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NCT06300307PHASE1, PHASE2Recruiting

Study of ATX-01 in Participants With DM1

ARTHEx Biotech S.L.

Start Date

10/15/2024

Completion Date

7/1/2027

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

Age Range: 18 years to 64 years

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

Interventions

DRUG

ATX-01

DRUG

Placebo

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Conditions

Myotonic Dystrophy 1

Locations

UCLA

Los Angeles, California 90095

United States

University of Florida

Gainesville, Florida 32610

United States

University of Iowa Health Care - Department of Neurology

Iowa City, Iowa 52242

United States

University of Kansas Medical Center, Department of Neurology

Fairway, Kansas 66205

United States

Virginia Commonwealth University

Richmond, Virginia 23298

United States

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

Chicoutimi, Quebec G7H 5H6

Canada

Institute of Myology

Paris, 75651

France

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

Milan, 20162

Italy

Fondazione Policlinico A. Gemelli- IRCCS

Rome, 00168

Italy

Radboudumc

Nijmegen, 6525

Netherlands

Hospital Universitario Donostia

Donostia / San Sebastian, 20014

Spain

St. George's University Hospital

London, SW17 0RE

United Kingdom