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Study of ATX-01 in Participants With DM1

RecruitingPhase 1, Phase 2Ages 18 Years - 64 YearsAllARTHEx Biotech S.L.
NCT06300307 on ClinicalTrials.gov ↗
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In plain words

What the trial is, and whether it could suit you

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.

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📋 Eligibility rules (as published)
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
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What it tests

  • ATX-01
  • Placebo
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Where

12 sites
UCLA
Los Angeles, United States
University of Florida
Gainesville, United States
University of Iowa Health Care - Department of Neurology
Iowa City, United States
University of Kansas Medical Center, Department of Neurology
Fairway, United States
Virginia Commonwealth University
Richmond, United States
Centre Intégré Universitaire de Santé et Services Sociaux du Saguenay-Lac-St-Jean
Chicoutimi, Canada
Institute of Myology
Paris, France
The NeMO Clinical Center in Milan, Neurorehabilitation Unit, University of Milan
Milan, Italy

…and 4 more on ClinicalTrials.gov.

🤝 Taking part is your choice, and you can leave at any time. Only the trial team can confirm you are eligible — always decide together with your own doctor.
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