PKP2-ACM Natural History Study

Not yet recruiting

• Conditions: Cardiomyopathies; Heart Diseases; Cardiovascular Diseases; Genetic Diseases

• Registration Number: NCT06644742
• Lead Sponsor: Rocket Pharmaceuticals Inc.

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.

Study Timeline

• Status Verified: 2024-10
• Study First Submitted: 2024-10-11
• Study First Submitted QC: 2024-10-14
• Last Update Submitted: 2024-10-14
• Study First Posted: 2024-10-16
• Last Update Posted: 2024-10-16
• Study Start: 2025-01
• Primary Completion: 2028-12
• Study Completion: 2028-12

Recruitment & Eligibility

• Status: NOT_YET_RECRUITING
• Sex: All
• Target Recruitment: 36

Inclusion Criteria

Not provided

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Exclusion Criteria

Not provided

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Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Heart rhythm and rate monitoring measures	36 months	Evaluate electrophysiology as assessed by heart rate and rhythm
Cardiac biomarkers	36 months	Evaluate heart health as assessed by cardiac biomarkers

Secondary Outcome Measures

Name	Time	Method
Characterize cardiovascular events	36 months	Evaluate cardiovascular health as assessed by the occurrence of clinical outcomes related to the cardiovascular system
Evaluate patient reported outcomes and quality of life measures	36 months	Evaluate patient reported outcomes and quality of life measures through questionnaires.
Interrogate ICDs	36 months	Evaluate heart health as assessed by interrogation of implanted cardioverter defibrillator
Evaluate changes in health status	36 months	Evaluate changes in health status as assessed by occurrence of clinical outcomes
Cardiac Structure and Performance	36 months	Evaluating heart changes through measures of cardiac structure and performance with echocardiogram.
Evaluate genetics	Cross sectional	Assess association with cardiomyopathy as assessed by genetic testing.