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Experimental gene therapy targets rare muscle disease

NCT ID NCT05224505

Disease control Sponsor: Atamyo Therapeutics 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 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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 gene therapy called ATA-100 for people with LGMDR9, a rare genetic muscle disease that causes progressive weakness. Six adults receive a single intravenous infusion of the therapy, which delivers a working copy of the FKRP gene. The main goal is to check safety and tolerability over five years, with secondary measures looking at muscle function and breathing.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance
ATA-100 (a gene therapy that delivers a working FKRP gene via a harmless virus)
What this could lead to
If successful, this could point toward a treatment that slows or stops muscle weakening in people with LGMDR9.
What could go wrong
This is a very early, small Phase 1 trial with only 6 participants, so safety and dosing are still being tested. It may not lead to a working treatment, and gene therapies can sometimes cause immune reactions.

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

6 people

The number who actually took part.

Started

Sep 2022

Expected to finish

Sep 2029

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

16 to 99 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: * 1\. Female and male ambulant patients * 2\. Patients ≥ 16 years old * 3\. Documented LGMDR9 diagnosis based on clinical presentation and genotyping confirming the FKRP gene mutations * 4\. Moderate diaphragmatic muscle impairment Exclusion Criteria: * 1\. Detectable serum neutralizing antibodies against AAV9 * 2\. Cardiomyopathy

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Conditions

The condition(s) this trial relates to.

autosomal recessive limb-girdle muscular dystrophy type 2I

As listed by the trial registrant

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

LGMDR9

Contacts and locations

Locations

  • Institute of Myology Pitié-Salpêtrière Hospital 47 Bd de l'Hôpital

    Paris, 75013, France

  • Rigshospitalet, University of Copenhagen Blegdamsvej 9

    Copenhagen, 2100, Denmark

  • Royal Victoria Infirmary Queen Victoria Road Level 6 Leazes Wing

    Newcastle upon Tyne, NE1 4LP, United Kingdom