Status:
COMPLETED
Copper Concentration & Histopathologic Changes in Liver Biopsy in Participants With Wilson Disease Treated With ALXN1840
Lead Sponsor:
Alexion Pharmaceuticals, Inc.
Conditions:
Wilson Disease
Eligibility:
All Genders
18+ years
Phase:
PHASE2
Brief Summary
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 ...
Detailed Description
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-...
Eligibility Criteria
Inclusion
- 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.
Exclusion
- 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.
Key Trial Info
Start Date :
December 2 2020
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
May 17 2023
Estimated Enrollment :
31 Patients enrolled
Trial Details
Trial ID
NCT04422431
Start Date
December 2 2020
End Date
May 17 2023
Last Update
October 18 2024
Active Locations (12)
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1
Research Site
Sacramento, California, United States, 95817
2
Research Site
Ann Arbor, Michigan, United States, 48109
3
Research Site
Dallas, Texas, United States, 75235
4
Research Site
Toronto, Ontario, Canada, M5G 2C4