NCT05885412 · Rocket Pharmaceuticals Inc.
A Phase 1, Dose Escalation Trial of RP-A601 in Subjects With PKP2 Variant-Mediated Arrhythmogenic Cardiomyopathy (PKP2-ACM)
What this study is about
This Phase 1 gradually increasing doses trial will assess the safety and preliminary effectiveness of a single dose given through a vein (IV) infusion of RP-A601 in high-risk adult patients with PKP2-ACM.
View original scientific description
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.
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
- Male or female ≥18 years at the time of signing the informed consent
- Capable and willing to provide signed informed consent
- Clinical diagnosis of ACM as defined by the 2010 revised Task Force Criteria (TFC)
- Documentation of a pathogenic or likely pathogenic truncating variant in PKP2
- History of Implantable Cardioverter-Defibrillator (ICD) implantation ≥6 months prior to enrollment
- PVC frequency ≥500 per 24 hours by ambulatory rhythm monitoring
- Left ventricular ejection fraction by echocardiogram or CMR ≥50% Key
Exclusion criteria
- Anti-AAVrh.74 capsid neutralizing antibody titer of \>1:40
- Cardiomyopathy related to a genetic etiology other than PKP2 truncating variant
- Previous participation in a study of gene transfer or gene editing
- Severe Right Ventricular (RV) dysfunction
- New York Heart Association (NYHA) Class IV heart failure.
Where
- La Jolla, California
- Durham, North Carolina
- Philadelphia, Pennsylvania
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 Apr 16, 2026 · Source of record for eligibility and locations