New hope for muscle stiffness: experimental drug ATX-01 enters human trials

NCT ID NCT06300307

Recruiting now Disease control Sponsor: ARTHEx Biotech S.L. Source: ClinicalTrials.gov ↗

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.

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.

myotonic dystrophy myotonic dystrophy type 1

As listed by the trial registrant

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

MYOTONIC DYSTROPHY 1

Contacts and locations

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Study contacts

Contact

Email: •••••@•••••

Locations

Centre Intégré Universitaire de Santé et Services Sociaux du Saguenay-Lac-St-Jean
RECRUITING

Chicoutimi, Quebec, G7H 5H6, Canada
Fondazione Policlinico A. Gemelli- IRCCS
RECRUITING

Rome, 00168, Italy
Hospital Universitario Donostia
RECRUITING

Donostia / San Sebastian, 20014, Spain
Institute of Myology
RECRUITING

Paris, 75651, France
Radboudumc
RECRUITING

Nijmegen, 6525, Netherlands
St. George's University Hospital
RECRUITING

London, SW17 0RE, United Kingdom
The NeMO Clinical Center in Milan, Neurorehabilitation Unit, University of Milan
RECRUITING

Milan, 20162, Italy
UCLA
RECRUITING

Los Angeles, California, 90095, United States
University of Florida
RECRUITING

Gainesville, Florida, 32610, United States
University of Iowa Health Care - Department of Neurology
RECRUITING

Iowa City, Iowa, 52242, United States
University of Kansas Medical Center, Department of Neurology
RECRUITING

Fairway, Kansas, 66205, United States
Virginia Commonwealth University
RECRUITING

Richmond, Virginia, 23298, United States

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