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Clinical Trial 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.


Clinical Trial 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. ;


Study Design


Related Conditions & MeSH terms

  • Arrhythmogenic Right Ventricular Cardiomyopathy
  • Arrhythmogenic Right Ventricular Dysplasia
  • Cardiomyopathies

NCT number NCT06228924
Study type Interventional
Source Tenaya Therapeutics
Contact Matthew Pollman, M.D.
Phone (650) 416-1186
Email mpollman@tenayathera.com
Status Recruiting
Phase Phase 1
Start date March 26, 2024
Completion date October 1, 2029

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