Open-label, Dose Escalation Study of Safety and Preliminary Efficacy of TN-401 in Adults With PKP2 Mutation-associated ARVC

NCT06228924 · tracked via the Priya Life Science USA tracker

Sponsor

Tenaya Therapeutics

Phase

Phase 1

Started

2024-03-26

Last updated

2025-02-06

Condition(s) studied

Arrhythmogenic Right Ventricular Cardiomyopathy

Investigational drug(s) / intervention(s)

TN-401

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.

Study 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.

Eligibility

Sex

ALL

Min age

18 Years

Max age

65 Years

Healthy volunteers

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

Primary outcome measure(s)

Number and severity of Adverse Events over the course of the study. — 52 weeks
Number of Serious Adverse Events related to study drug. — 5 years

Trial sites (7)

Facility	City	Region	Status
University of California San Francisco	San Francisco	California	Recruiting
University of Colorado - Anschutz Medical Campus	Aurora	Colorado	Recruiting
Johns Hopkins University	Baltimore	Maryland	Recruiting
Brigham and Women's Hospital	Boston	Massachusetts	Recruiting
Mayo Clinic	Rochester	Minnesota	Recruiting
NYU Langone Health	New York	New York	Recruiting
Cleveland Clinic	Cleveland	Ohio	Recruiting

More Tenaya Therapeutics trials in the USA

Natural History Study in Pediatric Patients With MYBPC3 Mutation-associated Cardiomyopathy Active, not recruiting

Non-interventional Study of Seroprevalence of Pre-existing Antibodies Against Adenovirus-associated Virus Vector (AAV9) and the Progression of Disease in Patients With Plakophilin 2 (PKP2)-Associated Arrhythmogenic Right Ventricular Cardiomyopathy (ARVC) Recruiting

Multi-center, Open-label, Single-ascending Dose Study of Safety and Tolerability of TN-201 in Adults With Symptomatic MYBPC3 Mutation-associated HCM Recruiting

Other trials for the same condition

Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT06228924 on ClinicalTrials.gov ↗ ← All trials in the USA