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RECRUITINGOBSERVATIONAL

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)

Seroprevalence Study of Pre-existing Antibodies Against Adenovirus-associated Virus Vector (AAV9) in Patients With Plakophilin 2 (PKP2)-Associated Arrhythmogenic Right Ventricular Cardiomyopathy (ARVC)

Important: This information is not medical advice. Talk to your doctor about whether a clinical trial is right for you.

About This Trial

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 May Be Eligible (Plain English)

Who May Qualify: - 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 Who Should NOT Join This Trial: - 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 weakened immune system 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. Always talk to your doctor about whether this trial is right for you.

Original Eligibility Criteria

View original clinical language
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.

Locations (20)

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
Nantes University Hospital
Nantes, France
Pitié-Salpêtrière Hospital
Paris, France
Hôpital Haut-Lévêque
Pessac, France
University Hospital Muenster
Münster, Germany
Wuerzburg University Hospital
Würzburg, Germany
Centro Cardiologico Monzino
Milan, Italy
Istituti Clinici Scientifici Maugeri SpA
Pavia, Italy
Skåne University Hospital
Malmo, Sweden
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