New study tracks rare heart disease to pave way for future treatments

NCT ID NCT06214507

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

Summary

This study follows 60 people with Danon disease, a rare genetic heart condition, to learn how it progresses over time. Researchers will monitor heart function and symptoms in males aged 8 and older and females aged 8 to 50. The goal is to gather natural history data that could support the development of new therapies.

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 crucial data to help design future treatments for Danon disease.

What could go wrong

This is an observational study, not a treatment trial, so it won't directly help participants. It may not lead to immediate breakthroughs.

Disclaimer Read more

This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.

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Conditions

The condition(s) this trial relates to.

cardiomyopathy Danon disease

As listed by the trial registrant

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

Contacts and locations

Study contacts

  • Contact

    Phone: •••-•••-•••• Email: •••••@•••••

Locations

  • Azienda Ospedaliera Universitaria Meyer IRCCS

    RECRUITING

    Florence, 50139, Italy

    Contact

    Contact Phone: •••-•••-•••• Email: •••••@•••••

  • Children's Hospital Colorado

    RECRUITING

    Aurora, Colorado, 80045, United States

    Contact

    Contact Phone: •••-•••-•••• Email: •••••@•••••

  • Columbia University Medical Center/New York Presbyterian

    RECRUITING

    New York, New York, 10032, United States

    Contact Phone: •••-•••-•••• Email: •••••@•••••

    Contact

    Contact Email: •••••@•••••

  • German Heart Center Munich

    RECRUITING

    München, Germany

    Contact Phone: •••-•••-•••• Email: •••••@•••••

    Contact

  • Hospital Universitario Puerta de Hierro Majadahonda

    RECRUITING

    Majadahonda, Madrid, Spain

    Contact Email: •••••@•••••

    Contact

  • Phoenix Children's Hospital

    RECRUITING

    Phoenix, Arizona, 85016, United States

    Contact Phone: •••-•••-•••• Email: •••••@•••••

    Contact

    Contact Phone: •••-•••-•••• Email: •••••@•••••

  • Sheba Medical Center

    RECRUITING

    Ramat Gan, 5265601, Israel

    Contact

    Contact Phone: •••-•••-•••• Email: •••••@•••••

  • Texas Children's Hospital

    RECRUITING

    Houston, Texas, 77030, United States

    Contact Phone: •••-•••-•••• Email: •••••@•••••

    Contact Phone: •••-•••-•••• Email: •••••@•••••

    Contact

  • University College London - Great Ormond Street Institute of Child Health

    RECRUITING

    London, WC1N 1DZ, United Kingdom

    Contact Phone: •••-•••-•••• Email: •••••@•••••

    Contact

    Contact

  • University of California, San Diego

    RECRUITING

    La Jolla, California, 92037, United States

    Contact Phone: •••-•••-•••• Email: •••••@•••••

    Contact

  • University of Florida College of Medicine

    RECRUITING

    Gainesville, Florida, 32610, United States

    Contact Phone: •••-•••-•••• Email: •••••@•••••

    Contact

  • University of Michigan

    RECRUITING

    Ann Arbor, Michigan, 48109, United States

    Contact

    Contact Email: •••••@•••••

    Contact

  • Washington University

    RECRUITING

    St Louis, Missouri, 63110, United States

    Contact Phone: •••-•••-•••• Email: •••••@•••••

    Contact

    Contact Phone: •••-•••-•••• Email: •••••@•••••