Non-interventional Study of Seroprevalence of Pre-existing Antibodies Against Adenovirus-associated Virus Vector (AAV9) and the Progression of Disease in Patients With Plakophilin 2 (PKP2)-Associated Arrhythmogenic Right Ventricular Cardiomyopathy (ARVC)

Last updated: October 30, 2024
Sponsor: Tenaya Therapeutics
Overall Status: Active - Recruiting

Phase

N/A

Condition

Congestive Heart Failure

Cardiomyopathy

Heart Defect

Treatment

N/A

Clinical Study ID

NCT06311708
TN-401-0011
  • Ages 14-65
  • All Genders

Study Summary

This is a multicenter, non-interventional study to observe the natural progression of the disease and to study the prevalence of pre-existing antibodies to AAV9 used for gene therapy in a population of patients with PKP2 gene-associated ARVC. Participation from all patients is encouraged regardless of interest in or eligibility for gene therapy.

Eligibility Criteria

Inclusion

Inclusion Criteria:

  • Ages 14-65 years, inclusive, at the time of consent

  • Pathogenic or likely pathogenic PKP2 gene mutation

  • Diagnosed with ARVC and meet 2010 Modified Task Force Criteria for ARVC as affected.

  • Functioning ICD

Exclusion

Exclusion Criteria:

  • Currently receiving systemic immunosuppressive therapy, cytotoxic chemotherapy,immunoglobulin therapy or monoclonal antibody therapy

  • History of clinically significant liver disease, hepatitis B virus, hepatitis Cvirus, human immunodeficiency virus, or tuberculosis infection

  • Previously dosed with any investigational or approved gene therapy product at anytime

  • Concurrent participation in another interventional clinical trial unless approved bythe Sponsor. Participation in a noninterventional study may be allowed at theinvestigator's discretion.

  • History of cardiac transplant.

Study Design

Total Participants: 200
Study Start date:
January 31, 2023
Estimated Completion Date:
July 11, 2030

Study Description

Patients will receive standard of care treatments and assessments under the care of their healthcare provider. Biologic samples will be collected annually to measure cardiac and other related biomarkers. Clinical and observational data will be collected prospectively for up to 5 years from the date of enrollment, or until the patient withdraws consent/assent, undergoes heart transplantation, or dies.

If consent is provided, there may be a one-time sample collection to evaluate genetics for research purposes. Quality of Life (QoL) questionnaires will be used to assess a patient's wellbeing and quality of life. If not included as part of a patient's standard of care, diagnostic Holter (or equivalent) monitoring will be required annually. No investigational product will be administered. Participation from all patients is encouraged regardless of interest in or eligibility for gene therapy.

Connect with a study center

  • Hopital Louis Pradel

    Bron,
    France

    Active - Recruiting

  • Nantes University Hospital

    Nantes, 44000
    France

    Active - Recruiting

  • Pitié-Salpêtrière Hospital

    Paris, 75013
    France

    Active - Recruiting

  • Hôpital Haut-Lévêque

    Pessac, 33604
    France

    Active - Recruiting

  • Ludwig-Maximilians - University of Munich

    München, 80539
    Germany

    Site Not Available

  • University Hospital Muenster

    Münster, 48149
    Germany

    Active - Recruiting

  • Wuerzburg University Hospital

    Würzburg, 97080
    Germany

    Active - Recruiting

  • Centro Cardiologico Monzino

    Milano, 20138
    Italy

    Active - Recruiting

  • Istituti Clinici Scientifici Maugeri SpA

    Pavia,
    Italy

    Active - Recruiting

  • University Hospital, Linkoeping

    Linköping, 581 85
    Sweden

    Site Not Available

  • Skåne University Hospital

    Malmö, 214 28
    Sweden

    Active - Recruiting

  • The Queen Elizabeth Hospital

    Glasgow, G514TF
    United Kingdom

    Active - Recruiting

  • Barts & The London Health NHS Trust

    London, E1 1BB
    United Kingdom

    Active - Recruiting

  • Royal Brompton & Harefield NHS Foundation Trust

    London, SW3 6NP
    United Kingdom

    Active - Recruiting

  • St. George's University Hospitals NHS Foundation Trust

    London, SW17 0QT
    United Kingdom

    Active - Recruiting

  • University of California San Francisco

    San Francisco, California 94143
    United States

    Active - Recruiting

  • University of Colorado, Denver

    Aurora, Colorado 80045
    United States

    Active - Recruiting

  • John Hopkins University School of Medicine

    Baltimore, Maryland 21287
    United States

    Active - Recruiting

  • Brigham and Women's Hospital

    Boston, Massachusetts 02115
    United States

    Active - Recruiting

  • Mayo Clinic

    Rochester, Minnesota 55905
    United States

    Active - Recruiting

  • New York University

    New York, New York 10016
    United States

    Active - Recruiting

  • Cleveland Clinic

    Cleveland, Ohio 44195
    United States

    Active - Recruiting

  • Medical University of South Carolina

    Charleston, South Carolina 29425
    United States

    Active - Recruiting

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