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)

Description

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.

Conditions

Arrhythmogenic Right Ventricular Cardiomyopathy

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Study Overview

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Study Details

Study overview

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.

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

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)

Condition
Arrhythmogenic Right Ventricular Cardiomyopathy
Intervention / Treatment

-

Contacts and Locations

San Francisco

University of California San Francisco, San Francisco, California, United States, 94143

Aurora

University of Colorado, Denver, Aurora, Colorado, United States, 80045

Baltimore

John Hopkins University School of Medicine, Baltimore, Maryland, United States, 21287

Boston

Brigham and Women's Hospital, Boston, Massachusetts, United States, 02115

Rochester

Mayo Clinic, Rochester, Minnesota, United States, 55905

New York

New York University, New York, New York, United States, 10016

Cleveland

Cleveland Clinic, Cleveland, Ohio, United States, 44195

Charleston

Medical University of South Carolina, Charleston, South Carolina, United States, 29425

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

  • * 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
  • * Currently receiving systemic immunosuppressive therapy, cytotoxic chemotherapy, immunoglobulin therapy or monoclonal antibody therapy
  • * History of clinically significant liver disease, hepatitis B virus, hepatitis C virus, human immunodeficiency virus, or tuberculosis infection
  • * Previously dosed with any investigational or approved gene therapy product at any time
  • * Concurrent participation in another interventional clinical trial unless approved by the Sponsor. Participation in a noninterventional study may be allowed at the investigator's discretion.
  • * History of cardiac transplant.

Ages Eligible for Study

14 Years to 65 Years

Sexes Eligible for Study

ALL

Accepts Healthy Volunteers

No

Collaborators and Investigators

Tenaya Therapeutics,

Study Record Dates

2030-07-11