RECRUITING

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

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Conditions

PKP2 Arrhythmogenic Cardiomyopathy (PKP2-ACM)Arrhythmogenic cardiomyopathyArrhythmogenic Right Ventricular CardiomyopathyArrhythmogenic Right Ventricular DysplasiaSudden Cardiac DeathGenetic cardiomyopathyGene therapyPKP2ARVCARVDACMCardiac ArrestVentricular Arrhythmia

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Study Overview

This clinical trial focuses on testing the efficacy of different digital interventions to promote re-engagement in cancer-related long-term follow-up care for adolescent and young adult (AYA) survivors of childhood cancer.

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.

Official Title

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)

Quick Facts

Study Start:2023-08-29
Study Completion:2026-09
Study Type:Not specified
Phase:Not Applicable
Enrollment:Not specified
Status:RECRUITING

Study ID

NCT05885412

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.

Ages Eligible for Study:18 Years
Sexes Eligible for Study:ALL
Accepts Healthy Volunteers:No
Standard Ages:ADULT, OLDER_ADULT
Inclusion CriteriaExclusion Criteria
  1. 1. Male or female ≥18 years at the time of signing the informed consent
  2. 2. Capable and willing to provide signed informed consent
  3. 3. Clinical diagnosis of ACM as defined by the 2010 revised Task Force Criteria (TFC)
  4. 4. Documentation of a pathogenic or likely pathogenic truncating variant in PKP2
  5. 5. History of Implantable Cardioverter-Defibrillator (ICD) implantation ≥6 months prior to enrollment
  6. 6. PVC frequency ≥500 per 24 hours by ambulatory rhythm monitoring
  7. 7. Left ventricular ejection fraction by echocardiogram or CMR ≥50%
  1. 1. Anti-AAVrh.74 capsid neutralizing antibody titer of \>1:40
  2. 2. Cardiomyopathy related to a genetic etiology other than PKP2 truncating variant
  3. 3. Previous participation in a study of gene transfer or gene editing
  4. 4. Severe Right ventricular (RV) dysfunction
  5. 5. New York Heart Association (NYHA) Class IV heart failure.

Contacts and Locations

Study Contact

Clinical Information
CONTACT
646-627-0033
clinicaltrials@rocketpharma.com

Principal Investigator

Barry Greenberg, MD
PRINCIPAL_INVESTIGATOR
University of California, San Diego

Study Locations (Sites)

University of California, San Diego
La Jolla, California, 92037
United States
Duke University
Durham, North Carolina, 27710
United States
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104
United States

Collaborators and Investigators

Sponsor: Rocket Pharmaceuticals Inc.

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

Study Record Dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

Study Start Date2023-08-29
Study Completion Date2026-09

Study Record Updates

Study Start Date2023-08-29
Study Completion Date2026-09

Terms related to this study

Keywords Provided by Researchers

  • Arrhythmogenic cardiomyopathy
  • Arrhythmogenic Right Ventricular Cardiomyopathy
  • Arrhythmogenic Right Ventricular Dysplasia
  • Sudden Cardiac Death
  • Genetic cardiomyopathy
  • Gene therapy
  • PKP2
  • ARVC
  • ARVD
  • ACM
  • Cardiac Arrest
  • Ventricular Arrhythmia

Additional Relevant MeSH Terms

  • PKP2 Arrhythmogenic Cardiomyopathy (PKP2-ACM)