Study of ATX-01 in Participants With DM1
Recruiting now · Phase 1/Phase 2 · Has a placebo group
Conditions studied: Myotonic Dystrophy 1
In brief
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.
Key facts
- Study ID
- NCT06300307
- Run by
- ARTHEx Biotech S.L.
- People needed
- 56
- Starts
- 2024-10-15
- Expected to finish
- 2027-07-01
- Last updated by the study team
- 2026-02-10
Who can join
Age: 18 and older, up to 64. Sex: any. Healthy volunteers: not accepted.
Where it is running
- UCLA — Los Angeles, California, United States (enrolling)
- University of Florida — Gainesville, Florida, United States (enrolling)
- University of Iowa Health Care - Department of Neurology — Iowa City, Iowa, United States (enrolling)
- University of Kansas Medical Center, Department of Neurology — Fairway, Kansas, United States (enrolling)
- Virginia Commonwealth University — Richmond, Virginia, United States (enrolling)
- Centre Intégré Universitaire de Santé et Services Sociaux du Saguenay-Lac-St-Jean — Chicoutimi, Quebec, Canada (enrolling)
- Institute of Myology — Paris, France (enrolling)
- The NeMO Clinical Center in Milan, Neurorehabilitation Unit, University of Milan — Milan, Italy (enrolling)
- Fondazione Policlinico A. Gemelli- IRCCS — Rome, Italy (enrolling)
- Radboudumc — Nijmegen, Netherlands (enrolling)
- Hospital Universitario Donostia — Donostia / San Sebastian, Spain (enrolling)
- St. George's University Hospital — London, United Kingdom (enrolling)
Full record on ClinicalTrials.gov
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