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Experimental gene therapy for rare muscle disease shows early promise but study halted

NCT ID NCT03652259

Disease control Sponsor: Sarepta Therapeutics, Inc. Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early This study
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This study tested a gene therapy called SRP-9003 for people with limb-girdle muscular dystrophy type 2E (LGMD2E), a rare genetic disease that causes muscle weakness. The treatment aimed to deliver a working gene to muscle cells to help them produce a missing protein. Only 6 people took part before the study was stopped early. The main goal was to check safety and see if the therapy could increase the protein levels in muscles.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 1/2

Runs two stages together: safety and dose first, then whether the treatment works.

Participants

6 people

The number who actually took part.

Started

Oct 2018

Finished

Jan 2025

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

4 to 15 years

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

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.

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Conditions

The condition(s) this trial relates to.

autosomal recessive limb-girdle muscular dystrophy type 2E limb-girdle muscular dystrophy Limb-girdle muscular dystrophy, type 2E

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Limb-girdle muscular dystrophy, type 2E

Contacts and locations

Locations

  • Nationwide Children's Hospital

    Columbus, Ohio, 43205, United States

More trials for these conditions

Other studies related to the condition(s) this trial covers.