New drug ATX-01 targets muscle stiffness in rare disease
NCT ID NCT06300307
First seen Feb 01, 2026 · Last updated May 14, 2026 · Updated 12 times
Summary
This early-stage trial tests a new drug, ATX-01, in 56 adults aged 18 to 64 with myotonic dystrophy type 1 (DM1), a condition causing muscle stiffness and weakness. The main goal is to see if ATX-01 is safe and tolerable compared to a placebo. Participants will receive either one or three doses of the drug or placebo by IV, and researchers will monitor side effects and drug levels in the body.
Disclaimer
Read more
Show less
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.
Get updates
Get notified about this study
Sign up to get updates when this study changes or when new studies for MYOTONIC DYSTROPHY 1 are added.
By submitting, you agree to our Terms of use
Contacts and locations
Show contact details
Enter your email to view the contact information for this study.
By submitting, you agree to our Terms of use
Study contacts
-
Contact
Phone: •••-•••-•••• Email: •••••@•••••
Locations
-
Centre Intégré Universitaire de Santé et Services Sociaux du Saguenay-Lac-St-Jean
RECRUITINGChicoutimi, Quebec, G7H 5H6, Canada
Contact Phone: •••-•••-•••• Email: •••••@•••••
-
Fondazione Policlinico A. Gemelli- IRCCS
RECRUITINGRome, 00168, Italy
Contact Email: •••••@•••••
-
Hospital Universitario Donostia
RECRUITINGDonostia / San Sebastian, 20014, Spain
Contact
Contact Email: •••••@•••••
-
Institute of Myology
RECRUITINGParis, 75651, France
Contact Email: •••••@•••••
-
Radboudumc
RECRUITINGNijmegen, 6525, Netherlands
Contact
Contact Email: •••••@•••••
-
St. George's University Hospital
RECRUITINGLondon, SW17 0RE, United Kingdom
Contact
Contact Email: •••••@•••••
-
The NeMO Clinical Center in Milan, Neurorehabilitation Unit, University of Milan
RECRUITINGMilan, 20162, Italy
Contact Email: •••••@•••••
-
UCLA
RECRUITINGLos Angeles, California, 90095, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
Contact
-
University of Florida
RECRUITINGGainesville, Florida, 32610, United States
Contact Email: •••••@•••••
-
University of Iowa Health Care - Department of Neurology
RECRUITINGIowa City, Iowa, 52242, United States
Contact
Contact Phone: •••-•••-•••• Email: •••••@•••••
-
University of Kansas Medical Center, Department of Neurology
RECRUITINGFairway, Kansas, 66205, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
-
Virginia Commonwealth University
RECRUITINGRichmond, Virginia, 23298, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
Contact
Conditions
Explore the condition pages connected to this study.