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)
Number and severity of Adverse Events over the course of the study. — 52 weeks
Number of Serious Adverse Events related to study drug. — 5 years
Trial sites (7)
Facility
City
Region
Status
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.
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