Testing RP-A601 for heart condition caused by PKP2 gene variants

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This Phase 1 dose escalation trial evaluates the safety and preliminary efficacy of RP-A601, administered as a single intravenous infusion, in high-risk adult patients diagnosed with PKP2 variant-mediated arrhythmogenic cardiomyopathy (PKP2-ACM). The study aims to determine the appropriate dosing and monitor any adverse effects while assessing initial treatment responses. Participants must meet specific inclusion criteria, including a documented PKP2 variant and a history of implantable cardioverter-defibrillator (ICD) implantation.

Why it matters

Who can join: the full rules

Key Inclusion Criteria:

1. Male or female ≥18 years at the time of signing the informed consent
2. Capable and willing to provide signed informed consent
3. Clinical diagnosis of ACM as defined by the 2010 revised Task Force Criteria (TFC)
4. Documentation of a pathogenic or likely pathogenic truncating variant in PKP2
5. History of Implantable Cardioverter-Defibrillator (ICD) implantation ≥6 months prior to enrollment
6. PVC frequency ≥500 per 24 hours by ambulatory rhythm monitoring
7. Left ventricular ejection fraction by echocardiogram or CMR ≥50%

Key Exclusion Criteria:

1. Anti-AAVrh.74 capsid neutralizing antibody titer of \>1:40
2. Cardiomyopathy related to a genetic etiology other than PKP2 truncating variant
3. Previous participation in a study of gene transfer or gene editing
4. Severe Right Ventricular (RV) dysfunction
5. New York Heart Association (NYHA) Class IV heart failure.

Where it's running

• University of California, San Diego
  La Jolla, California, United States Recruiting
• Duke University
  Durham, North Carolina, United States Recruiting
• Children's Hospital of Philadelphia
  Philadelphia, Pennsylvania, United States Withdrawn

Study contacts

• Principal investigator: Barry Greenberg, MD — University of California, San Diego
• Study coordinator: Clinical Information
• Email: clinicaltrials@rocketpharma.com
• Phone: 646-627-0033

Similar studies

Other studies for the same condition or treatment. Recruiting studies are listed first.

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• Natural history of PKP2-related arrhythmogenic cardiomyopathy

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• RP-A501 gene therapy for males with Danon disease

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How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.