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RecruitingPhase 1/Phase 2

Study of ATX-01 in Participants With DM1

ARTHEx Biotech S.L. · NCT06300307

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Opens the official ClinicalTrials.gov record, with the study team’s contacts. Bored Studies doesn’t enroll anyone.

Key facts

Compensation
Not posted. Ask the study team before screening.
Who can join
Ages 18–64 · All sexes
Healthy volunteers
Not accepted (a condition is required)
Study type
Interventional · Phase 1, Phase 2
Target enrollment
56
US sites recruiting
5
Study start
October 15, 2024
Record last updated
February 10, 2026

Status re-checked against ClinicalTrials.gov within the last 12 hours.

What the study is about

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.

Being studied: ATX-01; Placebo

Conditions: Myotonic Dystrophy 1

Eligibility criteria

Copied from the official record. The study team makes the final decision at screening.

Show full criteria

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

Recruiting US locations

  • UCLA

    Los Angeles, CA 90095

    Site contact: Michael Yan · 310-825-3264 · MYan@mednet.ucla.edu

  • University of Florida

    Gainesville, FL 32610

    Site contact: Brandy Donahue-Quails · Brandy.Donahue-Quails@neurology.ufl.edu

  • University of Iowa Health Care - Department of Neurology

    Iowa City, IA 52242

    Site contact: Heena Olalde · 319-356-8326 · heena-olalde@uiowa.edu

  • University of Kansas Medical Center, Department of Neurology

    Fairway, KS 66205

    Site contact: Cassidy Nelson · 913-588-3653 · cnelson15@kumc.edu

  • Virginia Commonwealth University

    Richmond, VA 23298

    Site contact: Jodie Howell · 804-828-6110 · jodie.howell@vcuhealth.org

Study contacts (from the official record)

  • Project Manager · +34676229821 · clinical@arthexbiotech.com
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Study of ATX-01 in Participants With DM1 (NCT06300307) | Bored Studies