for people ages 18 years and up (full criteria)
at Sacramento, California and other locations
study started



The main objective of the study is to evaluate the change in liver copper (Cu) concentration following 48 weeks of treatment with ALXN1840 in adult participants with Wilson Disease (WD) who have been previously treated for at least 1 year with standard of care (that is, trientine, penicillamine, or zinc). In the Treatment Period, efficacy and safety of ALXN1840 will be assessed at Week 48.

Official Title

A Phase 2, Single-arm Pathologist-blinded 48-week Study Using Liver Biopsy Specimens to Assess Copper Concentration and Histopathologic Changes in ALXN1840-treated Patients With Wilson Disease Followed by an up to 48-weeks Extension Period


Participants who complete the 48-week Treatment Period will be offered the opportunity to continue their treatment in a 48-week Extension Period that will offer additional time for evaluation of long-term efficacy and safety of ALXN1840. There will be no liver biopsies during the Extension Period.


Wilson Disease, Copper, ALXN1840, Histology, Liver Biopsy, Hepatolenticular Degeneration, Tetrathiomolybdate, Choline, Bis-Choline Tetrathiomolybdate


You can join if…

Open to people ages 18 years and up

  1. Diagnosis of WD by Leipzig Criteria ≥ 4 or by historical test results.
  2. Continuous treatment for WD with penicillamine, trientine or zinc for at least 1 year prior to screening.
  3. Body mass index < 30 kilograms/meter squared.
  4. Able to cooperate with a percutaneous liver biopsy.
  5. Willing and able to follow protocol-specified contraception requirements.
  6. Capable of giving signed informed consent.

You CAN'T join if...

  1. Decompensated cirrhosis or Model for End Stage Liver Disease score > 13.
  2. Modified Nazer score > 7.
  3. Clinically significant gastrointestinal bleed within past 3 months.
  4. Alanine aminotransferase > 2 × upper limit of normal.
  5. History of bleeding abnormality or known coagulopathy, including platelet count < 100,000, and international normalized ratio for prothrombin time ≥ 1.5.
  6. Participant unwilling to accept blood products, if required.
  7. Marked neurological disease requiring either nasogastric feeding tube or intensive inpatient medical care.
  8. Hemoglobin less than lower limit of the reference range for age and sex.
  9. Participants in renal failure, defined as in end-stage renal disease on dialysis (chronic kidney disease 5) or creatinine clearance < 30 milliliters/minute.

    10. Lymphoma, leukemia, or any malignancy within the past 5 years. 11. Current or chronic history of liver disease not associated with WD.


  • Clinical Trial Site
    Sacramento California 95817 United States
  • Clinical Trial Site
    Dallas Texas 75390 United States


accepting new patients
Start Date
Completion Date
Alexion Pharmaceuticals, Inc.
Sign up for this study
Phase 2 Wilson's Disease Research Study
Study Type
About 31 people participating
Last Updated