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Recruiting Phase 1

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

NCT05885412 · tracked via the Priya Life Science USA tracker
Phase
Phase 1
Started
2023-08-29
Last updated
2026-04-16

Condition(s) studied

PKP2 Arrhythmogenic Cardiomyopathy (PKP2-ACM)

Investigational drug(s) / intervention(s)

RP-A601

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)

Study summary

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.

Eligibility

Sex
ALL
Min age
18 Years
Max age
—
Healthy volunteers
No
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.

Primary outcome measure(s)

  • Evaluation of safety associated with RP-A601 — 12 months post-infusion
    Incidence of treatment emergent adverse events (TEAEs), incidence of Serious Adverse Events (SAEs), and identification of Dose Limiting Toxicities (DLTs)

Trial sites (3)

FacilityCityRegionStatus
University of California, San Diego La Jolla California Recruiting
Duke University Durham North Carolina Recruiting
Children's Hospital of Philadelphia Philadelphia Pennsylvania Withdrawn
Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT05885412 on ClinicalTrials.gov ↗ ← All trials in the USA