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

RECRUITINGPhase 1Sponsored by Tenaya Therapeutics
Actively Recruiting
PhasePhase 1
SponsorTenaya Therapeutics
Started2024-03-26
Est. completion2026-05-01
Eligibility
Age18 Years – 65 Years
Healthy vol.Accepted
Locations7 sites
View on ClinicalTrials.gov →

NCT06228924

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

Age: 18 Years – 65 YearsHealthy volunteers accepted
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

Conditions2

Arrhythmogenic Right Ventricular CardiomyopathyHeart Disease

Locations7 sites

University of California San Francisco
San Francisco, California, 94143
Vasanth Vedantham, MD
University of Colorado - Anschutz Medical Campus
Aurora, Colorado, 80045
Matthew Taylor, MD
Johns Hopkins University
Baltimore, Maryland, 21287
Andreas Barth, MD
Brigham and Women's Hospital
Boston, Massachusetts, 02115
Neal Lakdawala, MD
Mayo Clinic
Rochester, Minnesota, 55905
John R. Giudicessi, MD

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Trial data from ClinicalTrials.gov. Trial status and eligibility can change — verify directly with the study contact or on ClinicalTrials.gov.

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