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Clinical Trials in the USA / NCT06228924
Recruiting Phase 1

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
No
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)

Trial sites (7)

FacilityCityRegionStatus
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
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