A Phase 1 interventional study of RP-A601 in PKP2 Arrhythmogenic Cardiomyopathy (PKP2-ACM), sponsored by Rocket Pharmaceuticals Inc.. Recruiting at 3 sites in United States. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2026-04-16.
Sponsored by Rocket Pharmaceuticals Inc. · Phase 1, Interventional, and Treatment
This Phase 1 dose escalation trial will assess the safety and preliminary efficacy of a single dose intravenous infusion of RP-A601 in high-risk adult patients with PKP2-ACM.
48 studies on the registry are indexed under Arrhythmogenic Right Ventricular Dysplasia; 28 are open to participants now.
This study's planned enrollment of 9 is below the median of 22 across 15 interventional studies indexed under Arrhythmogenic Right Ventricular Dysplasia.
Browse Arrhythmogenic Right Ventricular Dysplasia 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.
Key Inclusion Criteria:
Key Exclusion Criteria:
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.
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.
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.
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.
Single ascending dose of RP-A601 in 2 consecutive cohorts
Genetic: RP-A601
RP-A601 is a recombinant viral vector composed of an AAV serotype rh.74 (AAVrh.74) capsid encapsulating the transgene, human plakophilin 2 (PKP2), transcript variant 2a (PKP2a)
Evaluation of safety associated with RP-A601
Incidence of treatment emergent adverse events (TEAEs), incidence of Serious Adverse Events (SAEs), and identification of Dose Limiting Toxicities (DLTs)
Time frame: 12 months post-infusion
Preliminary efficacy of RP-A601 - Myocardial PKP2 protein expression
Assessment of changes in myocardial PKP2 protein expression
Time frame: 12 months post-infusion
Preliminary efficacy of RP-A601 - Ventricular ectopy and arrhythmia
Assessment of changes in levels of ventricular ectopy and arrhythmia on cardiac rhythm monitoring
Time frame: 12 months post-infusion
Preliminary efficacy of RP-A601 - Cardiac biomarkers
Assessment of changes in circulating levels of cardiac biomarkers of injury and stress
Time frame: 12 months post-infusion
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Arrhythmogenic Right Ventricular Dysplasia →
Rocket Pharmaceuticals Inc.