First-in-Human, Open-Label, Safety, Tolerability, Dose-Finding, Pharmacodynamic and Cardiac Transgene Expression Study of TN-401, a Recombinant Adeno-associated Virus Serotype 9 (AAV9) Containing Plakophilin-2 (PKP2) Transgene, in Adults With PKP2 Mutation-Associated Arrhythmogenic Right Ventricular Cardiomyopathy (ARVC)

TN-401-0012

This first-in-human study is designed to evaluate the safety, and preliminary efficacy (PD) of TN-401 gene therapy in adult patients with symptomatic PKP2 mutation-associated ARVC.

2024-04-01

2026-05-01

2029-10-01

Recruiting

Early phase I

15

Inclusion Criteria: * PKP2 mutation (pathogenic or likely pathogenic) * Arrhythmogenic Right Ventricular Cardiomyopathy as defined by the 2010 revised Task Force Criteria * Left Ventricular Ejection Fraction ≥50% * Functioning Implantable Cardiac Defibrillator with remote integration capabilities ≥12 months prior to enrollment * NYHA Functional Class I, II, or III * Frequent premature ventricular contractions (PVCs) Exclusion Criteria: * High AAV9 neutralizing antibody titer * Prior myocardial infarction * Right Ventricular Heart Failure * Class IV Heart Failure * Clinically significant renal disease * Clinically significant liver disease

{'studyType': 'INTERVENTIONAL', 'phases': ['PHASE1'], 'designInfo': {'allocation': 'NON_RANDOMIZED', 'interventionModel': 'SEQUENTIAL', 'primaryPurpose': 'TREATMENT', 'maskingInfo': {'masking': 'NONE'}}, 'enrollmentInfo': {'count': 15, 'type': 'ESTIMATED'}}

2024-03-29