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

Status

Indicates the current recruitment status or the expanded access status.
Active, not recruiting

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

Study Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Interventional

Enrollment (Actual)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
17

Phase

Phase

The stage of a clinical trial studying a drug or biological product, based on definitions developed by the U.S. Food and Drug Administration (FDA). The phase is based on the study's objective, the number of participants, and other characteristics. There are five phases: Early Phase 1 (formerly listed as Phase 0), Phase 1, Phase 2, Phase 3, and Phase 4. Not Applicable is used to describe trials without FDA-defined phases, including trials of devices or behavioral interventions.
  • 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 Contact

Study Contact

The name and contact information for the person who can answer enrollment questions for a clinical study. Each location where the study is being conducted may also have a specific contact, who may be better able to answer those questions.
  • Name: Sarepta Therapeutics Inc. For Clinical Trial Information, Select Option 4
  • Phone Number: 1-888-SAREPTA (1-888-727-3782)
  • Email: SareptAlly@sarepta.com

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 Cà 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

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

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

Participant Group / Arm

A group or subgroup of participants in a clinical trial that receives a specific intervention/treatment, or no intervention, according to the trial's protocol.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
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

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
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

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
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

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
Sarepta Therapeutics, Inc.

Investigators

Investigators

A researcher involved in a clinical study. Related terms include site principal investigator, site sub-investigator, study chair, study director, and study principal investigator.
  • 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)

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
January 15, 2024

Primary Completion (Actual)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
March 4, 2025

Study Completion (Estimated)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
November 30, 2029

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
January 30, 2024

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
January 30, 2024

First Posted (Actual)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
February 7, 2024

Study Record Updates

Last Update Posted (Actual)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
March 2, 2026

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
February 27, 2026

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
February 1, 2026

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant 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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