Efficacy and Safety Study of WTX101 (ALXN1840) in Adult Wilson Disease Patients
Status:
Completed
Trial end date:
2018-11-07
Target enrollment:
Participant gender:
Summary
The main purpose of the study was to evaluate the efficacy of ALXN1840 (formerly WTX101) for
24 weeks on non-ceruloplasmin-bound copper (NCC) concentrations adjusted for molybdenum
plasma concentration in participants newly diagnosed with Wilson Disease (WD) who were aged
18 and older and who had NCC concentrations within or above the reference range at the time
of enrollment in the study. The study consisted of a 24-week Treatment Period, followed by a
planned 36-month Extension Period.