nctId: NCT06311708 id: TN-401-0011 briefTitle: 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) acronym: RIDGE overallStatus: RECRUITING date: 2023-01-31 date: 2030-03-29 date: 2030-07-11 date: 2024-03-15 date: 2024-03-15 name: Tenaya Therapeutics class: INDUSTRY name: University of California, San Francisco name: Brigham and Women's Hospital name: New York University name: University of Colorado, Denver name: Johns Hopkins University name: The Cleveland Clinic name: Mayo Clinic name: Medical University of South Carolina name: Hopital Louis Pradel name: Istituti Clinici Scientifici Maugeri SpA name: The Queen Elizabeth Hospital name: St George's University Hospitals NHS Foundation Trust name: Royal Brompton & Harefield NHS Foundation Trust name: Barts & The London NHS Trust briefSummary: 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 studyType: OBSERVATIONAL observationalModel: OTHER timePerspective: OTHER count: 200 type: ESTIMATED measure: Investigate the seroprevalence of pre-existing antibodies to AAV9 in patients with PKP2-associated ARVC measure: To characterize the burden of illness in patients with pathogenic or likely pathogenic PKP2 mutations measure: To characterize arrhythmic risk in patients with pathogenic or likely pathogenic PKP2 mutations measure: To evaluate functional status and Quality of Life (QoL) in patients with pathogenic or likely pathogenic PKP2 mutations measure: To evaluate heart function as assessed by imaging in patients with pathogenic or likely pathogenic PKP2 mutations measure: • To monitor biomarkers associated with disease progression and inflammation in patients with pathogenic or likely pathogenic PKP2 mutations measure: To evaluate genetics associated with PKP2-associated ARVC measure: To evaluate the effect of other cardiac mutations or other genetic variants that might affect the penetrance and/or expressivity of PKP2 mutations in patients with pathogenic or likely pathogenic PKP2 mutations measure: To evaluate health care utilization in patients with pathogenic or likely pathogenic PKP2 mutations sex: ALL minimumAge: 14 Years maximumAge: 65 Years stdAges: CHILD stdAges: ADULT stdAges: OLDER_ADULT facility: University of California San Francisco status: RECRUITING city: San Francisco state: California zip: 94143 country: United States name: Vasanth Vedantham, MD, PhD role: CONTACT lat: 37.77493 lon: -122.41942 facility: University of Colorado, Denver status: RECRUITING city: Aurora state: Colorado zip: 80045 country: United States name: Matthew Taylor, MD, PhD role: CONTACT lat: 39.72943 lon: -104.83192 facility: John Hopkins University School of Medicine status: RECRUITING city: Baltimore state: Maryland zip: 21287 country: United States name: Hugh G Calkins, MD role: CONTACT lat: 39.29038 lon: -76.61219 facility: Brigham and Women's Hospital status: RECRUITING city: Boston state: Massachusetts zip: 02115 country: United States name: Neal Lakdawala, MD role: CONTACT lat: 42.35843 lon: -71.05977 facility: Mayo Clinic status: RECRUITING city: Rochester state: Minnesota zip: 55905 country: United States name: John Giudicessi, MD PhD role: CONTACT lat: 44.02163 lon: -92.4699 facility: New York University status: RECRUITING city: New York state: New York zip: 10016 country: United States name: Larry Chinitz, MD role: CONTACT lat: 40.71427 lon: -74.00597 facility: Cleveland Clinic status: RECRUITING city: Cleveland state: Ohio zip: 44195 country: United States name: Wai Hong Wilson Tang, MD role: CONTACT lat: 41.4995 lon: -81.69541 facility: Medical University of South Carolina status: RECRUITING city: Charleston state: South Carolina zip: 29425 country: United States name: Daniel Judge, MD role: CONTACT lat: 32.77657 lon: -79.93092 facility: Hopital Louis Pradel status: RECRUITING city: Bron country: France name: Philippe Chevalier, MD, PhD role: CONTACT lat: 45.73333 lon: 4.91667 facility: Istituti Clinici Scientifici Maugeri SpA status: RECRUITING city: Pavia country: Italy name: Silvia Priori, MD, PhD role: CONTACT lat: 45.19205 lon: 9.15917 facility: The Queen Elizabeth Hospital status: RECRUITING city: Glasgow zip: G514TF country: United Kingdom name: Caroline Coats, MD role: CONTACT lat: 55.86515 lon: -4.25763 facility: Barts & The London Health NHS Trust status: RECRUITING city: London zip: E1 1BB country: United Kingdom name: Perry Elliott, MD role: CONTACT lat: 51.50853 lon: -0.12574 facility: St. George's University Hospitals NHS Foundation Trust status: RECRUITING city: London zip: SW17 0QT country: United Kingdom name: Elijah Behr, MD role: CONTACT lat: 51.50853 lon: -0.12574 facility: Royal Brompton & Harefield NHS Foundation Trust status: RECRUITING city: London zip: SW3 6NP country: United Kingdom name: Antonio Pantazis, MD role: CONTACT lat: 51.50853 lon: -0.12574 hasResults: False