Myotonic Dystrophy
Myotonic dystrophy is a genetic disorder that affects muscle function, causing prolonged muscle contractions, weakness, and other systemic issues. It can also lead to complications such as heart problems and endocrine issues.
Myotonic Dystrophy Type 1: ATX-01 Treatment Study
We are evaluating the safety and effects of ATX-01 in adults with myotonic dystrophy type 1. The study aims to see how it impacts muscle function and daily living activities.
6-12 monthsSafety phase (I)Post-Trial Drug AccessInvestigational MedicinesInjectionInternal MedicineNeurology
What diseases are being studied?
Health conditions and diseases that the clinical trial is designed to study and treat.
Who can participate in the clinical trial?
These questions help us understand your situation so we can let the trial team know whether you might be a potential match. This is not a medical evaluation and is not part of the official screening, the study doctor will make the final decision..
- Are you between 18 and 64 years old?
- Do you have a diagnosis of myotonic dystrophy type 1 (DM1)?
- Are you currently taking any medications for myotonia, like mexiletine, within the last 5 half-lives?
- Do you have a body mass index (BMI) of less than 35?
- Have you had any major heart problems, like a heart attack or heart failure, in the past?
What experimental substances are being tested?
Experimental substances that are being investigated in this clinical trial. These are not yet approved for general use.
Atx-01
16-Base Single Stranded Rna Targeting Mir-23b Linked To Oleic Acid
Where is the clinical trial being conducted?
Locations and medical facilities where this clinical trial is currently recruiting or conducting research. Select the locations where you would like to participate.
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Hopital Universitaire Pitie Salpetriere
Neuromyology
Salpêtrière, France
Centro Clinico Nemo
Centro clinico NeMO di Milano
Bresso, Italy
Fondazione Policlinico Universitario Agostino Gemelli IRCCS
Women's, child and public health sciences
Rome, Italy
Sponsor: Arthex Biotech S.L.
Last updated: Nov 26, 2025
Disclaimer: Parts of this content have been automatically extracted from the EU Clinical Trials registry. While we strive for accuracy, please always contact the trial site or sponsor directly for correct and official information before making any decisions about participation. View on EU Clinical Trials.