Study of ATX-01 in Participants With DM1

NCT06300307 · tracked via the Priya Life Science UK tracker

Sponsor

ARTHEx Biotech S.L.

Phase

Phase 1/2

Started

2024-10-15

Last updated

2026-02-10

Condition(s) studied

Myotonic Dystrophy 1

Investigational drug(s) / intervention(s)

ATX-01Placebo

ATX-01: Solution for infusion

Placebo: Solution for infusion

Study summary

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.

Eligibility

Sex

ALL

Min age

18 Years

Max age

64 Years

Healthy volunteers

Key Inclusion Criteria: * Participants with a documented clinical diagnosis of DM1 (CTG expansion of \>150 repeats in DMPK gene measured in peripheral blood mononuclear cells) * Ambulatory, defined as able to complete a 10-meter walk/run test at screening without the use of assistive devices such as canes, walkers, or orthoses, except for ankle-foot orthoses * Presence for \>3 seconds of grip myotonia as confirmed by a central reader Key Exclusion Criteria: * Participants with congenital DM1 * Medical Research Council Muscle Scale score of less than 4 on ankle dorsiflexion or significant tibialis anterior atrophy that prevents a muscle biopsy * Use of mexiletine or other agent for myotonia within 21 days or 5 half-lives, whichever is longer, prior to screening

Primary outcome measure(s)

Incidence of adverse events — Up to 120 days
To evaluate the safety and tolerability of ATX-01 in adult participants with DM1

Trial sites (12)

Facility	City	Region	Status
UCLA	Los Angeles	California	Recruiting
University of Florida	Gainesville	Florida	Recruiting
University of Iowa Health Care - Department of Neurology	Iowa City	Iowa	Recruiting
University of Kansas Medical Center, Department of Neurology	Fairway	Kansas	Recruiting
Virginia Commonwealth University	Richmond	Virginia	Recruiting
Centre Intégré Universitaire de Santé et Services Sociaux du Saguenay-Lac-St-Jean	Chicoutimi	Quebec	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

Other trials for the same condition

Study of SRP-1003 in Participants With Type 1 Myotonic Dystrophy Recruiting

A Clinical Study of PGN-EDODM1 in People With Myotonic Dystrophy Type 1 Recruiting

Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT06300307 on ClinicalTrials.gov ↗ ← All trials in the UK