Condition: Myotonic Dystrophy 1 · Sponsor: ARTHEx Biotech S.L.
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.
This description comes directly from the study's public registry record.
Project Manager · +34676229821 · clinical@arthexbiotech.com
Always discuss trial participation with your own doctor first.
| UCLA | Los Angeles, California, United States | Recruiting |
| University of Florida | Gainesville, Florida, United States | Recruiting |
| University of Iowa Health Care - Department of Neurology | Iowa City, Iowa, United States | Recruiting |
| University of Kansas Medical Center, Department of Neurology | Fairway, Kansas, United States | Recruiting |
| Virginia Commonwealth University | Richmond, Virginia, United States | Recruiting |
| Centre Intégré Universitaire de Santé et Services Sociaux du Saguenay-Lac-St-Jean | Chicoutimi, Quebec, Canada | Recruiting |
| Institute of Myology | Paris, France | Recruiting |
| The NeMO Clinical Center in Milan, Neurorehabilitation Unit, University of Milan | Milan, Italy | Recruiting |
| Fondazione Policlinico A. Gemelli- IRCCS | Rome, Italy | Recruiting |
| Radboudumc | Nijmegen, Netherlands | Recruiting |
| Hospital Universitario Donostia | Donostia / San Sebastian, Spain | Recruiting |
| St. George's University Hospital | London, United Kingdom | Recruiting |
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Source record: clinicaltrials.gov/study/NCT06300307