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Open-label, Dose Escalation Study of Safety and Preliminary Efficacy of TN-401 in Adults With PKP2 Mutation-associated ARVC
Sponsor: Tenaya Therapeutics
Summary
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.
Official title: 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)
Key Details
Gender
All
Age Range
18 Years - 65 Years
Study Type
INTERVENTIONAL
Enrollment
15
Start Date
2024-03-26
Completion Date
2029-10-01
Last Updated
2025-02-06
Healthy Volunteers
No
Interventions
TN-401
TN-401 is a recombinant adeno-associated virus serotype 9 (AAV9) gene therapy containing Plakophilin-2 (PKP2) transgene. It is a single (one-time) intravenous dose.
Locations (7)
University of California San Francisco
San Francisco, California, United States
University of Colorado - Anschutz Medical Campus
Aurora, Colorado, United States
Johns Hopkins University
Baltimore, Maryland, United States
Brigham and Women's Hospital
Boston, Massachusetts, United States
Mayo Clinic
Rochester, Minnesota, United States
NYU Langone Health
New York, New York, United States
Cleveland Clinic
Cleveland, Ohio, United States