PKP2-ACM Natural History Study
Trial statusRecruiting
Trial phaseNot listed
Trial typeObservational
Biological sexAll
Age12+
SponsorRocket Pharmaceuticals Inc.
About this trial
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.
Eligibility criteria
This trial does not accept healthy volunteersQualifiers
None
Disqualifiers
Male or female age 12 years or older at the time of providing informed consent (i.e., ICF provision).
Capable and willing to provide signed informed consent and/or assent, which includes compliance with the requirements and restrictions listed in the ICF and protocol.
Clinical diagnosis of arrhythmogenic cardio myopathy (ACM)
Documentation of a pathogenic or likely pathogenic variant in PKP2 by a CLIA-certified genetic testing laboratory
Trial population
Patients 12 years of age and over with confirmed PKP2-ACM.
Trial design
Design model
Cohort
Time perspective
Prospective
Treatments tested in this trial
Not listed
Trial groups
36 Participants
are grouped into 1 trial groupGroup A: Prospective and Retrospective Cohort (No Intervention)
Trial outcomes
Primary outcomes
1
Heart rhythm and rate monitoring measures
Evaluate electrophysiology as assessed by heart rate and rhythm
Time frame
36 months2
Cardiac biomarkers
Evaluate heart health as assessed by cardiac biomarkers
Time frame
36 monthsSecondary outcomes
1
Characterize cardiovascular events
Evaluate cardiovascular health as assessed by the occurrence of clinical outcomes related to the cardiovascular system
Time frame
36 months2
Evaluate patient reported outcomes and quality of life measures
Evaluate patient reported outcomes and quality of life measures through questionnaires.
Time frame
36 months3
Interrogate ICDs
Evaluate heart health as assessed by interrogation of implanted cardioverter defibrillator
Time frame
36 months4
Evaluate changes in health status
Evaluate changes in health status as assessed by occurrence of clinical outcomes
Time frame
36 monthsSponsors and contacts
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Source ClinicalTrials.gov