Efficacy and Safety of ALXN1840 (Formerly Named WTX101) Administered for 48 Weeks Versus Standard of Care in Patients With Wilson Disease With an Extension Period of up to 60 Months
Efficacy and Safety of ALXN1840 (Formerly Named WTX101) Administered for 48 Weeks Versus Standard of Care in Patients With Wilson Disease With an Extension Period of up to 60 Months
The purpose of this study is to evaluate the efficacy of ALXN1840 (formerly called WTX101)
 administered for 48 weeks compared to standard of care (SoC) in WD patients aged 12 and
 older.
Inclusion:

 -  Established diagnosis of Wilson disease by Leipzig-Score = or > than 4

 -  12 years of age or older

 -  Female patients of childbearing potential, if heterosexually active, must be willing
 to follow protocol-specified guidance for highly effective contraception starting at
 least 6 weeks before Day 1 visit and continuing through 28 days after the last dose of
 either ALXN1840 or SoC

 -  Male patients, if heterosexually active, must be willing to follow protocol-specified
 guidance for highly effective contraception beginning at Day 1 and continuing through
 90 days after last dose of either SoC or ALXN1840

 Exclusion:

 -  Decompensated hepatic cirrhosis

 -  MELD score > 13

 -  Modified Nazer score > 7

 -  Clinically significant GI bleed within past 3 months

 -  Alanine aminotransferase > 2 X upper limit of normal (ULN) for patients treated for >
 28 days with WD therapy (Cohort 1)

 -  Alanine aminotransferase > 5 X ULN for treatment naïve patients or patients who have
 been treated for < or = to 28 days (Cohort 2)

 -  Marked neurological disease requiring either nasogastric feeding or intensive
 inpatient medical care

 -  Hemoglobin < 9 g/dL

 -  History of seizure activity within 6 months prior to informed consent

 -  Pregnant (or women who are planning to become pregnant) or breastfeeding women

 -  Active infection with hepatitis B virus (positive hepatitis B surface antigen) or C
 virus or seropositivity for human immunodeficiency virus (HIV)

 -  Previous treatment with tetrathiomolybdate

 -  Patients with end-stage renal disease on dialysis (CKD 5) or creatinine clearance < 30
 mL/min