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)

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

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.

Conditions2

Locations8 sites

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