The Natural History of Arrhythmogenic Cardiomyopathy With Pathogenic Plakophilin-2 Variants (PKP2-ACM): An Observational Cohort Study
Overview
- Phase
- Not Applicable
- Intervention
- Not specified
- Conditions
- Cardiomyopathies
- Sponsor
- Rocket Pharmaceuticals Inc.
- Enrollment
- 36
- Locations
- 3
- Primary Endpoint
- Heart rhythm and rate monitoring measures
- Status
- Recruiting
- Last Updated
- 7 months ago
Overview
Brief Summary
The goal of this study is to describe the natural history and clinical events for patients who have Arrhythmogenic Cardiomyopathy with Pathogenic Plakophilin-2 Variants (PKP2-ACM) managed with standard of care.
Detailed Description
This is an observational study with both retrospective and prospective data collection. The study is designed to describe the natural history of PKP2-ACM including the signs and symptoms, key clinical events, and impact of the disease on quality of life. A hybrid (retrospective and prospective data collection) approach is being taken with the aim of achieving robust and longitudinal data generation.
Investigators
Eligibility Criteria
Inclusion Criteria
- Not provided
Exclusion Criteria
- Not provided
Outcomes
Primary Outcomes
Heart rhythm and rate monitoring measures
Time Frame: 36 months
Evaluate electrophysiology as assessed by heart rate and rhythm
Cardiac biomarkers
Time Frame: 36 months
Evaluate heart health as assessed by cardiac biomarkers
Secondary Outcomes
- Characterize cardiovascular events(36 months)
- Evaluate patient reported outcomes and quality of life measures(36 months)
- Interrogate ICDs(36 months)
- Evaluate changes in health status(36 months)
- Cardiac Structure and Performance(36 months)
- Evaluate genetics(Cross sectional)