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RecruitingPhase 1

A Phase 1, Dose Escalation Trial of RP-A601 in Subjects With PKP2 Variant-Mediated Arrhythmogenic Cardiomyopathy (PKP2-ACM)

Rocket Pharmaceuticals Inc. · NCT05885412

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Opens the official ClinicalTrials.gov record, with the study team’s contacts. Bored Studies doesn’t enroll anyone.

Key facts

Compensation
Not posted. Ask the study team before screening.
Who can join
Ages 18+ · All sexes
Healthy volunteers
Not accepted (a condition is required)
Study type
Interventional · Phase 1
Target enrollment
9
US sites recruiting
2
Study start
August 29, 2023
Record last updated
April 16, 2026

Status re-checked against ClinicalTrials.gov within the last 12 hours.

What the study is about

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.

Being studied: RP-A601

Conditions: PKP2 Arrhythmogenic Cardiomyopathy (PKP2-ACM)

Eligibility criteria

Copied from the official record. The study team makes the final decision at screening.

Show full criteria

Key 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.

Recruiting US locations

  • University of California, San Diego

    La Jolla, CA 92037

    Site contact: Barry Greenberg, MD · 858-246-2987 · Bgreenberg@health.ucsd.edu

  • Duke University

    Durham, NC 27710

    Site contact: Meghan Pergola, MPH · meghan.pergola@duke.edu

Study contacts (from the official record)

  • Clinical Information · 646-627-0033 · clinicaltrials@rocketpharma.com
All heart & blood pressure studies in California →
A Phase 1, Dose Escalation Trial of RP-A601 in Subjects With PKP2… (NCT05885412) | Bored Studies