TN-401-0011
Research type
Research Study
Full title
Seroprevalence Study of Pre-existing Antibodies Against Adenovirus-associated Virus Vector (AAV) in Patients with Plakophilin 2 (PKP2)-associated Arrhythmogenic Right Ventricular Cardiomyopathy (ARVC).
IRAS ID
323224
Contact name
Elijah Behr
Contact email
Sponsor organisation
Tenaya Therapeutics, Inc.
Duration of Study in the UK
5 years, 0 months, 0 days
Research summary
The main purpose of this study is to collect information about patients with Arrhythmogenic Right Ventricular Cardiomyopathy (ARVC) to learn more about their disease and pre-existing antibodies to adeno-associated virus (AAV). Having preexisting antibodies to AAV may change how gene therapies that use AAV work in patients with PKP2-associated ARVC. Information gained from this study will help future development of gene therapies and provide a more thorough understanding of the disease course and how to manage it.
Participants between 14 to 65 years of age and with confirmed mutations in PKP2 gene with a diagnosis of ARVC will be eligible for this study.
Each participant will be followed for up to 5 years from their date of enrollment.
The study will be conducted globally in approximately 30 study centers located in North America and Europe.
The study is expected to enroll up to approximately 200 patients.
Study participation is entirely voluntary and information describing the study will be provided.
Consent/assent will be obtained from each participant before any study procedures are performed.
REC name
East Midlands - Nottingham 2 Research Ethics Committee
REC reference
23/EM/0068
Date of REC Opinion
12 Apr 2023
REC opinion
Further Information Favourable Opinion