Copper Concentration & Histopathologic Changes in Liver Biopsy in Participants With Wilson Disease Treated With ALXN1840

Completed · Phase 2

Conditions studied: Wilson Disease

In brief

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.

Key facts

Study ID

NCT04422431

Run by

Alexion Pharmaceuticals, Inc.

People needed

31

Starts

2020-12-02

Expected to finish

2023-05-17

Last updated by the study team

2024-10-18

Who can join

Age: 18 and older. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

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

You may not qualify if…

• Decompensated cirrhosis or Model for End Stage Liver Disease score > 13.
• Modified Nazer score > 7.
• Clinically significant gastrointestinal bleed within past 3 months.
• Alanine aminotransferase > 2 × upper limit of normal.
• History of bleeding abnormality or known coagulopathy, including platelet count < 100,000, and international normalized ratio for prothrombin time ≥ 1.5.
• Participant unwilling to accept blood products, if required.
• Marked neurological disease requiring either nasogastric feeding tube or intensive inpatient medical care.
• Hemoglobin less than lower limit of the reference range for age and sex.
• Participants in renal failure, defined as in end-stage renal disease on dialysis (chronic kidney disease 5) or creatinine clearance < 30 milliliters/minute.
• Lymphoma, leukemia, or any malignancy within the past 5 years.
• Current or chronic history of liver disease not associated with WD.

Where it is running

• Research Site — Sacramento, California, United States
• Research Site — Ann Arbor, Michigan, United States
• Research Site — Dallas, Texas, United States
• Research Site — Toronto, Ontario, Canada
• Research Site — Århus N, Denmark
• Research Site — Grafton, New Zealand
• Research Site — Moscow, Russia
• Research Site — Saint Petersburg, Russia
• Research Site — Singapore, Singapore
• Research Site — Barcelona, Spain
• Research Site — Valencia, Spain
• Research Site — London, United Kingdom

Full record on ClinicalTrials.gov

Trial information comes from ClinicalTrials.gov and is refreshed daily. TrialsForMe does not provide medical care and does not run the studies it lists.