Actively Recruiting
Study of ATX-01 in Participants With DM1
Led by ARTHEx Biotech S.L. · Updated on 2026-02-10
56
Participants Needed
12
Research Sites
141 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
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.
CONDITIONS
Official Title
Study of ATX-01 in Participants With DM1
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Participants with a documented clinical diagnosis of DM1 (CTG expansion of >150 repeats in DMPK gene measured in peripheral blood mononuclear cells)
- Able to walk or run 10 meters at screening without assistive devices except ankle-foot orthoses
- Presence of grip myotonia lasting more than 3 seconds as confirmed by a central reader
You will not qualify if you...
- Participants with congenital DM1
- Medical Research Council Muscle Scale score less than 4 on ankle dorsiflexion or significant tibialis anterior atrophy preventing muscle biopsy
- Use of mexiletine or other agent for myotonia within 21 days or 5 half-lives, whichever is longer, prior to screening
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 12 locations
1
UCLA
Los Angeles, California, United States, 90095
Actively Recruiting
2
University of Florida
Gainesville, Florida, United States, 32610
Actively Recruiting
3
University of Iowa Health Care - Department of Neurology
Iowa City, Iowa, United States, 52242
Actively Recruiting
4
University of Kansas Medical Center, Department of Neurology
Fairway, Kansas, United States, 66205
Actively Recruiting
5
Virginia Commonwealth University
Richmond, Virginia, United States, 23298
Actively Recruiting
6
Centre Intégré Universitaire de Santé et Services Sociaux du Saguenay-Lac-St-Jean
Chicoutimi, Quebec, Canada, G7H 5H6
Actively Recruiting
7
Institute of Myology
Paris, France, 75651
Actively Recruiting
8
The NeMO Clinical Center in Milan, Neurorehabilitation Unit, University of Milan
Milan, Italy, 20162
Actively Recruiting
9
Fondazione Policlinico A. Gemelli- IRCCS
Rome, Italy, 00168
Actively Recruiting
10
Radboudumc
Nijmegen, Netherlands, 6525
Actively Recruiting
11
Hospital Universitario Donostia
Donostia / San Sebastian, Spain, 20014
Actively Recruiting
12
St. George's University Hospital
London, United Kingdom, SW17 0RE
Actively Recruiting
Research Team
P
Project Manager
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
TRIPLE
Allocation
RANDOMIZED
Model
PARALLEL
Primary Purpose
TREATMENT
Number of Arms
2
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