Recruiting
Phase 1

TN-401

Sponsor:

Tenaya Therapeutics

Code:

NCT06228924

Conditions

Arrhythmogenic Right Ventricular Cardiomyopathy

Eligibility Criteria

Sex: All

Age: 18 - 65

Healthy Volunteers: Not accepted

Interventions

TN-401

Study Details

Brief 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.

Conditions

Arrhythmogenic Right Ventricular Cardiomyopathy

Study ID

NCT06228924

Start date

Mar 26, 2024

Status verified date

Sep, 2024

Completion date

Oct 1, 2029

Anticipated

Primary completion date

May 1, 2026

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 18 - 65

Healthy Volunteers: Not accepted

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 at least 9 months prior to Screening
  • NYHA Functional Class I, II, or III
  • Frequent premature ventricular contractions (PVCs)

Exclusion Criteria:

  • Ventricular tachycardia (VT) ablation within 6 months of Screening or planned VT ablation within 6 months after Screening
  • High AAV9 neutralizing antibody titer
  • Prior myocardial infarction
  • Right Ventricular Heart Failure
  • Class IV Heart Failure
  • Clinically significant renal disease
  • Clinically significant liver disease

Study Design

Enrollment

15 participants

Anticipated

Allocation

Non randomized

Intervention Model

Sequential

Primary purpose

Treatment

Interventions and Outcome Measures

Arms

experimental: Cohort 1

Dose for Cohort 1 will be 3E13 vg/kg

experimental: Cohort 2

Dose for Cohort 2 will be 6E13 vg/kg

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.

Primary outcome measure

  • Number and severity of Adverse Events over the course of the study. [ Time Frame: 52 weeks ]
  • Number of Serious Adverse Events related to study drug. [ Time Frame: 5 years ]

Central Contacts and Locations

Central contacts

Matthew Pollman, M.D.

(650) 416-1186mpollman@tenayathera.com

Niha Kamat

clinical.trials@tenayathera.com

Locations

University of California San Francisco

Recruiting

San Francisco, California, United States, 94143

University of Colorado - Anschutz Medical Campus

Recruiting

Aurora, Colorado, United States, 80045

Johns Hopkins University

Recruiting

Baltimore, Maryland, United States, 21287

Principal Investigator:

Andreas Barth, MD

Brigham and Women's Hospital

Recruiting

Boston, Massachusetts, United States, 02115

Mayo Clinic

Recruiting

Rochester, Minnesota, United States, 55905

Principal Investigator:

John R. Giudicessi, MD

NYU Langone Health

Recruiting

New York, New York, United States, 10016

Cleveland Clinic

Recruiting

Cleveland, Ohio, United States, 44195

More Information

Sponsor

Tenaya Therapeutics

Last update posted

Feb 6, 2025

Last verified

Sep, 2024

Keywords

  • PKP2 Mutation Arrhythmogenic Right Ventricular Cardiomyopathy (ARVC)
  • Arrhythmogenic Cardiomyopathy (ACM)
  • PKP2-associated ARVC
  • PKP2-ARVC
  • PKP2-ACM
  • Adeno Associated Virus (AAV)
  • Gene Therapy
  • Gene Transfer
  • Genetic cardiomyopathy
  • Heart Failure

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-10. This information was provided to ClinicalTrials.gov by Tenaya Therapeutics on 2025-02-06.