed Up and Go
Time Frame: Baseline through Month 60
Baseline through Month 60
Part A and B: Change from Baseline in gamma-SG Expression at Day 60 in Ambulatory Participants Post-dose as Measured by IF Percent Fiber Intensity (PFI)
Time Frame: Baseline, Day 60
Baseline, Day 60
Part A and B: Change from Baseline in gamma-SG Expression at Day 60 in Ambulatory Participants Post-dose as Measured by Western Assay
Time Frame: Baseline, Day 60
Baseline, Day 60
Part A and B: Change from Baseline to Day 60 in Quantity of gamma-SG Protein Expression in Non-ambulatory Participants as Measured by IF PPF
Time Frame: Baseline to Day 60
Baseline to Day 60
Part A and B: Change from Baseline to Day 60 in Quantity of gamma-SG Protein Expression in Non-ambulatory Participants as Measured by IF PFI
Time Frame: Baseline to Day 60
Baseline to Day 60
Part A and B: Change from Baseline to Day 60 in Quantity of gamma-SG Protein Expression in Non-ambulatory Participants as Measured by Western Assay
Time Frame: Baseline to Day 60
Baseline to Day 60
Part A and B: Change from Baseline to Month 24 in Quantity of gamma-SG Protein Expression as Measured by IF PPF
Time Frame: Baseline to Month 24
Baseline to Month 24
Part A and B: Change from Baseline to Month 24 in Quantity of gamma-SG Protein Expression as Measured by IF PFI
Time Frame: Baseline to Month 24
Baseline to Month 24
Part A and B: Change from Baseline to Month 24 in Quantity of gamma-SG Protein Expression as Measured by Western Assay
Time Frame: Baseline to Month 24
Baseline to Month 24
Part A and B: Change from Baseline Through Month 60 in Creatine Kinase Levels
Time Frame: Baseline through Month 60
Baseline through Month 60
Part A and B: Loss of Ambulation
Time Frame: Baseline through Month 60
Baseline through Month 60
Part A and B: Change from Baseline Through Month 60 in Skeletal Muscle Magnetic Resonance Imaging (MRI)
Time Frame: Baseline through Month 60
Baseline through Month 60
Part A and B: Change from Baseline Through Month 60 in Wearable Device Stride Velocity 95th Percentile (SV95C) (Ambulatory Participants Only)
Time Frame: Baseline through Month 60
Baseline through Month 60
Part A and B: Change from Baseline Through Month 60 in Pulmonary Functions as Assessed by Forced Vital Capacity (FVC)
Time Frame: Baseline through Month 60
Baseline through Month 60
Part A and B: Change from Baseline Through Month 60 in Pulmonary Functions as Assessed by Forced Vital Capacity Percent Predicted (FVC%p)
Time Frame: Baseline through Month 60
Baseline through Month 60
Part A and B: Change from Baseline Through Month 60 in Pulmonary Functions as Assessed by Forced Expiratory Volume in 1 Second (FEV1)
Time Frame: Baseline through Month 60
Baseline through Month 60
Part A and B: Change from Baseline Through Month 60 in Pulmonary Functions as Assessed by Forced Expiratory Volume in 1 Second % Predicted (FEV1%p)
Time Frame: Baseline through Month 60
Baseline through Month 60
Part A and B: Change from Baseline Through Month 60 in Pulmonary Functions as Assessed by Peak Expiratory Flow Rate (PEFR)
Time Frame: Baseline through Month 60
Baseline through Month 60
Part A and B: Change from Baseline through Month 60 in Pulmonary Functions as Assessed by Maximal Inspiratory Pressure (MIP)
Time Frame: Baseline through Month 60
Baseline through Month 60
Part A and B: Change from Baseline Through Month 60 in Pulmonary Functions as Assessed by Percent Predicted Maximal Inspiratory Pressure (MIP%p)
Time Frame: Baseline through Month 60
Baseline through Month 60
Part A and B: Change from Baseline through Month 60 in Pulmonary Functions as Assessed by Maximal Expiratory Pressure (MEP)
Time Frame: Baseline through Month 60
Baseline through Month 60
Part A and B: Change from Baseline Through Month 60 in Pulmonary Functions as Assessed by Percent Predicted Maximal Expiratory Pressure (MEP%p)
Time Frame: Baseline through Month 60
Baseline through Month 60
Part B: Number of Participants with TEAEs
Time Frame: Baseline through Month 60
Baseline through Month 60

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sarepta Therapeutics, Inc.

Investigators

  • Study Director: Medical 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 Major Dates

Study Start (Estimated)

June 30, 2025

Primary Completion (Estimated)

November 30, 2030

Study Completion (Estimated)

March 30, 2032

Study Registration Dates

First Submitted

April 23, 2025

First Submitted That Met QC Criteria

April 23, 2025

First Posted (Actual)

May 1, 2025

Study Record Updates

Last Update Posted (Actual)

August 6, 2025

Last Update Submitted That Met QC Criteria

August 1, 2025

Last Verified

July 1, 2025

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • SRP-9005-101

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

Clinical Trials on Muscular Dystrophies, Limb-Girdle

Clinical Trials on SRP-9005

Search Similar Trials

Sponsors and Collaborators

Medical Conditions

Drug Interventions

CROs by country

CROs in South Korea

Conditions

Rare Diseases

Drug Interventions

Dietary Supplements

Sponsor/Collaborators

Locations

Subscribe