Seroprevalence Study of Pre-existing Antibodies Against Adenovirus-associated Virus Vector (AAV9) in Patients With Plakophilin 2 (PKP2)-Associated Arrhythmogenic Right Ventricular Cardiomyopathy (ARVC)
Status: Recruiting
Location: See all (21) locations...
Study Type: Observational
SUMMARY
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.
Eligibility
Participation Requirements
Sex: All
Minimum Age: 14
Maximum Age: 65
Healthy Volunteers: f
View:
• 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
Locations
United States
California
University of California San Francisco
RECRUITING
San Francisco
Colorado
University of Colorado, Denver
RECRUITING
Aurora
Massachusetts
Brigham and Women's Hospital
RECRUITING
Boston
Maryland
John Hopkins University School of Medicine
RECRUITING
Baltimore
Minnesota
Mayo Clinic
RECRUITING
Rochester
New York
New York University
RECRUITING
New York
Ohio
Cleveland Clinic
RECRUITING
Cleveland
South Carolina
Medical University of South Carolina
RECRUITING
Charleston
Other Locations
France
Hopital Louis Pradel
RECRUITING
Bron
Nantes University Hospital
RECRUITING
Nantes
Pitié-Salpêtrière Hospital
RECRUITING
Paris
Hôpital Haut-Lévêque
RECRUITING
Pessac
Germany
University Hospital Muenster
RECRUITING
Münster
Wuerzburg University Hospital
RECRUITING
Würzburg
Italy
Centro Cardiologico Monzino
RECRUITING
Milan
Istituti Clinici Scientifici Maugeri SpA
RECRUITING
Pavia
Sweden
Skåne University Hospital
RECRUITING
Malmo
United Kingdom
The Queen Elizabeth Hospital
RECRUITING
Glasgow
Barts & The London Health NHS Trust
RECRUITING
London
Royal Brompton & Harefield NHS Foundation Trust
RECRUITING
London
St. George's University Hospitals NHS Foundation Trust
RECRUITING
London
Contact Information
Primary
Matthew Pollman, MD
mpollman@tenayathera.com
650-209-8092
Backup
Niharika Kamat, MS
clinical.trials@tenayathera.com
Time Frame
Start Date: 2023-01-31
Estimated Completion Date: 2030-07-11
Participants
Target number of participants: 200
Treatments
Retrospective and Prospective
Patients who meet the eligibility criteria are observed and data collected both prospectively and retrospectively.
Related Therapeutic Areas
Sponsors
Collaborators: St George's University Hospitals NHS Foundation Trust, Mayo Clinic, Medical University of South Carolina, Royal Brompton & Harefield NHS Foundation Trust, Brigham and Women's Hospital, Barts & The London NHS Trust, Hôpital Haut-Lévêque, Nantes University Hospital, The Cleveland Clinic, The Queen Elizabeth Hospital, Hopital Louis Pradel, New York University, Skane University Hospital, Wuerzburg University Hospital, Pitié-Salpêtrière Hospital, University Hospital Muenster, Johns Hopkins University, University of Colorado, Denver, University of California, San Francisco, Centro Cardiologico Monzino, Istituti Clinici Scientifici Maugeri SpA
Leads: Tenaya Therapeutics