Study of ATX-01 in Participants with DM1

Last updated: February 26, 2025
Sponsor: ARTHEx Biotech S.L.
Overall Status: Active - Recruiting

Phase

1/2

Condition

Muscular Dystrophy

Treatment

Placebo

ATX-01

Clinical Study ID

NCT06300307
CT-ATX-01-DM1-1.1
  • Ages 18-64
  • All Genders

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 Criteria

Inclusion

Key Inclusion Criteria:

  • Participants with a documented clinical diagnosis of DM1 (CTG expansion of >150repeats in DMPK gene measured in peripheral blood mononuclear cells)

  • Ambulatory, defined as able to complete a 10-meter walk/run test at screeningwithout the use of assistive devices such as canes, walkers, or orthoses, except forankle-foot orthoses

  • Presence for >3 seconds of grip myotonia as confirmed by a central reader

Exclusion

Key Exclusion Criteria:

  • Participants with congenital DM1

  • Medical Research Council Muscle Scale score of less than 4 on ankle dorsiflexion orsignificant 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

Study Design

Total Participants: 56
Treatment Group(s): 2
Primary Treatment: Placebo
Phase: 1/2
Study Start date:
October 15, 2024
Estimated Completion Date:
June 30, 2026

Connect with a study center

  • Centre Intégré Universitaire de Santé et Services Sociaux du Saguenay-Lac-St-Jean

    Chicoutimi, Quebec G7H 5H6
    Canada

    Active - Recruiting

  • Institute of Myology

    Paris, 75651
    France

    Active - Recruiting

  • The NeMO Clinical Center in Milan, Neurorehabilitation Unit, University of Milan

    Milan, 20162
    Italy

    Active - Recruiting

  • Fondazione Policlinico A. Gemelli- IRCCS

    Rome, 00168
    Italy

    Active - Recruiting

  • Hospital Universitario Donostia

    Donostia, 20014
    Spain

    Active - Recruiting

  • St. George's University Hospital

    London, SW17 0RE
    United Kingdom

    Active - Recruiting

  • University of Florida

    Gainesville, Florida 32610
    United States

    Active - Recruiting

  • University of Kansas Medical Center, Department of Neurology

    Fairway, Kansas 66205
    United States

    Active - Recruiting

  • Virginia Commonwealth University

    Richmond, Virginia 23298
    United States

    Active - Recruiting

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