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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)

RecruitingObservational study

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

Who can join

Ages 14 to 65 · All sexes

Full eligibility criteria
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.

About the study

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.

Sponsor: Tenaya Therapeutics · Participants: 200 · Started: Jan 31, 2023

Contact the study team

Official record on ClinicalTrials.gov — NCT06311708

Locations in the U.S.

CaliforniaUniversity of California San Francisco, San Francisco
ColoradoUniversity of Colorado, Denver, Aurora
MarylandJohn Hopkins University School of Medicine, Baltimore
MassachusettsBrigham and Women's Hospital, Boston
MinnesotaMayo Clinic, Rochester
New YorkNew York University, New York
OhioCleveland Clinic, Cleveland
South CarolinaMedical University of South Carolina, Charleston

Conditions

From ClinicalTrials.gov, data retrieved Sep 29, 2026. Each study sets its own eligibility; the study team decides who can join.