drugset / Trial / NCT06228924
Open-label, Dose Escalation Study of Safety and Preliminary Efficacy of TN-401 in Adults With PKP2 Mutation-associated ARVC
Non-randomizedSequentialOpen-labelTreatment
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.
Timeline
- Start
- 2024-03-26
- Primary completion
- 2026-05-01
- Completion
- 2029-10-01
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | TN-401 | Gene therapy (AAV / viral vector) | 3e+13 vg/kg | — |
| Subject | TN-401 | Gene therapy (AAV / viral vector) | 6e+13 vg/kg | — |