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.
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.
Tenaya Therapeutics
Lead sponsor
University of California, San Francisco
Collaborator
Brigham and Women's Hospital
Collaborator
New York University
Collaborator
University of Colorado, Denver
Collaborator
Johns Hopkins University
Collaborator
The Cleveland Clinic
Collaborator
Mayo Clinic
Collaborator
Medical University of South Carolina
Collaborator
Hopital Louis Pradel
Collaborator
Istituti Clinici Scientifici Maugeri SpA
Collaborator
The Queen Elizabeth Hospital
Collaborator
St George's University Hospitals NHS Foundation Trust
Collaborator
Royal Brompton & Harefield NHS Foundation Trust
Collaborator
Barts & The London NHS Trust
Collaborator
Skane University Hospital
Collaborator
Centro Cardiologico Monzino
Collaborator
Hôpital Haut-Lévêque
Collaborator
Nantes University Hospital
Collaborator
Pitié-Salpêtrière Hospital
Collaborator
Wuerzburg University Hospital
Collaborator
University Hospital Muenster
Collaborator