A Trial to Learn More About an Experimental Gene Therapy Called Bidridistrogene Xeboparvovec (SRP-9003) as a Possible Treatment for Limb Girdle Muscular Dystrophy 2E/R4 (EMERGENE)

February 27, 2026 updated by: Sarepta Therapeutics, Inc.

A Phase 3 Multinational, Open-label, Systemic Gene Delivery Study to Evaluate the Safety and Efficacy of SRP-9003 in Subjects With Limb Girdle Muscular Dystrophy 2E/R4

This is a multicenter, global study of the effects of a single systemic dose of SRP-9003 on beta-sarcoglycan (β-SG) gene expression in participants with limb-girdle muscular dystrophy, type 2E/R4 (LGMD2E/R4). This study will consist of both ambulatory participants (Cohort 1) and non-ambulatory participants (Cohort 2).

Study Overview

Status

Active, not recruiting

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Actual)

17

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

  • Belgium
      • Ghent, Belgium, 9000
        • NMRC Gent (UZ Gent)
    • Vlaams Brabant
      • Leuven, Vlaams Brabant, Belgium, 3000
        • University Hospital Leuven (UZ Leuven)
  • Germany
    • North Rhine-Westphalia
      • Essen, North Rhine-Westphalia, Germany, 45147
        • Universitatsklinikum Essen; Kinderklinik I, Sozialpadiatrisches Zentrum
  • Italy
      • Milan, Italy, 20122
        • Fondazione IRCCS Ca Granda Ospedale Maggiore Policlinico
  • Spain
      • Barcelona, Spain, 8950
        • Hospital Sant Joan de Déu
  • United Kingdom
      • Newcastle upon Tyne, United Kingdom, NE1 3BZ
        • Newcastle University
  • United States
    • California
      • La Jolla, California, United States, 92037
        • University of California, San Diego-Altman Clinical and Translational Research Institute
    • Ohio
      • Columbus, Ohio, United States, 43205
        • Nationwide Childrens Hospital
    • Pennsylvania
      • Philadelphia, Pennsylvania, United States, 19104
        • The Children's Hospital of Philadelphia
    • Virginia
      • Norfolk, Virginia, United States, 23507
        • Children's Hospital of The King's Daughter

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

Inclusion Criteria:

  • Cohort 1, only ambulatory participants:

    • Able to walk without assistive aid
    • 10MWR <30 seconds
    • NSAD ≥25

  • Cohort 2, only non-ambulatory participants:

    • 10MWR ≥30 seconds or unable to perform
    • PUL 2.0 entry scale score ≥3
  • Participants must possess 1 homozygous or 2 heterozygous pathogenic and/or likely pathogenic β-SG DNA gene mutations
  • Able to cooperate with muscle testing
  • Participants must have adeno-associated virus serotype rh74 (AAVrh74) antibody titers <1:400 (that is, not elevated) as determined by AAVrh74 antibody enzyme-linked immunosorbent assay.

Exclusion Criteria:

  • Left ventricular ejection fraction < 40% or clinical signs and/or symptoms of cardiomyopathy
  • Forced vital capacity ≤40% of predicted value and/or requirement for nocturnal ventilation
  • Diagnosis of (or ongoing treatment for) an autoimmune disease and on active immunosuppressant treatment
  • Presence of any other clinically significant illness or medical condition (other than LGMD2E/R4)

Other inclusion/exclusion criteria 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-9003
Participants will receive a single intravenous (IV) infusion of SRP-9003.
Biological: SRP-9003
Solution for single IV infusion
Other Names:
  • scAAVrh74.MHCK7.hSGCB, bidridistrogene xeboparvovec
Drug: Glucocorticoid
Oral tablet (prophylactic)
Other Names:
  • Prednisone or equivalent

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
Cohort 1: Change from Baseline in β-SG Expression at Day 60 Post-dose as Measured by Immunofluorescence (IF) Percent β-SG Positive Fibers
Time Frame: Baseline, Day 60
Baseline, Day 60

Secondary Outcome Measures

Outcome Measure
Time Frame
Cohort 1: Change From Baseline Through Month 60 in the Time to Rise from the Floor Test
Time Frame: Baseline through Month 60
Baseline through Month 60
Cohort 1: Change From Baseline Through Month 60 in the Time to Complete the 10-meter Walk/Run (10MWR) Test
Time Frame: Baseline through Month 60
Baseline through Month 60
Cohort 1: Change From Baseline Through Month 60 in the Time to Ascend 4 Steps Test
Time Frame: Baseline through Month 60
Baseline through Month 60
Cohort 1: Change From Baseline Through Month 60 in the Time to Complete the 100-meter Walk/Run (100MWR) Test
Time Frame: Baseline through Month 60
Baseline through Month 60
Cohort 1: Change From Baseline Through Month 60 in the Timed Up and Go Test
Time Frame: Baseline through Month 60
Baseline through Month 60
Change From Baseline Through Month 60 in Creatine Kinase Level
Time Frame: Baseline through Month 60
Baseline through Month 60
Time to Change of Loss of Ambulation
Time Frame: Baseline through Month 60
Baseline through Month 60
Cohort 1 and Cohort 2: Change From Baseline in β-SG Expression at Day 60 Post-dose as Measured by IF Percent Fluorescent Intensity (PFI)
Time Frame: Baseline, Day 60
Baseline, Day 60
Cohort 2: Change From Baseline in β-SG Expression at Day 60 Post-dose as Measured by IF Percent β-SG Positive Fibers (PβSGPF)
Time Frame: Baseline, Day 60
Baseline, Day 60
Cohort 1 and Cohort 2: Change From Baseline Through Month 60 in Performance of Upper Limb Version 2.0 (PUL 2.0) Total Score
Time Frame: Baseline through Month 60
Baseline through Month 60
Cohort 1 and Cohort 2: Change From Baseline in β-SG Expression at Day 60 Post-dose as Measured by Western Assay
Time Frame: Baseline, Day 60
Baseline, Day 60
Cohort 1 and Cohort 2: Change From Baseline Through Month 60 in North Star Assessment for Limb-girdle Muscular Dystrophies (NSAD) Total Score
Time Frame: Baseline through Month 60
Baseline through Month 60
Number of Participants Experiencing Treatment-emergent Adverse Events (TEAEs), Treatment-emergent Serious Adverse Events (SAEs) and Adverse Events of Special Interests (AESIs)
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 (Actual)

January 15, 2024

Primary Completion (Actual)

March 4, 2025

Study Completion (Estimated)

November 30, 2029

Study Registration Dates

First Submitted

January 30, 2024

First Submitted That Met QC Criteria

January 30, 2024

First Posted (Actual)

February 7, 2024

Study Record Updates

Last Update Posted (Actual)

March 2, 2026

Last Update Submitted That Met QC Criteria

February 27, 2026

Last Verified

February 1, 2026

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • SRP-9003-301
  • 2022-503112-17-00 (Other Identifier: Clinical Trials Information System (CTIS))

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.

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