Study of ATX-01 in Participants With DM1
Start Date
10/15/2024
Completion Date
7/1/2027
Summary
The goal of this clinical trial is to test ATX-01 in participants with myotonic dystrophy type 1 (DM1). The main question it aims to answer is if ATX-01 is safe and well tolerated. The trial will compare the safety and tolerability of ATX-01 and a matching placebo. There will be a single-ascending dose part of the trial and a multiple-ascending dose part. In the single-ascending dose, participants will receive one dose of ATX-01 or placebo. In the multiple-ascending dose part, participants will receive three doses of ATX-01 or placebo. ATX-01 is a novel anti-miR (synthetic single stranded oligonucleotide) that inhibits a microRNA called miR-23b.
Eligibility Criteria
Age Range: 18 years to 64 years
Interventions
ATX-01
Placebo
Conditions
Locations
UCLA
Los Angeles, California 90095
United States
University of Florida
Gainesville, Florida 32610
United States
University of Iowa Health Care - Department of Neurology
Iowa City, Iowa 52242
United States
University of Kansas Medical Center, Department of Neurology
Fairway, Kansas 66205
United States
Virginia Commonwealth University
Richmond, Virginia 23298
United States
Centre Intégré Universitaire de Santé et Services Sociaux du Saguenay-Lac-St-Jean
Chicoutimi, Quebec G7H 5H6
Canada
Institute of Myology
Paris, 75651
France
The NeMO Clinical Center in Milan, Neurorehabilitation Unit, University of Milan
Milan, 20162
Italy
Fondazione Policlinico A. Gemelli- IRCCS
Rome, 00168
Italy
Radboudumc
Nijmegen, 6525
Netherlands
Hospital Universitario Donostia
Donostia / San Sebastian, 20014
Spain
St. George's University Hospital
London, SW17 0RE
United Kingdom