RECRUITING

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

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Conditions

Arrhythmogenic Right Ventricular CardiomyopathyPKP2 Mutation Arrhythmogenic Right Ventricular Cardiomyopathy (ARVC)Arrhythmogenic Cardiomyopathy (ACM)PKP2-associated ARVCPKP2-ARVCPKP2-ACMAdeno Associated Virus (AAV)Gene TherapyGene TransferGenetic cardiomyopathyHeart Failure

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Study Overview

This clinical trial focuses on testing the efficacy of different digital interventions to promote re-engagement in cancer-related long-term follow-up care for adolescent and young adult (AYA) survivors of childhood cancer.

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.

Official Title

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)

Quick Facts

Study Start:2024-03-26
Study Completion:2029-10-01
Study Type:Not specified
Phase:Not Applicable
Enrollment:Not specified
Status:RECRUITING

Study ID

NCT06228924

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.

Ages Eligible for Study:18 Years to 65 Years
Sexes Eligible for Study:ALL
Accepts Healthy Volunteers:No
Standard Ages:ADULT, OLDER_ADULT
Inclusion CriteriaExclusion Criteria
  1. * PKP2 mutation (pathogenic or likely pathogenic)
  2. * Arrhythmogenic Right Ventricular Cardiomyopathy as defined by the 2010 revised Task Force Criteria
  3. * Left Ventricular Ejection Fraction ≥50%
  4. * Functioning Implantable Cardiac Defibrillator with remote integration capabilities at least 9 months prior to Screening
  5. * NYHA Functional Class I, II, or III
  6. * Frequent premature ventricular contractions (PVCs)
  1. * Ventricular tachycardia (VT) ablation within 6 months of Screening or planned VT ablation within 6 months after Screening
  2. * High AAV9 neutralizing antibody titer
  3. * Prior myocardial infarction
  4. * Right Ventricular Heart Failure
  5. * Class IV Heart Failure
  6. * Clinically significant renal disease
  7. * Clinically significant liver disease

Contacts and Locations

Study Contact

Matthew Pollman, M.D.
CONTACT
(650) 416-1186
mpollman@tenayathera.com
Niha Kamat
CONTACT
clinical.trials@tenayathera.com

Study Locations (Sites)

University of California San Francisco
San Francisco, California, 94143
United States
University of Colorado - Anschutz Medical Campus
Aurora, Colorado, 80045
United States
Johns Hopkins University
Baltimore, Maryland, 21287
United States
Brigham and Women's Hospital
Boston, Massachusetts, 02115
United States
Mayo Clinic
Rochester, Minnesota, 55905
United States
NYU Langone Health
New York, New York, 10016
United States
Cleveland Clinic
Cleveland, Ohio, 44195
United States

Collaborators and Investigators

Sponsor: Tenaya Therapeutics

Study Record Dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

Study Start Date2024-03-26
Study Completion Date2029-10-01

Study Record Updates

Study Start Date2024-03-26
Study Completion Date2029-10-01

Terms related to this study

Keywords Provided by Researchers

  • 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

Additional Relevant MeSH Terms

  • Arrhythmogenic Right Ventricular Cardiomyopathy