An observational study in Cardiomyopathies, Heart Diseases and Cardiovascular Diseases, sponsored by Rocket Pharmaceuticals Inc.. Recruiting at 3 sites in 2 countries. Open to participants aged 12 Years and older. Per ClinicalTrials.gov, last updated 2026-10-14.
Sponsored by Rocket Pharmaceuticals Inc. · Observational
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.
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.
553 studies on the registry are indexed under Cardiomyopathies; 152 are open to participants now.
This study's planned enrollment of 36 is below the median of 200 across 263 observational studies indexed under Cardiomyopathies.
Browse Cardiomyopathies studies →Rocket Pharmaceuticals Inc. is the lead sponsor of 19 studies on the registry; 7 are open to participants now.
Counted across the registry records on this site, refreshed daily.
Patients 12 years of age and over with confirmed PKP2-ACM.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Patients must meet all the following criteria (and none of the exclusion criteria) to be eligible for study participation:
Exclusion Criteria
Patients meeting any of the following criteria are excluded from study participation:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
The share of blood the heart’s main pumping chamber (the left ventricle) pushes out with each beat, measured by an echocardiogram (heart ultrasound) or a MUGA scan. About 50–70% is normal.
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
The New York Heart Association’s four classes of how much heart failure limits physical activity.
Studies set their own limit — many outside heart failure leave out class III–IV, while heart failure studies may ask for a particular class. Check the class this study names.
The human immunodeficiency virus, which weakens the immune system.
Many studies now accept people whose HIV is controlled on treatment, judged by the viral load and the CD4 count. Check the limits this study sets.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
LVEF (left ventricular ejection fraction)
The share of blood the heart’s main pumping chamber (the left ventricle) pushes out with each beat, measured by an echocardiogram (heart ultrasound) or a MUGA scan. About 50–70% is normal.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Investigational drug or device
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
NYHA class (heart failure)
The New York Heart Association’s four classes of how much heart failure limits physical activity.
Studies set their own limit — many outside heart failure leave out class III–IV, while heart failure studies may ask for a particular class. Check the class this study names.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
HIV
The human immunodeficiency virus, which weakens the immune system.
Many studies now accept people whose HIV is controlled on treatment, judged by the viral load and the CD4 count. Check the limits this study sets.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Heart rhythm and rate monitoring measures
Evaluate electrophysiology as assessed by heart rate and rhythm
Time frame: 36 months
Cardiac biomarkers
Evaluate heart health as assessed by cardiac biomarkers
Time frame: 36 months
Characterize cardiovascular events
Evaluate cardiovascular health as assessed by the occurrence of clinical outcomes related to the cardiovascular system
Time frame: 36 months
Evaluate patient reported outcomes and quality of life measures
Evaluate patient reported outcomes and quality of life measures through questionnaires.
Time frame: 36 months
Interrogate ICDs
Evaluate heart health as assessed by interrogation of implanted cardioverter defibrillator
Time frame: 36 months
Evaluate changes in health status
Evaluate changes in health status as assessed by occurrence of clinical outcomes
Time frame: 36 months
Cardiac Structure and Performance
Evaluating heart changes through measures of cardiac structure and performance with echocardiogram.
Time frame: 36 months
Evaluate genetics
Assess association with cardiomyopathy as assessed by additional genetic testing.
Time frame: Cross sectional
Plan to share: No
No publications or documents are linked to this record.
From the registry record's own update history. This site started tracking changes on Sep 25, 2026; for anything earlier, see the record history on ClinicalTrials.gov ↗
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Rocket Pharmaceuticals Inc.