Gene therapy TN-401 tested in adults with PKP2-related arrhythmogenic right ventricular cardiomyopathy
Tests TN-401 gene therapy for safety in adults with inherited right heart disease.
Plain-language title generated from the registry record
Registry title: Open-label, Dose Escalation Study of Safety and Preliminary Efficacy of TN-401 in Adults With PKP2 Mutation-associated ARVC
Key facts
- Where
- Aurora, Baltimore, Boston, Cleveland, New York, Rochester, and San Francisco, United StatesSee the 7 sites
- Phase
- Phase 1
Could this be for you?
Answer for yourself. This is not a decision: only the study team can say whether you can take part.
- Are you between 18 and 65 years old?
Source: NCT06228924, who can take part
Where in United States
- University of Colorado Anschutz Medical CampusAurora, United StatesTaking people
- Johns Hopkins UniversityBaltimore, United StatesTaking people
- Brigham and Women's HospitalBoston, United StatesTaking people
- Cleveland ClinicCleveland, United StatesTaking people
- NYU Langone HealthNew York, United StatesTaking people
- Mayo ClinicRochester, United StatesTaking people
- University of California San FranciscoSan Francisco, United StatesTaking people
Questions to ask the study team
- How often do I need to come in, and for how long each time?
- Are travel costs paid?
- Can I keep my own doctor?
- What happens if I want to stop?
Trial details
- Who runs it
- Tenaya Therapeutics
- Trial code
- NCT06228924
- Record updated
- 6 February 2025
- Original 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.Read full original descriptionShow less
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)
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.
Inclusion: 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: High AAV9 neutralizing antibody titer; Prior myocardial infarction; Right Ventricular Heart Failure; Class IV Heart Failure; Clinically significant renal disease; Clinically significant liver disease; Ventricular tachycardia (VT) ablation within 6 months of Screening or planned VT ablation within 6 months after Screening.