Experimental gene therapy targets rare muscle disease
NCT ID NCT05224505
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.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
Locations
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Institute of Myology Pitié-Salpêtrière Hospital 47 Bd de l'Hôpital
Paris, 75013, France
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Rigshospitalet, University of Copenhagen Blegdamsvej 9
Copenhagen, 2100, Denmark
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Royal Victoria Infirmary Queen Victoria Road Level 6 Leazes Wing
Newcastle upon Tyne, NE1 4LP, United Kingdom