NCT06644742 · Rocket Pharmaceuticals Inc.
PKP2-ACM Natural History Study
What this study is about
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 the usual treatment.
View original scientific description
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.
Primary outcome measures
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
Who can participate
This study lists these criteria on ClinicalTrials.gov. A study coordinator reviews eligibility during screening — this page does not determine whether you qualify.
Inclusion criteria
- Patients must meet all the following criteria (and none of the
Exclusion criteria
- ) to be eligible for study participation:
- 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
- History of ICD implantation ≥6 months prior to ICF provision
- Left ventricular ejection fraction by echocardiogram or cardiac magnetic resonance (CMR) ≥50% at ≤12 months prior to ICF provision Exclusion Criteria Patients meeting any of the following criteria are excluded from study participation:
- Gene testing indicates that the subject's arrhythmia or cardiomyopathy may be related to a genetic etiology other than PKP2 truncating variant.
- Concurrent participation in any other clinical investigation involving use of an investigational agent that could confound results of this study.
- Previous participation in a study of gene transfer or gene editing.
- NYHA Class IV heart failure.
- Presence or requirement for mechanical circulatory support (MCS) or predicted need for MCS or heart transplantation within 6 months of enrollment.
- Prior cardiac or other organ (lung, liver, other) transplantation.
- Pacemaker dependent rhythm documented, as assessed by the principal investigator ≤12 months prior to enrollment.
- Positive human immunodeficiency virus (HIV) antibody test.
- Unwillingness to comply with study procedures, including follow-up as specified by this protocol, or unwillingness to fully cooperate with the investigator.
Where
- Boston, Massachusetts
- Durham, North Carolina
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Frequently asked questions
What is a clinical trial?
A clinical trial is a research study that tests new medical treatments, drugs, devices, or procedures to determine their safety and effectiveness. Trials are carefully designed and monitored to protect participants while advancing medical knowledge.
Is it safe to participate?
Clinical trials follow strict safety guidelines and ethical standards. Trials must be reviewed and approved, and participants are closely monitored by medical professionals throughout the study. You can withdraw at any time if you choose.
Will I be compensated?
Many clinical trials offer compensation for your time, travel expenses, and inconvenience. The specific compensation varies by study and will be discussed during the screening process. All study-related medical care is typically provided at no cost to participants.
Will I receive a placebo instead of treatment?
When effective treatment exists, participants typically receive either the standard treatment plus the study intervention, or the standard treatment plus placebo. You would not be denied effective care. Placebos are primarily used when no proven treatment is available, or in addition to standard care. Your trial consent form will clearly explain what treatments you may receive.
Can I leave a trial if I change my mind?
Absolutely. Participation in clinical trials is completely voluntary. You have the right to withdraw from the study at any time, for any reason, without penalty or loss of benefits to which you are otherwise entitled.
How long does a clinical trial last?
Trial duration varies widely depending on the study design and purpose. Some trials last just a few weeks, while others may continue for months or years. The study coordinator will provide specific timeline information during your screening call.
Data: ClinicalTrials.gov · synced Feb 27, 2026 · Source of record for eligibility and locations