Status:
UNKNOWN
The Natural History of Danon Disease
Lead Sponsor:
University of California, San Diego
Collaborating Sponsors:
Rocket Pharmaceuticals Inc.
Conditions:
Danon Disease
Eligibility:
All Genders
Up to 100 years
Brief Summary
Danon disease (DD) is a rare, X-linked disorder associated with severe cardiomyopathy, and in many cases, skeletal myopathy, and cognitive impairment caused by mutations in the LAMP2 gene. There is st...
Detailed Description
The study design will include: 1. Collection of information for all patients from: * Survey of patient or caregiver (if the patient has deceased) * Medical Charts 2. Collection of information ...
Eligibility Criteria
Inclusion
- • Living or deceased patients with a diagnosis of Danon disease(including patients who may or may not have undergone heart transplantation) based on a genetic test positive for the LAMP2 mutation
Exclusion
- • Patients without a genetic test positive for a LAMP2 mutation
Key Trial Info
Start Date :
November 30 2018
Trial Type :
OBSERVATIONAL
Allocation :
ESTIMATED
End Date :
November 1 2024
Estimated Enrollment :
200 Patients enrolled
Trial Details
Trial ID
NCT03766386
Start Date
November 30 2018
End Date
November 1 2024
Last Update
May 12 2022
Active Locations (1)
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1
University of California, San Diego
San Diego, California, United States, 92122-5671