Open-label, Dose Escalation Study of Safety and Preliminary Efficacy of TN-401 in Adults With PKP2 Mutation-associated ARVC
Start Date
3/26/2024
Completion Date
10/1/2029
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.
Detailed Description
The RIDGE-1™ open-label Safety, Tolerability, Dose-finding, PD and Cardiac Transgene Expression Study will enroll up to 15 patients in two planned dose cohorts. Patients in each cohort will receive a single intravenous (IV) dose of TN-401. Following Data Safety Monitoring Board (DSMB) review of each dose cohort, the next dose cohort will be initiated. DSMB review will also be needed to expand the dose cohorts. The dose for Cohorts 1/1a will be 3E13 (3 × 1013) vg/kg and the dose for Cohorts 2/2a will be 6E13 (6 × 1013) vg/kg.
Eligibility Criteria
Age Range: 18 years to 65 years
Interventions
TN-401
Conditions
Locations
University of California San Francisco
San Francisco, California 94143
United States
University of Colorado - Anschutz Medical Campus
Aurora, Colorado 80045
United States
Johns Hopkins University
Baltimore, Maryland 21287
United States
Brigham and Women's Hospital
Boston, Massachusetts 02115
United States
Mayo Clinic
Rochester, Minnesota 55905
United States
NYU Langone Health
New York, New York 10016
United States
Cleveland Clinic
Cleveland, Ohio 44195
United States