Gene Delivery Clinical Trial of SRP-9003 (Bidridistrogene Xeboparvovec) for Participants With Limb-Girdle Muscular Dystrophy, Type 2E (LGMD2E) (Beta-Sarcoglycan Deficiency)

January 12, 2026 updated by: Sarepta Therapeutics, Inc.

A Single-Center, Open-Label, Systemic Gene Delivery Study to Evaluate the Safety, Tolerability, and Efficacy of SRP-9003 Administered by Systemic Infusion in Subjects With LGMD2E (β-Sarcoglycan Deficiency)

The proposed clinical trial is the first-in-human, single-center, open-label, gene delivery study of SRP-9003 (bidridistrogene xeboparvovec) in participants with LGMD2E.

Study Overview

Status

Terminated

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Actual)

6

Phase

  • Phase 2
  • Phase 1

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
    • Ohio
      • Columbus, Ohio, United States, 43205
        • Nationwide Children's Hospital

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

4 years to 15 years (Child)

Accepts Healthy Volunteers

No

Description

INCLUSION CRITERIA

  • Males or females of any ethnic group
  • β-SG deoxyribonucleic acid (DNA) gene mutations at both alleles
  • Weakness demonstrated based on history of difficulty in running, jumping and climbing stairs
  • A 100 meter walk/run (MWR) test result: ≥40 % of predicted for age-, height-, gender-, and weight-matched healthy controls at the screening visit

EXCLUSION CRITERIA

  • Active viral infection based on clinical observations
  • Cardiac magnetic resonance imaging (MRI) determined left ventricular ejection fraction (LVEF) <40%
  • Serological evidence of human immunodeficiency virus (HIV), hepatitis B, or hepatitis C infection
  • Diagnosis of (or ongoing treatment for) an autoimmune disease
  • Abnormal laboratory values considered clinically significant
  • Concomitant illness or requirement for chronic drug treatment that, in the opinion of the Principal Investigator, creates unnecessary risks for gene transfer.

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: Non-Randomized
  • Interventional Model: Sequential Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Cohort 1: SRP-9003
Participants will receive a single intravenous (IV) infusion of SRP-9003 at a prespecified dose.
Genetic: SRP-9003
SRP-9003 will be administered through a single systemic injection.
Other Names:
  • LGMD2E vector
  • bidridistrogene xeboparvovec
Experimental: Cohort 2: SRP-9003
Participants will receive a single IV infusion of SRP-9003. Dose will be determined based on the findings from Cohort 1.
Genetic: SRP-9003
SRP-9003 will be administered through a single systemic injection.
Other Names:
  • LGMD2E vector
  • bidridistrogene xeboparvovec

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
Number of Treatment-Emergent Adverse Events (AEs) and Treatment-Emergent Serious Adverse Events (SAEs)
Time Frame: Baseline up to 7 years
Baseline up to 7 years

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Change From Baseline in Quantity of Beta-Sarcoglycan (β-SG) Protein Expression at Day 60, as Measured by Western Blot
Time Frame: Baseline, Day 60
β-SG gene expression levels will be quantified by Western Blot and compared between pre and post muscle biopsies.
Baseline, Day 60
Change From Baseline in Quantity of β-SG Protein Expression at Day 60, as Measured by Immunofluorescence
Time Frame: Baseline, Day 60
β-SG gene expression levels will be quantified by immunofluorescence and compared between pre and post muscle biopsies.
Baseline, Day 60
Change From Baseline in Quantity of β-SG Protein Expression at Day 60, as Measured by Immunohistochemistry Percent B-SG Positive Fibers
Time Frame: Baseline, Day 60
Baseline, Day 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.

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

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)

October 27, 2018

Primary Completion (Actual)

January 14, 2025

Study Completion (Actual)

January 14, 2025

Study Registration Dates

First Submitted

August 28, 2018

First Submitted That Met QC Criteria

August 28, 2018

First Posted (Actual)

August 29, 2018

Study Record Updates

Last Update Posted (Actual)

January 20, 2026

Last Update Submitted That Met QC Criteria

January 12, 2026

Last Verified

January 1, 2026

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • SRP-9003-101
  • IRB17-00253 (Other Identifier: Sarepta Therapeutics)

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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