New hope for muscle stiffness: experimental drug ATX-01 enters human trials
NCT ID NCT06300307
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This study tests a new drug called ATX-01 in 56 adults with myotonic dystrophy type 1, a condition that causes muscle stiffness and weakness. The drug aims to block a molecule that may contribute to the disease. The trial will first give a single dose, then multiple doses, to check safety and how well it works compared to a placebo.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
Active substance
ATX-01 (a synthetic molecule that blocks a specific microRNA)
What this could lead to
If successful, this could point toward a treatment that eases muscle symptoms in people with myotonic dystrophy type 1.
What could go wrong
This is an early-phase trial with only 56 participants, so results may not apply to everyone. The drug may not work or could cause side effects.
Disclaimer
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This is a summary of
the original study
.
Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
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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
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Study contacts
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
Locations
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Centre Intégré Universitaire de Santé et Services Sociaux du Saguenay-Lac-St-Jean
RECRUITINGChicoutimi, Quebec, G7H 5H6, Canada
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Fondazione Policlinico A. Gemelli- IRCCS
RECRUITINGRome, 00168, Italy
Contact Email: •••••@•••••
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Hospital Universitario Donostia
RECRUITINGDonostia / San Sebastian, 20014, Spain
Contact
Contact Email: •••••@•••••
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Institute of Myology
RECRUITINGParis, 75651, France
Contact Email: •••••@•••••
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Radboudumc
RECRUITINGNijmegen, 6525, Netherlands
Contact
Contact Email: •••••@•••••
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St. George's University Hospital
RECRUITINGLondon, SW17 0RE, United Kingdom
Contact
Contact Email: •••••@•••••
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The NeMO Clinical Center in Milan, Neurorehabilitation Unit, University of Milan
RECRUITINGMilan, 20162, Italy
Contact Email: •••••@•••••
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UCLA
RECRUITINGLos Angeles, California, 90095, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
Contact
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University of Florida
RECRUITINGGainesville, Florida, 32610, United States
Contact Email: •••••@•••••
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University of Iowa Health Care - Department of Neurology
RECRUITINGIowa City, Iowa, 52242, United States
Contact
Contact Phone: •••-•••-•••• Email: •••••@•••••
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University of Kansas Medical Center, Department of Neurology
RECRUITINGFairway, Kansas, 66205, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Virginia Commonwealth University
RECRUITINGRichmond, Virginia, 23298, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
Contact