Efficacy and Safety Study of WTX101 (ALXN1840) in Adult Wilson Disease Patients

A Phase 2, Multi-centre, Open-label, Study to Evaluate the Efficacy and Safety of WTX101 Administered for 24 Weeks in Newly Diagnosed Wilson Disease Patients Aged 18 and Older With an Extension Phase of 36 Months

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.

Inclusion Criteria:

• Able to understand and willing to comply with study procedures, restrictions, and requirements, as judged by the Investigator.
• Newly established diagnosis of WD by Leipzig-Score ≥ 4 documented by testing as outlined in 2012 European Association for the Study of the Liver Wilson Disease Clinical Practice Guidelines.
• NCC levels within or above the normal reference range (0.8 to 2.3 micromole).
• Willing to undergo 48 hour washout from current WD treatment

Exclusion Criteria:

• Treatment for greater than 24 months for WD with chelation therapy (for example, penicillamine, trientine hydrochloride) or zinc therapy.
• Decompensated hepatic cirrhosis.
• Model for End-Stage Liver Disease score > 11.
• Modified Nazer score > 6.
• Gastrointestinal bleed within past 6 months.
• Alanine aminotransferase > 5 x upper limit of normal.
• Marked neurological disease requiring either nasogastric feeding or intensive in-patient medical care.
• Severe anemia with a hemoglobin < 9 grams/deciliter.