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New hope for muscle stiffness: experimental drug ATX-01 enters human trials

NCT ID NCT06300307

Disease control Sponsor: ARTHEx Biotech S.L. Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now This study
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This study tests a new drug called ATX-01 in 56 adults with myotonic dystrophy type 1, a condition that causes muscle stiffness and weakness. The drug aims to block a molecule that may contribute to the disease. The trial will first give a single dose, then multiple doses, to check safety and how well it works compared to a placebo.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance
ATX-01 (a synthetic molecule that blocks a specific microRNA)
What this could lead to
If successful, this could point toward a treatment that eases muscle symptoms in people with myotonic dystrophy type 1.
What could go wrong
This is an early-phase trial with only 56 participants, so results may not apply to everyone. The drug may not work or could cause side effects.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 1/2

Runs two stages together: safety and dose first, then whether the treatment works.

Participants

About 56 people

The number the study aims to enrol. It can still change while the study runs.

Started

Oct 2024

Expected to finish

Jul 2027

An estimate. End dates often move.

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

18 to 64 years

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

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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Conditions

The condition(s) this trial relates to.

myotonic dystrophy myotonic dystrophy type 1

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Myotonic dystrophy 1

How to take part

Only the study team decides who joins. These are the ways to reach them.

  1. The study's own enquiry address

    This study publishes an address for enquiries. See it below .

  2. The places running it

    12 sites in 7 countries. The list below names each one and where it is.

  3. The official record

    ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.

    Open the record ↗

  4. A doctor treating you

    A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.

Contacts and locations

Study contacts

  • Contact

    Email: •••••@•••••

Locations

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

    RECRUITING

    Chicoutimi, Quebec, G7H 5H6, Canada

  • Fondazione Policlinico A. Gemelli- IRCCS

    RECRUITING

    Rome, 00168, Italy

  • Hospital Universitario Donostia

    RECRUITING

    Donostia / San Sebastian, 20014, Spain

  • Institute of Myology

    RECRUITING

    Paris, 75651, France

  • Radboudumc

    RECRUITING

    Nijmegen, 6525, Netherlands

  • St. George's University Hospital

    RECRUITING

    London, SW17 0RE, United Kingdom

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

    RECRUITING

    Milan, 20162, Italy

  • UCLA

    RECRUITING

    Los Angeles, California, 90095, United States

  • University of Florida

    RECRUITING

    Gainesville, Florida, 32610, United States

  • University of Iowa Health Care - Department of Neurology

    RECRUITING

    Iowa City, Iowa, 52242, United States

  • University of Kansas Medical Center, Department of Neurology

    RECRUITING

    Fairway, Kansas, 66205, United States

  • Virginia Commonwealth University

    RECRUITING

    Richmond, Virginia, 23298, United States

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