Actively Recruiting
A Phase 1/2a Study to Assess Safety, Tolerability, and Effects of IV ATX-01 in Adults 18-64 With Classic Myotonic Dystrophy Type 1
Led by ARTHEx Biotech S.L. · Updated on 2026-02-10
56
Participants Needed
12
Research Sites
N/A
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are evaluating the safety and tolerability of ATX-01 in adults aged 18 to 64 with classic myotonic dystrophy type 1 (DM1). This phase 1/2a trial aims to compare ATX-01, a new synthetic single-stranded oligonucleotide that inhibits microRNA miR-23b, with a placebo. The study focuses on whether ATX-01 is safe, well tolerated, and its effects on patients with DM1. The trial includes two parts: a single-ascending dose phase where participants receive one dose of ATX-01 or placebo, and a multiple-ascending dose phase where participants receive three doses. Both treatments are given intravenously. The study groups consist of those receiving ATX-01 and those receiving a matching placebo. Participants will be monitored for up to 120 days to assess adverse events, laboratory tests, heart activity via ECG, vital signs, and any changes in suicidal thoughts or behavior. Other measurements include blood levels of ATX-01, hand opening time, ankle strength, and daily living activity impacts. Safety and tolerability are carefully tracked throughout the study period under a randomized, triple-blind design.
CONDITIONS
Brief Title
Study of ATX-01 in Participants With DM1
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Adults aged 18 to 64 with a confirmed clinical diagnosis of myotonic dystrophy type 1 (more than 150 CTG repeats in DMPK gene)
- Able to walk or run 10 meters at screening without assistive devices except ankle-foot orthoses
- Presence of grip muscle stiffness (myotonia) lasting more than 3 seconds confirmed by a central reader
You will not qualify if you...
- Having congenital myotonic dystrophy
- Muscle weakness score below 4 on ankle dorsiflexion or significant tibialis anterior muscle wasting preventing biopsy
- Use of mexiletine or other myotonia treatments within 21 days or 5 half-lives before screening
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - Up to 120 days
Participants receive intravenous ATX-01 or placebo and are monitored for safety, tolerability, and effects on muscle function.
Multiple visits during treatment period
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
How is the study designed?
Study Type
INTERVENTIONAL
Masking
TRIPLE
Allocation
RANDOMIZED
Model
PARALLEL
Primary Purpose
TREATMENT
Number of Arms
2
Similar Trials
Frequently Asked Questions
Have more questions? Get in touch with our team for quick support
Not the Right Trial for You?
Explore thousands of other clinical trials that might be a better match.
Sign up to get personalized trial recommendations delivered to your inbox.
Already have an account? Log in here