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Gene therapy hope for kids with rare muscle-wasting disease

NCT ID NCT05973630

Disease control Sponsor: Atamyo Therapeutics Source: ClinicalTrials.gov ↗

What the study statuses mean

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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 This study
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
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This status has not been confirmed recently, so it may be out of date.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 early-stage trial tests a single intravenous dose of a gene therapy (ATA-200) in 4 children aged 6-12 with limb-girdle muscular dystrophy type 2C/R5 (LGMD2C), a rare genetic muscle-weakening condition. The goal is to see if the treatment is safe and tolerable by delivering a working copy of the missing gene. Participants must be able to walk and have no antibodies against the virus used to deliver the gene.

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

The first testing in people. Mainly checks safety and dose, usually in a small group.

Participants

4 people

The number who actually took part.

Started

Feb 2025

Expected to finish

Jan 2031

An estimate. End dates often move.

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

6 to 13 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: * Confirmed diagnosis of LGMDR5 before age of 10, based on clinical presentation and genotyping * Ambulant male or female patients aged 6 to less than 12 years of age at screening * Able to perform the 10-meter walk test (10MWT) in less than 15 sec and to rise from chair with or without arm support Exclusion Criteria: * Detectable neutralizing antibodies against AAV8 * Cardiomyopathy with left ventricular ejection fraction (LVEF) \< 50% * Respiratory assistance * Concomitant medical condition that might interfere with LGMDR5 evolution * Acute illness within 4 weeks of anticipated IMP administration * Current participation in another clinical trial with investigational medicinal product * Previous participation in gene and cell therapy trials * Any condition that would contraindicate immunosuppressant treatment * Presence of any permanent items (e.g., metal braces) precluding undergoing MRI * Any vaccination 1 month prior to planned IMP administration * Serology consistent with HIV exposure or active hepatitis B or C infection * Grade 2 or higher lab abnormalities for liver function tests, creatinine, hemogram and coagulation

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Conditions

The condition(s) this trial relates to.

autosomal recessive limb-girdle muscular dystrophy type 2C

As listed by the trial registrant

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

LGMD2C

Contacts and locations

Locations

  • Child Health Research Institute

    Gainsville, Florida, 32610, United States

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