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700-Patient study seeks key clues to muscle disease

NCT ID NCT03981575

Knowledge-focused Sponsor: Virginia Commonwealth University Source: ClinicalTrials.gov ↗

What the study statuses mean

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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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 is following 700 adults with myotonic dystrophy type 1 (DM1) over two years. Researchers are measuring walking speed, lung function, and muscle tissue changes to find reliable markers of disease progression. The goal is to improve future clinical trials by better understanding how DM1 behaves over time.

What this could mean

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

What this could lead to
If successful, this study could provide the tools needed to design better treatments for myotonic dystrophy type 1.
What could go wrong
This is an observational study, not a treatment trial. It may not directly benefit participants and results depend on consistent data collection over 24 months.

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.

Participants

About 700 people

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

Started

Jan 2019

Expected to finish

Dec 2026

An estimate. End dates often move.

Lead sponsor

Other sponsor

The registry's catch-all category, for sponsors it does not file as a company, a government agency, or a research network.

Who can take part

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

Who is studied

DM1 has a prevalence rate of approximately 1 per 2,300. There are no expected gender differences. Both men and women will be selected for this study. Children with DM1 are not included in this project because the pathophysiological basis of congenital and childhood DM1 appears to be mechanistically distinct.

Ages

18 to 70 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.

Inclusion criteria: * Age 18 to 70 (inclusive) * Competent to provide informed consent * Clinical diagnosis of DM1 based on research criteria1 or positive genetic test * Comment: The clinical research criteria require myotonia, muscle weakness in a characteristic distribution, and history of similar findings in a first degree relative. Genetic testing confirmed the diagnosis of DM1 in \> 99% of individuals who satisfied these criteria.2 Exclusion criteria: * Symptomatic renal or liver disease, uncontrolled diabetes or thyroid disorder, or active malignancy other than skin cancer. * Current alcohol or substance abuse * Concurrent enrollment in clinical trial for DM1, or participation in trial within 6 months of entry. * Concurrent pregnancy or planned pregnancy during the course of the study. * Concurrent medical condition that would, in the opinion of the investigator or clinical evaluator, compromise performance on study measures. * Note: non-ambulatory participants are not excluded, but are limited to \<15% of enrollment. Inclusion criteria for participants in the muscle biopsy sub-study: • Of the 95 patients undergoing the tibialis anterior muscle biopsy, at least half will have at least moderate weakness of ankle dorsiflexion, defined as MRC score ≤ 4+. This is in order to obtain a muscle tissue sample in a person more severely affected with myotonic dystrophy. Approximately 10 patients at each site will undergo the muscle biopsy. Exclusion criteria for 95 participants in the muscle biopsy sub-study: * Known CTG repeat expansion size less than 100 repeats, unless there are clear cut signs of limb weakness and muscle wasting. This is in order to obtain a muscle tissue sample in a person more severely affected with myotonic dystrophy. * Use of anticoagulant such as warfarin or a direct oral anticoagulant (e.g. dabigatran) due to the increased risk of bleeding. * Use of aspirin or non-steroidal anti-inflammatory agents should be discontinued 3 days prior to the biopsy procedure, if possible. * Platelet count \<50,000 (if known) due to the increased risk of bleeding. * History of a bleeding disorder due to the increased risk of bleeding. * Advanced wasting of tibialis anterior (TA) muscle that precludes needle muscle biopsy in order to ensure that a sample taken would be of muscle and not just fat and fascia. * Previous muscle biopsy of either TA in order to provide muscle tissue samples of non-biopsied muscles.

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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.

DM1 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

    15 sites in 5 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

  • Centro Clinico NeMO

    COMPLETED

    Milan, Italy

  • Friedrich Baur Institute, Ludwig-Maximilians-Universität München

    RECRUITING

    München, Germany

  • Houston Methodist Neurological Institute

    RECRUITING

    Houston, Texas, 77030, United States

  • Kansas University Medical Center

    RECRUITING

    Kansas City, Kansas, 66160, United States

  • Ohio State University

    RECRUITING

    Columbus, Ohio, 43210, United States

  • Radboud University Medical Center

    RECRUITING

    Nijmegen, Netherlands

  • St. George's, University of London

    RECRUITING

    London, United Kingdom

  • University College London

    RECRUITING

    London, United Kingdom

  • University of Auckland

    RECRUITING

    Auckland, New Zealand

  • University of California, Los Angeles

    RECRUITING

    Los Angeles, California, 90095, United States

  • University of California, San Diego

    RECRUITING

    La Jolla, California, 92703, United States

  • University of Colorado - Denver

    RECRUITING

    Denver, Colorado, 80204, United States

  • University of Florida

    RECRUITING

    Gainesville, Florida, 32611, United States

  • University of Iowa

    RECRUITING

    Iowa City, Iowa, 52242, United States

  • University of Rochester

    RECRUITING

    Rochester, New York, 14642, United States

  • Université de Sherbrooke

    ACTIVE_NOT_RECRUITING

    Québec, Canada

  • Virginia Commonwealth University

    RECRUITING

    Richmond, Virginia, 23298, United States

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