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NCT Number: NCT05885412

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

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.

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Key information

Conditions

PKP2 Arrhythmogenic Cardiomyopathy (PKP2-ACM) Arrhythmogenic Right Ventricular Dysplasia Cardiomyopathies Cardiovascular Abnormalities Cardiovascular Diseases Congenital Abnormalities Congenital, Hereditary, and Neonatal Diseases and Abnormalities Death Death, Sudden Death, Sudden, Cardiac Heart Arrest Heart Defects, Congenital Heart Diseases Pathologic Processes Pathological Conditions, Signs and Symptoms

Age range

18 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 1

Primary location

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

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Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

RP-A601

Genetic

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)

Primary outcomes

  1. Evaluation of safety associated with RP-A601

    Time frame: 12 months post-infusion

    Incidence of treatment emergent adverse events (TEAEs), incidence of Serious Adverse Events (SAEs), and identification of Dose Limiting Toxicities (DLTs)

Secondary outcomes

  1. Preliminary efficacy of RP-A601 - Myocardial PKP2 protein expression

    Time frame: 12 months post-infusion

    Assessment of changes in myocardial PKP2 protein expression

  2. Preliminary efficacy of RP-A601 - Ventricular ectopy and arrhythmia

    Time frame: 12 months post-infusion

    Assessment of changes in levels of ventricular ectopy and arrhythmia on cardiac rhythm monitoring

  3. Preliminary efficacy of RP-A601 - Cardiac biomarkers

    Time frame: 12 months post-infusion

    Assessment of changes in circulating levels of cardiac biomarkers of injury and stress

Study contacts

Contact information is provided by the study sponsor or research team.

Clinical Information

CONTACT

[email protected]

646-627-0033

Sponsors and collaborators

Lead sponsor

Rocket Pharmaceuticals Inc.

Industry

Registry information

Official study 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)

Important dates

Study start
2023
Primary completion
2028
Study completion
2029
First posted
Jun 2, 2023
Registry last updated
Apr 16, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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