Efficacy and Safety Study of WTX101 (ALXN1840) in Adult Wilson Disease Patients
Efficacy and Safety Study of WTX101 (ALXN1840) in Adult Wilson Disease Patients
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 (Mo)
 plasma concentration in participants newly diagnosed with Wilson Disease (WD) aged 18 and
 older and with 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
 36-month Extension Period.
Inclusion Criteria:

 -  Willing and able to give informed consent for participation in the study.

 -  Male or female participants, aged 18 years or older as of signing the informed consent
 form.

 -  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 (MELD) score > 11.

 -  Modified Nazer score > 6.

 -  Gastrointestinal bleed within past 6 months.

 -  Alanine aminotransferase > 5 x upper limit of normal (ULN).

 -  Marked neurological disease requiring either nasogastric feeding or intensive
 in-patient medical care.

 -  Severe anemia with a hemoglobin < 9 gram/deciliter (g/dL).