RecruitingNCT06311708

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)

Sponsor

Tenaya Therapeutics

Enrollment

200 participants

Start Date

Jan 31, 2023

Study Type

OBSERVATIONAL

Conditions

Arrhythmogenic Right Ventricular Cardiomyopathy

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

Min Age: 14 YearsMax Age: 65 Years

Plain Language Summary

Simplified for easier understanding

This study collects blood samples and disease data from patients with a specific hereditary heart condition called PKP2-associated arrhythmogenic right ventricular cardiomyopathy (ARVC) — a genetic disease where heart muscle is slowly replaced by fat and scar tissue, leading to dangerous irregular heartbeats. The goal is to understand disease progression and check for pre-existing antibodies that might affect future gene therapy eligibility. **You may be eligible if...** - You are between 14 and 65 years old - You carry a confirmed disease-causing mutation in the PKP2 gene - You meet the clinical diagnostic criteria for ARVC - You have an implanted defibrillator (ICD) for rhythm management **You may NOT be eligible if...** - You are currently on immune-suppressing medications or chemotherapy - You have significant liver disease, hepatitis B or C, HIV, or tuberculosis - You have previously received any gene therapy treatment - You have had a heart transplant Talk to your doctor to see if this trial is right for you.

This summary was AI-generated to explain the trial in plain language. It is not medical advice. Always discuss eligibility with your doctor before enrolling in a clinical trial.

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Locations(21)

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

Malmö, 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

Royal Brompton & Harefield NHS Foundation Trust

London, United Kingdom

View Full Details on ClinicalTrials.gov

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