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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)
NCT06311708 · Tenaya Therapeutics
In plain English
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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)
About this study
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.
Eligibility criteria
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 Criteria:
* 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.
Study design
Enrollment target: 200 participants
Age groups: child, adult, older_adult
Timeline
Starts: 2023-01-31
Estimated completion: 2030-07-11
Last updated: 2024-11-01
Primary outcomes
- • Investigate the seroprevalence of pre-existing antibodies to AAV9 in patients with PKP2-associated ARVC (5 years)
Sponsor
Tenaya Therapeutics · industry
With: University of California, San Francisco, Brigham and Women's Hospital, New York University, University of Colorado, Denver, Johns Hopkins University, The Cleveland Clinic, Mayo Clinic, Medical University of South Carolina, Hopital Louis Pradel, Istituti Clinici Scientifici Maugeri SpA, The Queen Elizabeth Hospital, St George's University Hospitals NHS Foundation Trust, Royal Brompton & Harefield NHS Foundation Trust, Barts & The London NHS Trust, Skane University Hospital, Centro Cardiologico Monzino, Hôpital Haut-Lévêque, Nantes University Hospital, Pitié-Salpêtrière Hospital, Wuerzburg University Hospital, University Hospital Muenster
Contacts & investigators
ContactMatthew Pollman, MD · contact · mpollman@tenayathera.com · 650-209-8092
ContactNiharika Kamat, MS · contact · clinical.trials@tenayathera.com
All locations (21)
University of California San FranciscoRecruiting
San Francisco, California, United States
University of Colorado, DenverRecruiting
Aurora, Colorado, United States
John Hopkins University School of MedicineRecruiting
Baltimore, Maryland, United States
Brigham and Women's HospitalRecruiting
Boston, Massachusetts, United States
Mayo ClinicRecruiting
Rochester, Minnesota, United States
New York UniversityRecruiting
New York, New York, United States
Cleveland ClinicRecruiting
Cleveland, Ohio, United States
Medical University of South CarolinaRecruiting
Charleston, South Carolina, United States
Hopital Louis PradelRecruiting
Bron, France
Nantes University HospitalRecruiting
Nantes, France
Pitié-Salpêtrière HospitalRecruiting
Paris, France
Hôpital Haut-LévêqueRecruiting
Pessac, France
University Hospital MuensterRecruiting
Münster, Germany
Wuerzburg University HospitalRecruiting
Würzburg, Germany
Centro Cardiologico MonzinoRecruiting
Milan, Italy
Istituti Clinici Scientifici Maugeri SpARecruiting
Pavia, Italy
Skåne University HospitalRecruiting
Malmo, Sweden
The Queen Elizabeth HospitalRecruiting
Glasgow, United Kingdom
Barts & The London Health NHS TrustRecruiting
London, United Kingdom
St. George's University Hospitals NHS Foundation TrustRecruiting
London, United Kingdom
Royal Brompton & Harefield NHS Foundation TrustRecruiting
London, United Kingdom