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

NCT ID NCT06503367

Recruiting now Knowledge-focused Sponsor: Nationwide Children's Hospital Source: ClinicalTrials.gov ↗

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

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.

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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)

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

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