Actively Recruiting
Danon Disease Natural History Study
Led by Rocket Pharmaceuticals Inc. · Updated on 2026-05-05
60
Participants Needed
13
Research Sites
323 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
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).
CONDITIONS
Official Title
Danon Disease Natural History Study
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Documentation of a pathogenic or likely pathogenic variant of the LAMP2 gene by a CLIA-certified genetic testing laboratory
- Patient or parent/legal guardian able and willing to provide signed informed consent
- Age 8 years or older at enrollment
- Female prospective cohort: evidence of left ventricular hypertrophy within 12 months before or at enrollment
- Retrospective cohort: prior cardiac transplantation or mechanical circulatory support with at least 30 days of medical records before these events
- Female retrospective cohort: prior evidence of left ventricular hypertrophy
You will not qualify if you...
- Concurrent enrollment in any other clinical study involving an investigational agent that could affect this study's interpretation
- Previous treatment with gene therapy
- Prospective cohort: prior mechanical circulatory support at enrollment
- Prospective cohort: prior cardiac transplantation at enrollment
- Female patients older than 51 years at enrollment
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 13 locations
1
Phoenix Children's Hospital
Phoenix, Arizona, United States, 85016
Actively Recruiting
2
University of California, San Diego
La Jolla, California, United States, 92037
Actively Recruiting
3
Children's Hospital Colorado
Aurora, Colorado, United States, 80045
Actively Recruiting
4
University of Florida College of Medicine
Gainesville, Florida, United States, 32610
Actively Recruiting
5
University of Michigan
Ann Arbor, Michigan, United States, 48109
Actively Recruiting
6
Washington University
St Louis, Missouri, United States, 63110
Actively Recruiting
7
Columbia University Medical Center/New York Presbyterian
New York, New York, United States, 10032
Actively Recruiting
8
Texas Children's Hospital
Houston, Texas, United States, 77030
Actively Recruiting
9
German Heart Center Munich
München, Germany
Actively Recruiting
10
Sheba Medical Center
Ramat Gan, Israel, 5265601
Actively Recruiting
11
Azienda Ospedaliera Universitaria Meyer IRCCS
Florence, Italy, 50139
Actively Recruiting
12
Hospital Universitario Puerta de Hierro Majadahonda
Majadahonda, Madrid, Spain
Actively Recruiting
13
University College London - Great Ormond Street Institute of Child Health
London, United Kingdom, WC1N 1DZ
Actively Recruiting
Research Team
C
Clinical Information
CONTACT
How is the study designed?
Study Type
OBSERVATIONAL
Masking
N/A
Allocation
N/A
Model
N/A
Primary Purpose
N/A
Number of Arms
2
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