RECRUITING

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)

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Conditions

Arrhythmogenic Right Ventricular CardiomyopathyPKP2 Mutation Arrhythmogenic Right Ventricular Cardiomyopathy (ARVC)Arrhythmogenic Cardiomyopathy (ACM)PKP2-associated ARVCPKP2-ARVCPKP2-ACMAdeno Associated Virus (AAV)Adeno-Associated Virus Serotype 9 (AAV9)Gene TherapyGene TransferGenetic cardiomyopathyHeart Failure

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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 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.

Official Title

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

Quick Facts

Study Start:2023-01-31
Study Completion:2030-07-11
Study Type:Not specified
Phase:Not Applicable
Enrollment:Not specified
Status:RECRUITING

Study ID

NCT06311708

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:14 Years to 65 Years
Sexes Eligible for Study:ALL
Accepts Healthy Volunteers:No
Standard Ages:CHILD, ADULT, OLDER_ADULT
Inclusion CriteriaExclusion Criteria
  1. * Ages 14-65 years, inclusive, at the time of consent
  2. * Pathogenic or likely pathogenic PKP2 gene mutation
  3. * Diagnosed with ARVC and meet 2010 Modified Task Force Criteria for ARVC as affected.
  4. * Functioning ICD
  1. * Currently receiving systemic immunosuppressive therapy, cytotoxic chemotherapy, immunoglobulin therapy or monoclonal antibody therapy
  2. * History of clinically significant liver disease, hepatitis B virus, hepatitis C virus, human immunodeficiency virus, or tuberculosis infection
  3. * Previously dosed with any investigational or approved gene therapy product at any time
  4. * 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.
  5. * History of cardiac transplant.

Contacts and Locations

Study Contact

Matthew Pollman, MD
CONTACT
650-209-8092
mpollman@tenayathera.com
Niharika Kamat, MS
CONTACT
clinical.trials@tenayathera.com

Study Locations (Sites)

University of California San Francisco
San Francisco, California, 94143
United States
University of Colorado, Denver
Aurora, Colorado, 80045
United States
John Hopkins University School of Medicine
Baltimore, Maryland, 21287
United States
Brigham and Women's Hospital
Boston, Massachusetts, 02115
United States
Mayo Clinic
Rochester, Minnesota, 55905
United States
New York University
New York, New York, 10016
United States
Cleveland Clinic
Cleveland, Ohio, 44195
United States
Medical University of South Carolina
Charleston, South Carolina, 29425
United States

Collaborators and Investigators

Sponsor: Tenaya Therapeutics

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 Date2023-01-31
Study Completion Date2030-07-11

Study Record Updates

Study Start Date2023-01-31
Study Completion Date2030-07-11

Terms related to this study

Keywords Provided by Researchers

  • PKP2 Mutation Arrhythmogenic Right Ventricular Cardiomyopathy (ARVC)
  • Arrhythmogenic Cardiomyopathy (ACM)
  • PKP2-associated ARVC
  • PKP2-ARVC
  • PKP2-ACM
  • Adeno Associated Virus (AAV)
  • Adeno-Associated Virus Serotype 9 (AAV9)
  • Gene Therapy
  • Gene Transfer
  • Genetic cardiomyopathy
  • Heart Failure

Additional Relevant MeSH Terms

  • Arrhythmogenic Right Ventricular Cardiomyopathy