Copper Concentration & Histopathologic Changes in Liver Biopsy in Participants With Wilson Diseas… (NCT04422431) | Clinical Trial Compass
CompletedPhase 2
Copper Concentration & Histopathologic Changes in Liver Biopsy in Participants With Wilson Disease Treated With ALXN1840
United States, Canada, Denmark31 participantsStarted 2020-12-02
Plain-language 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 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.
Who can participate
Age range
18 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion criteria
. 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 criteria
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
Change From Baseline in Liver Cu Concentration at Week 48 (Treatment Period)
. 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.