Safety and Dose-Finding Study of TN-401 Gene Therapy for Adults with PKP2 Mutation-Related ARVC

What this Trial Is About

Researchers are evaluating the safety and early effects of TN-401 gene therapy in adults with symptomatic arrhythmogenic right ventricular cardiomyopathy ARVC caused by PKP2 gene mutations. This first-in-human study focuses on how well this gene therapy is tolerated and its preliminary impact on the condition. The study is in its initial phase and aims to understand both safety and gene expression in the heart. Participants are divided into two groups receiving different doses of TN-401, a one-time intravenous gene therapy using a recombinant adeno-associated virus containing the PKP2 gene. The first group receives a dose of 3 x 1013 vector genomes per kilogram, and the second group receives 6 x 1013 vector genomes per kilogram. After safety reviews by an independent board, dosing for each group may be expanded. During the study, participants will be monitored for side effects and serious adverse events over one to five years. Researchers will track daily heart rhythm irregularities, frequency of implantable defibrillator therapies, and sustained ventricular tachycardia episodes over 52 weeks. The study involves regular assessments to check heart function, device performance, and gene therapy effects, ensuring close safety and treatment monitoring throughout the participation period.

Open-label, Dose Escalation Study of Safety and Preliminary Efficacy of TN-401 in Adults With PKP2 Mutation-associated ARVC