drugset / Trial / NCT04537377
A Phase I/II Study of VTX-801 in Adult Patients With Wilson's Disease
NaSequentialOpen-labelTreatment
Summary
The objectives of this clinical trial are to assess, for up to 5 years, the safety, tolerability and pharmacological activity of a single ascending doses of VTX-801, a gene therapy, administered intravenously (IV) to adult patients with Wilson's Disease prior to and following background WD therapy withdrawal.
Timeline
- Start
- 2021-09-03
- Primary completion
- 2024-12-17
- Completion
- 2029-06-18
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | NIMATPAGENE PARIPARVOVEC | Gene therapy (AAV / viral vector) | — | Intravenous |