- recruitingTherapeutic use (Phase IV)
Open label, Multicenter, Prospective Study to Characterize the Pharmacokinetics and Pharmacodynamics of Cufence (Trientine Dihydrochloride) and to Investigate the Efficacy and Safety in Wilson’s Disease Patients
This study looks at a medicine called Cufence for people with Wilson's Disease. It checks how the body handles the medicine and how well it works. It also makes sure it's safe to use.
Germany · France · Denmark - recruitingPhase I and Phase II (Integrated)- First administration to humans
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
This study is testing a new medicine, VTX-801, for adults with Wilson's disease. Researchers want to see if it's safe, how well people tolerate it, and if it helps by looking at things like copper levels and brain scans over five years. It's an early-stage study.
Germany · Denmark - active not recruitingPHASE1, PHASE2
A Phase I/II Study of VTX-801 in Adult Patients With Wilson's Disease
This study is testing a new gene therapy, VTX-801, for adults with Wilson's Disease. Researchers want to see if it's safe, how well it works, and if people can reduce their current Wilson's Disease medication over five years.
United States · Denmark · Germany - recruiting
International Wilson's Disease Patient Registry (iWilson Registry)
This registry is collecting information from people with Wilson’s disease during their regular clinic visits. It aims to understand the condition better, how it's treated, and how it affects people's lives over time. It's not a treatment study, but helps improve future care.
Belgium · France · Germany