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

Description

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

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Study Overview

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Study Details

Study overview

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.

First-in-Human, Open-Label, Safety, Tolerability, Dose-Finding, Pharmacodynamic and Cardiac Transgene Expression Study of TN-401, a Recombinant Adeno-associated Virus Serotype 9 (AAV9) Containing Plakophilin-2 (PKP2) Transgene, in Adults With PKP2 Mutation-Associated Arrhythmogenic Right Ventricular Cardiomyopathy (ARVC)

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

Condition
Arrhythmogenic Right Ventricular Cardiomyopathy
Intervention / Treatment

-

Contacts and Locations

San Francisco

University of California San Francisco, San Francisco, California, United States, 94143

Aurora

University of Colorado - Anschutz Medical Campus, Aurora, Colorado, United States, 80045

Baltimore

Johns Hopkins University, Baltimore, Maryland, United States, 21287

Boston

Brigham and Women's Hospital, Boston, Massachusetts, United States, 02115

Rochester

Mayo Clinic, Rochester, Minnesota, United States, 55905

New York

NYU Langone Health, New York, New York, United States, 10016

Cleveland

Cleveland Clinic, Cleveland, Ohio, United States, 44195

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

For general information about clinical research, read Learn About Studies.

Eligibility 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)
  • * 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

Ages Eligible for Study

18 Years to 65 Years

Sexes Eligible for Study

ALL

Accepts Healthy Volunteers

No

Collaborators and Investigators

Tenaya Therapeutics,

Study Record Dates

2029-10-01