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)
Recruiting · NCT06311708 · Observational (researchers observe without assigning treatment) · Lead sponsor: Tenaya Therapeutics
View the official record on ClinicalTrials.gov →What this trial is about
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.
Who can take part
Age range
14 Years to 65 Years
Sex
All (male and female)
Healthy volunteers
No - a diagnosis or condition is required
Phase
Not specified
Study type
Observational (researchers observe without assigning treatment)
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.
Where it is running
21 locations listed across 8 US states.
- University of California San Francisco - San Francisco, California, United States
- University of Colorado, Denver - Aurora, Colorado, United States
- John Hopkins University School of Medicine - Baltimore, Maryland, United States
- Brigham and Women's Hospital - Boston, Massachusetts, United States
- Mayo Clinic - Rochester, Minnesota, United States
- New York University - New York, New York, United States
- Cleveland Clinic - Cleveland, Ohio, United States
- Medical University of South Carolina - Charleston, South Carolina, United States
- Hopital Louis Pradel - Bron, France
- The Queen Elizabeth Hospital - Glasgow, United Kingdom
- Barts & The London Health NHS Trust - London, United Kingdom
- St. George's University Hospitals NHS Foundation Trust - London, United Kingdom
- Royal Brompton & Harefield NHS Foundation Trust - London, United Kingdom
- Skåne University Hospital - Malmö, Sweden
- Centro Cardiologico Monzino - Milan, Italy
- University Hospital Muenster - Münster, Germany
- Nantes University Hospital - Nantes, France
- Pitié-Salpêtrière Hospital - Paris, France
- Istituti Clinici Scientifici Maugeri SpA - Pavia, Italy
- Hôpital Haut-Lévêque - Pessac, France
- Wuerzburg University Hospital - Würzburg, Germany