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

NCT ID NCT06214507

Recruiting now Knowledge-focused Sponsor: Rocket Pharmaceuticals Inc. Source: ClinicalTrials.gov ↗

First seen Jun 27, 2026 · Last updated Jul 29, 2026 · Updated 4 times

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.

This is an AI summary of the original study and may miss details. Read our disclaimer.

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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.

DANON DISEASE

Contacts and locations

Locations

Azienda Ospedaliera Universitaria Meyer IRCCS
RECRUITING

Florence, 50139, Italy
Children's Hospital Colorado
RECRUITING

Aurora, Colorado, 80045, United States
Columbia University Medical Center/New York Presbyterian
RECRUITING

New York, New York, 10032, United States
German Heart Center Munich
RECRUITING

München, Germany
Hospital Universitario Puerta de Hierro Majadahonda
RECRUITING

Majadahonda, Madrid, Spain
Phoenix Children's Hospital
RECRUITING

Phoenix, Arizona, 85016, United States
Sant Joan de Déu Hospital
RECRUITING

Barcelona, 08950, Spain
Sheba Medical Center
RECRUITING

Ramat Gan, 5265601, Israel
Texas Children's Hospital
RECRUITING

Houston, Texas, 77030, United States
University College London - Great Ormond Street Institute of Child Health
RECRUITING

London, WC1N 1DZ, United Kingdom
University of California, San Diego
RECRUITING

La Jolla, California, 92037, United States
University of Florida College of Medicine
RECRUITING

Gainesville, Florida, 32610, United States
University of Michigan
RECRUITING

Ann Arbor, Michigan, 48109, United States
University of Texas, Southwestern
RECRUITING

Dallas, Texas, 75235, United States
Washington University
RECRUITING

St Louis, Missouri, 63110, United States

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