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Displaying 3 studies
The purpose of this multicenter, seroprevalence study to assess pre-existing antibodies to Adenovirus-associated Virus Vector (AAV) in a population of patients with Plakophilin 2 (PKP2)-associated Arrhythmogenic Right Ventricular Cardiomyopathy (ARVC), who may be eligible for subsequent gene therapy trials. Biological samples and clinical data will be collected over a period of up to 5 years from the date of enrollment. No investigational product will be administered.
The purpose of this study is to evaluate the safety, tolerability, and pharmacodynamics (PD) of TN-401 in adult patients with symptomatic PKP2 mutation-associated ARVC. The trial will consist of 2 escalating dose cohorts. All patients will receive active drug.
The purpose of this study is to identify hereditary causes of cardiomyopathy.