Danon Disease Natural History Study

Recruiting now

Conditions studied: Danon Disease

In brief

The goal of this international observational study is to learn about the natural history of Danon disease in male patients (≥8 years of age) and female patients (8 to 50 years of age).

Key facts

Study ID

NCT06214507

Run by

Rocket Pharmaceuticals Inc.

People needed

60

Starts

2023-12-20

Expected to finish

2030-03-01

Last updated by the study team

2026-07-28

Who can join

Age: 8 and older. Sex: any. Healthy volunteers: not accepted.

Where it is running

• Phoenix Children's Hospital — Phoenix, Arizona, United States (enrolling)
• University of California, San Diego — La Jolla, California, United States (enrolling)
• Children's Hospital Colorado — Aurora, Colorado, United States (enrolling)
• University of Florida College of Medicine — Gainesville, Florida, United States (enrolling)
• University of Michigan — Ann Arbor, Michigan, United States (enrolling)
• Washington University — St Louis, Missouri, United States (enrolling)
• Columbia University Medical Center/New York Presbyterian — New York, New York, United States (enrolling)
• University of Texas, Southwestern — Dallas, Texas, United States (enrolling)
• Texas Children's Hospital — Houston, Texas, United States (enrolling)
• German Heart Center Munich — München, Germany (enrolling)
• Sheba Medical Center — Ramat Gan, Israel (enrolling)
• Azienda Ospedaliera Universitaria Meyer IRCCS — Florence, Italy (enrolling)
• Hospital Universitario Puerta de Hierro Majadahonda — Majadahonda, Madrid, Spain (enrolling)
• Sant Joan de Déu Hospital — Barcelona, Spain (enrolling)
• University College London - Great Ormond Street Institute of Child Health — London, United Kingdom (enrolling)

Full record on ClinicalTrials.gov

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