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This non-interventional (observational) study looks at how common pre-existing antibodies against the AAV9 (adeno-associated virus) vector—used to carry gene therapies—are, and how the disease progresses in patients with PKP2 gene–associated arrhythmogenic right ventricular cardiomyopathy (ARVC). It enrolls people aged 14-65 who carry a disease-causing PKP2 mutation, have an ARVC diagnosis, and have a functioning ICD (heart device). It is an observational study with no assigned phase and is currently recruiting participants.
The summary above is a plain-language rendering of the official record. The original English title is shown for reference.
Criteria are reproduced from the ClinicalTrials.gov record in the original English. Only the trial team can determine 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.