NCT06644742

PKP2-ACM Natural History Study

The Natural History of Arrhythmogenic Cardiomyopathy With Pathogenic Plakophilin-2 Variants (PKP2-ACM): An Observational Cohort Study

• Status: Recruiting
• Phase: —
• Study type: Observational
• Enrollment: 36 (estimated)
• Sponsor: Rocket Pharmaceuticals Inc. · Industry
• Sex: All
• Age: 12 Years
• Healthy volunteers: Not accepted

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.

Conditions

• Cardiomyopathies
• Heart Diseases
• Cardiovascular Diseases
• Genetic Diseases

Timeline

• Start date: 2026-03-01
• Primary completion: 2030-12-01
• Completion: 2031-04-01
• First posted: 2024-10-16
• Last updated: 2026-02-27

Locations

3 sites across 2 countries: United States, Netherlands