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New study tracks toddlers with rare muscle disease to prep for future treatments

NCT ID NCT06503367

Knowledge-focused Sponsor: Nationwide Children's Hospital 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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 Aug 20, 2026 · Updated 2 times

Summary

This study watches how children under 5 with LAMA2-related muscular dystrophy move and develop over two years. Researchers will test muscle function, swallowing, breathing, and collect blood samples. The goal is to learn more about the disease so future treatments can be tested effectively. About 44 children will take part.

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 44 people

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

Started

May 2025

Expected to finish

Sep 2028

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

Study population will be neuromuscular clinic patients of site principal investigators, as well as individuals identified by our genetic counselor.

Ages

Up to 5 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: * Signed informed consent by the subject, parent(s) or legally authorized representative (LAR) and/or assent by the subject (when applicable). * Subject must be aged birth to less than 5.0 years of age at time of consent. * A confirmed diagnosis of LAMA2-RD confirmed via: a: Two pathogenic variants in the LAMA2 gene (via a CLIA-approved laboratory) or: b. muscle biopsy with absence of merosin (laminin-211) and at least one pathogenic variant in the LAMA2 gene * Absence of another confirmed genetic disease. * Willingness to maintain current exercise and/or physical therapy regimen for the duration of the clinical study. * Willingness to comply with the study protocol, including but not limited to, all study procedures and visits. Exclusion Criteria: * Acute medical illness or hospitalization within 30 days prior to informed consent. * Participation in a previous trial of any investigational agent for LAMA2-RD within 1 month prior to informed consent, or use of any other investigational therapy (including off-label use of Losartan) within 30 days prior to informed consent, or participation in other clinical studies, within 30 days (or 3 half-lives, whichever is longer) prior to informed consent, which in the opinion of the PI, may potentially confound results from this study. * Other significant medical condition, which in the opinion of the site Principal Investigator may confound interpretation of the clinical course of LAMA2- RD.

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Conditions

The condition(s) this trial relates to.

congenital merosin-deficient muscular dystrophy 1A LAMA2-related muscular dystrophy Muscular dystrophy congenital, merosin negative

As listed by the trial registrant

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

LAMA2-MD \(merosin deficient congenital muscular dystrophy, MDC1A\)

How to take part

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

  1. The places running it

    14 sites. 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

  • Ann & Robert H. Lurie Children's Hospital of Chicago

    RECRUITING

    Chicago, Illinois, 60611, United States

  • Boston Children's Hospital

    RECRUITING

    Boston, Massachusetts, 02115, United States

  • Children's Hospital of Pittsburgh

    RECRUITING

    Pittsburgh, Pennsylvania, 15224, United States

  • Cincinnati Children's Hospital Medical Center

    RECRUITING

    Cincinnati, Ohio, 45229, United States

  • National Institute of Neurological Disorders and Stroke, NIH

    RECRUITING

    Bethesda, Maryland, 20892, United States

  • Nationwide Children's Hospital

    RECRUITING

    Columbus, Ohio, 43205, United States

  • Stanford University

    RECRUITING

    Stanford, California, 94305, United States

  • University of California, Los Angeles

    RECRUITING

    Los Angeles, California, 90095, United States

  • University of Iowa Stead Family Children's Hospital

    RECRUITING

    Iowa City, Iowa, 52242, United States

  • University of Minnesota

    RECRUITING

    Saint Paul, Minnesota, 55108, United States

  • University of Rochester

    RECRUITING

    Rochester, New York, 14627, United States

  • University of Texas Southwestern

    RECRUITING

    Dallas, Texas, 75235, United States

  • University of Utah

    NOT_YET_RECRUITING

    Salt Lake City, Utah, 84112, United States

  • Washington University in St. Louis

    RECRUITING

    St Louis, Missouri, 63130, United States

More trials for these conditions

Other studies related to the condition(s) this trial covers.