RecruitingPhase 1NCT06228924

Open-label, Dose Escalation Study of Safety and Preliminary Efficacy of TN-401 in Adults With 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)


Sponsor

Tenaya Therapeutics

Enrollment

15 participants

Start Date

Mar 26, 2024

Study Type

INTERVENTIONAL

Conditions

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

Min Age: 18 YearsMax Age: 65 Years

Plain Language Summary

Simplified for easier understanding

This study is testing a gene therapy called TN-401 for a rare inherited heart condition called ARVC (arrhythmogenic right ventricular cardiomyopathy) caused by a mutation in the PKP2 gene. This condition causes dangerous heart rhythm problems. **You may be eligible if...** - You have a confirmed PKP2 gene mutation (pathogenic or likely pathogenic) - You have been diagnosed with ARVC based on standard diagnostic criteria - You have a functioning implantable defibrillator (ICD) already in place for at least 9 months - You have frequent irregular heartbeats (premature ventricular contractions) - Your heart pumping function is reasonably preserved (ejection fraction ≥50%) - Your heart failure symptoms are mild to moderate (NYHA Class I-III) **You may NOT be eligible if...** - You have had a procedure to ablate (burn) heart tissue within 6 months or plan to have one - You have had a prior heart attack - You have severe right heart failure or Class IV heart failure - You have significant kidney or liver disease Talk to your doctor to see if this trial is right for you.

This summary was AI-generated to explain the trial in plain language. It is not medical advice. Always discuss eligibility with your doctor before enrolling in a clinical trial.

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Interventions

GENETICTN-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.


Locations(7)

University of California San Francisco

San Francisco, California, United States

University of Colorado - Anschutz Medical Campus

Aurora, Colorado, United States

Johns Hopkins University

Baltimore, Maryland, United States

Brigham and Women's Hospital

Boston, Massachusetts, United States

Mayo Clinic

Rochester, Minnesota, United States

NYU Langone Health

New York, New York, United States

Cleveland Clinic

Cleveland, Ohio, United States

View Full Details on ClinicalTrials.gov

For the most up-to-date information, visit the official listing.

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NCT06228924


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