Open-label, Dose Escalation Study of Safety and Preliminary Efficacy of TN-401 in Adults With PKP2 Mutation-associated ARVC (NCT06228924)
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)
🩺 Plain-English Summary (8th-Grade Level)
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.
Who can join: Review the eligibility criteria below.
Location: University of California San Francisco — San Francisco, California
Age range: 18 Years to 65 Years
🧪 Interventions in This Study
- TN-401 (GENETIC)
📋 "Do I Qualify?" — 1-Minute Self Screener
Instant ChecklistFull eligibility criteria are available on the official NIH protocol. View the criteria ↗
📍 All Participating Trial Locations (7 Sites)
- University of California San Francisco — San Francisco, California
- University of Colorado - Anschutz Medical Campus — Aurora, Colorado
- Johns Hopkins University — Baltimore, Maryland
- Brigham and Women's Hospital — Boston, Massachusetts
- Mayo Clinic — Rochester, Minnesota
- NYU Langone Health — New York, New York
- Cleveland Clinic — Cleveland, Ohio
📞 Contact the Study Coordinator
Phone: (650) 416-1186
Email: mpollman@tenayathera.com
Coordinator details come directly from the registered NIH protocol. Confirm the study is still recruiting before traveling.
💰 Cost, Insurance & Patient Rights
- 100% Free Items: The investigational treatment and protocol-required procedures are supplied at zero cost by the sponsor.
- Routine Patient Care: Under federal law (ACA § 2709) and many state statutes, routine care costs are covered during participation.
- Voluntary Participation: You may withdraw at any time without affecting your standard medical care.
More studies in Arrhythmogenic Right Ventricular Cardiomyopathy
- Mayo AVC Registry and Biobank — Rochester, Minnesota
- Non-interventional Study of Seroprevalence of Pre-existing Antibodies Against Adenovirus-associated Virus Vector (AAV9) and the Progression of Disease in Patients With Plakophilin 2 (PKP2)-Associated Arrhythmogenic Right Ventricular Cardiomyopathy (ARVC) — San Francisco, California
Explore by condition: All Arrhythmogenic Right Ventricular Cardiomyopathy clinical trials
💡 How to Participate
- Check the checklist above to see if you may qualify.
- Contact the study coordinator using the phone/email above.
- Ask about the visit schedule, what is covered at no cost, and any travel support.
- Confirm with your own doctor before making a decision.