Status:
RECRUITING
Danon Disease Natural History Study
Lead Sponsor:
Rocket Pharmaceuticals Inc.
Conditions:
Danon Disease
Eligibility:
All Genders
8+ years
Brief Summary
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).
Detailed Description
This is an international observational study with both retrospective and prospective data collection. The study is designed to describe the natural history of Danon disease, a rare X-linked genetic di...
Eligibility Criteria
Inclusion
- Prospective Cohort:
- Documentation of a pathogenic or likely pathogenic variant of the LAMP2 gene by a CLIA-certified genetic testing laboratory
- Patient or parent/legal guardian are capable and willing to provide signed informed consent
- Age ≥ 8 years at enrollment
- Female Prospective Cohort:
- Evidence of left ventricular hypertrophy in the 12 months prior to or at enrollment.
- Retrospective (only) Cohort:
- Documentation of a pathogenic or likely pathogenic variant of the LAMP2 gene by a CLIA-certified genetic testing laboratory
- Patient or parent/legal guardian are capable and willing to provide signed informed consent, as required by local regulations
- Age ≥ 8 years at enrollment
- Prior cardiac transplantation or prior mechanical circulatory support
- At least 30 days of retrospective medical records available prior to cardiac transplantation or mechanical circulatory support
- Female Retrospective (only) Cohort:
- Prior evidence of left ventricular hypertrophy.
- Key Exclusion Criteria:
- All Cohorts:
- Concurrent enrollment in any other clinical investigation involving use of an investigational agent for any condition at time of enrollment to this study that could confound interpretation of this study
- Previous treatment with a gene therapy
- Prospective Cohort:
- Prior mechanical circulatory support at time of enrollment to this study
- Prior cardiac transplantation at time of enrollment to this study
- Female patients:
- Age \>51 years at enrollment
Exclusion
Key Trial Info
Start Date :
December 20 2023
Trial Type :
OBSERVATIONAL
Allocation :
ESTIMATED
End Date :
March 1 2030
Estimated Enrollment :
60 Patients enrolled
Trial Details
Trial ID
NCT06214507
Start Date
December 20 2023
End Date
March 1 2030
Last Update
January 8 2026
Active Locations (13)
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1
Phoenix Children's Hospital
Phoenix, Arizona, United States, 85016
2
University of California, San Diego
La Jolla, California, United States, 92037
3
Children's Hospital Colorado
Aurora, Colorado, United States, 80045
4
University of Florida College of Medicine
Gainesville, Florida, United States, 32610