Danon Disease Natural History Study
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).
| Condition(s) | Danon Disease |
|---|---|
| Status | Recruiting |
| Study type | Observational |
| 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). |
| Who can participate | Prospective Cohort: 1. Documentation of a pathogenic or likely pathogenic variant of the LAMP2 gene by a CLIA-certified genetic testing laboratory 2. Patient or parent/legal guardian are capable and willing to provide signed informed consent 3. Age ≥ 8 years at enrollment Female Prospective Cohort: 4. Evidence of left ventricular hypertrophy in the 12 months prior to or at enrollment. Retrospective (only) Cohort: 5. Documentation of a pathogenic or likely pathogenic variant of the LAMP2 gene by a CLIA-certified genetic testing laboratory 6. Patient or parent/legal guardian are capable and willing to provide signed informed consent, as required by local regulations 7. Age ≥ 8 years at enrollment 8. Prior cardiac transplantation or prior mechanical circulatory support 9. At least 30 days of |
| Ages | 8 Years |
| Sex | All |
| Lead sponsor | Rocket Pharmaceuticals Inc. |
| Locations | Phoenix, Arizona, United States; La Jolla, California, United States; Aurora, Colorado, United States; Gainesville, Florida, United States; Ann Arbor, Michigan, United States; St Louis, Missouri, United States (+7 more sites) |
| Start date | 2023-12-20 |
| NCT ID | NCT06214507 |
| Official listing | https://clinicaltrials.gov/study/NCT06214507 |