Ignite Creation Date:
2024-05-06 @ 7:59 PM
Last Modification Date:
2024-10-26 @ 3:18 PM
Study NCT ID:
NCT06214507
Status:
RECRUITING
Last Update Posted:
2024-04-15
First Post:
2023-11-17
Brief Title:
Danon Disease Natural History Study
Sponsor:
Rocket Pharmaceuticals Inc
Organization:
Rocket Pharmaceuticals Inc
Study Overview
Official Title:
An Observational Study of Genetic Cardiomyopathy Danon Disease
Status:
RECRUITING
Status Verified Date:
2024-08
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
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 The key objectives include assessing change over time in cardiac structure left ventricular mass and wall thickness cardiac biomarkers symptoms and quality of life and the incidence of clinical events such as HF hospitalization cardiac transplantation and death
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 which is a rare x-linked genetic disorder and one of the most severe and penetrant forms of inherited cardiomyopathy This study will collect data about the clinical course of Danon disease including signs and symptoms key clinical events and the impact of the disease on quality of life managed with current standard of care A hybrid retrospective and prospective data collection approach is being taken with the aim of achieving robust and longitudinal data generation
Subset of patients will be used as an External Control Arm for comparison to RP-A501 Trial participants
Subset of patients will be used as an External Control Arm for comparison to RP-A501 Trial participants
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
False
Is a FDA Regulated Device?:
False
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None