for people ages 12 years and up (full criteria)
at Los Angeles, California and other locations
study started
estimated completion



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.

Official Title

A Phase 3, Randomized, Rater-Blinded, Multi-Center Study To Evaluate the Efficacy and Safety of ALXN1840 Administered For 48 Weeks Versus Standard of Care in Patients With Wilson Disease Aged 12 Years and Older With an Extension Period of Up To 60 Months


Wilson Disease Hepatolenticular Degeneration ALXN1840 SoC Therapy ALXN1840 15-60 mg


For people ages 12 years and up


  • 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


  • 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


  • University of California, Los Angeles (UCLA) - David Geffen School of Medicine
    Los Angeles California 90095 United States
  • Seattle Children's Hospital
    Seattle Washington 98105 United States


accepting new patients
Start Date
Completion Date
Alexion Pharmaceuticals
Sign up for this study
Phase 3
Study Type
Last Updated