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A Phase 1/2a Double-Blind, Placebo-controlled, Single- and Multiple Ascending Dose Study to Assess the Safety, Tolerability, PK, PD and Efficacy of IV Administration of ATX-01 In Male and Female Participants Aged 18 to 64 With Classic DM1
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.
Age
18 - 64 years
Sex
ALL
Healthy Volunteers
No
UCLA
Los Angeles, California, United States
University of Florida
Gainesville, Florida, United States
University of Iowa Health Care - Department of Neurology
Iowa City, Iowa, United States
University of Kansas Medical Center, Department of Neurology
Fairway, Kansas, United States
Virginia Commonwealth University
Richmond, Virginia, United States
Centre Intégré Universitaire de Santé et Services Sociaux du Saguenay-Lac-St-Jean
Chicoutimi, Quebec, Canada
Institute of Myology
Paris, France
The NeMO Clinical Center in Milan, Neurorehabilitation Unit, University of Milan
Milan, Italy
Fondazione Policlinico A. Gemelli- IRCCS
Rome, Italy
Radboudumc
Nijmegen, Netherlands
Start Date
October 15, 2024
Primary Completion Date
July 1, 2027
Completion Date
July 1, 2027
Last Updated
February 10, 2026
56
ESTIMATED participants
ATX-01
DRUG
Placebo
DRUG
Lead Sponsor
ARTHEx Biotech S.L.
Data Source & Attribution
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