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New hope for muscle disease: experimental drug ARO-DM1 enters human trials

NCT ID NCT06138743

Disease control Sponsor: Sarepta Therapeutics, Inc. 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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 25, 2026 · Last updated Aug 12, 2026 · Updated 3 times

Summary

This study tests a new drug called ARO-DM1 in 78 adults aged 18-65 with type 1 myotonic dystrophy, a genetic muscle disorder. The goal is to check safety and how the body processes the drug. Participants receive either ARO-DM1 or a placebo by IV or injection. It is an early-stage trial, so results will guide future research.

What this could mean

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

Active substance
ARO-DM1
What this could lead to
If successful, this could lead to a treatment that controls symptoms of myotonic dystrophy type 1, improving muscle function and quality of life.
What could go wrong
This is an early-phase trial (Phase 1/2a) with only 78 participants, so safety and effectiveness are not yet proven. The drug may cause side effects or fail to show benefit.

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 78 people

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

Started

Mar 2024

Expected to finish

Dec 2026

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 65 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: * Genetically confirmed diagnosis of DM1 * Clinician-assessed signs of DM1 including clinically apparent myotonia * Onset of DM1 symptoms occurred after the age of 12 years * Walk for at least 10 meters independently at screening * Participants of childbearing potential must agree to use highly effective contraception in addition to a condom during the study and for at least 90 days following the end of study or last dose of study drug, whichever is later. Participants must not donate sperm or eggs during the study and for at least 90 days following the end of study or last dose of study drug whichever is later. Key Exclusion Criteria: * Inadequately controlled diabetes * Confirmed diagnosis of congenital DM1 * Uncontrolled hypertension * History of tibialis anterior (TA) biopsy within 3 months of Day 1 or planning to undergo TA biopsies during the study period * Clinically significant cardiac, liver or renal disease * Human immunodeficiency virus infection (seropositive) at screening * Seropositive for hepatitis B or hepatitis C at screening * Untreated or poorly controlled epilepsy * Treatment with anti-myotonia medication within a period of 5 half-lives of the medication prior to screening. * Abnormal coagulation parameters at screening including platelet count, international normalized ratio, prothrombin time, and activated partial thromboplastin time Note: Additional inclusion/exclusion criteria may apply per protocol

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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 places running it

    35 sites in 11 countries. The list below names each one and where it is.

  2. 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 ↗

  3. 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

Locations

  • Alfred Health

    RECRUITING

    Melbourne, Victoria, 3004, Australia

  • Assistance Publique Hôpitaux De Paris

    RECRUITING

    Paris, France

  • Azienda Ospedaliera Universitaria Pisana (AOUP)

    RECRUITING

    Pisa, Italy

  • CHU de Nice - Hôpital Pasteur 2

    RECRUITING

    Nice, France

  • Centre Hospitalier Universitaire De Bordeaux

    RECRUITING

    Bordeaux, France

  • Centre Hospitalier Universitaire De Lille

    RECRUITING

    Lille, France

  • Centro Clinico Nemo (Milan)

    RECRUITING

    Milan, Italy

  • China Medical University Hospital

    RECRUITING

    Taichung, 40447, Taiwan

  • Deutsches Zentrum für Neurodegenerative Erkrankungen e.V.

    RECRUITING

    Ulm, Germany

  • Fondazione Policlinico Universitario Agostino Gemelli IRCCS

    RECRUITING

    Roma, Italy

  • Fondazione Serena Onlus - Centro Clinico Nemo Brescia

    RECRUITING

    Gussago, Italy

  • Hospital Universitari Vall D Hebron

    RECRUITING

    Barcelona, Spain

  • Hospital Universitario Infanta Sofia

    RECRUITING

    Madrid, Spain

  • Hospital Universitario Torrecárdenas

    RECRUITING

    Almería, Spain

  • Klinikum der Universität München AöR

    RECRUITING

    Munich, Germany

  • Lampang Hospital

    RECRUITING

    Lampang, 52000, Thailand

  • Liverpool Hospital

    RECRUITING

    Liverpool, New South Wales, 2170, Australia

  • Mater Hospital Brisbane

    RECRUITING

    South Brisbane, Australia

  • McGill University

    RECRUITING

    Montreal, Canada

  • National Taiwan University Hospital

    RECRUITING

    Taipei, Taiwan

  • New Zealand Clinical Research Christchurch

    RECRUITING

    Christchurch, 8011, New Zealand

  • Nottingham University Hospitals NHS Trust

    RECRUITING

    Nottingham, United Kingdom

  • Nucleus Network Brisbane Clinic

    RECRUITING

    Herston, Australia

  • Royal Brisbane And Women's Hospital

    RECRUITING

    Herston, Queensland, Australia

  • Siriraj Hospital

    RECRUITING

    Bangkok, 10700, Thailand

  • Songklanagarind Hospital

    RECRUITING

    Hat Yai, Changwat Songkhla, 90110, Thailand

  • Southampton General Hospital, NHS Foundation Trust

    RECRUITING

    Southampton, United Kingdom

  • Sunshine Coast University Hospital

    RECRUITING

    Birtinya, Queensland, 4575, Australia

  • Taipei Veterans General Hospital

    RECRUITING

    Taipei, Taiwan

  • The National Hospital for Neurology and Neurosurgery, NHS Foundation Trust

    RECRUITING

    London, United Kingdom

  • UZ Leuven

    RECRUITING

    Leuven, Belgium

  • Universitair Ziekenhuis Gent

    RECRUITING

    Ghent, Belgium

  • University Hospital of Angers

    RECRUITING

    Angers, France

  • University Of Alberta

    RECRUITING

    Edmonton, Canada

  • University Of Calgary

    RECRUITING

    Calgary, Canada

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