clinical trial · NCT04537377
A Phase I/II Study of VTX-801 in Adult Patients With Wilson's Disease
Wilson's DiseaseVTX-801
brief 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.
started
Sep 3, 2021
primary completion
Dec 17, 2024
completion
Jun 18, 2029
last updated
Jan 30, 2026
official title
A Phase I/II, Multicenter, Non-randomized, Open Label, Adaptive Design, 5-year Follow-up, Single Dose-escalation Study of VTX-801 in Adult Patients With Wilson's Disease
sourced from ClinicalTrials.gov · pharmadog mirrors structured fields, not the full protocol