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

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.

Conditions

PKP2 Arrhythmogenic Cardiomyopathy (PKP2-ACM)

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Study Overview

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Study Details

Study overview

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.

A Phase 1 Dose Escalation Trial Evaluating an Intravenously Administered Recombinant Adeno-Associated Virus Serotype rh.74 (AAVrh.74) Vector Containing the Human Plakophilin-2a (PKP2a) Coding Sequence (RP-A601; AAVrh.74-PKP2a) in Subjects With Arrhythmogenic Cardiomyopathy Arising From Pathogenic PKP2 Variants (PKP2-ACM)

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

Condition
PKP2 Arrhythmogenic Cardiomyopathy (PKP2-ACM)
Intervention / Treatment

-

Contacts and Locations

La Jolla

University of California, San Diego, La Jolla, California, United States, 92037

Durham

Duke University, Durham, North Carolina, United States, 27710

Philadelphia

Children's Hospital of Philadelphia, Philadelphia, Pennsylvania, United States, 19104

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

For general information about clinical research, read Learn About Studies.

Eligibility 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%
  • 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.

Ages Eligible for Study

18 Years to

Sexes Eligible for Study

ALL

Accepts Healthy Volunteers

No

Collaborators and Investigators

Rocket Pharmaceuticals Inc.,

Barry Greenberg, MD, PRINCIPAL_INVESTIGATOR, University of California, San Diego

Study Record Dates

2026-09