Trial results
AI generated results summary. Written by an AI model from the official source data and checked on a sample basis. It can contain mistakes, so confirm anything important against the original source. How we use AI
According to the results reported on ClinicalTrials.gov, this trial enrolled people with Wilson disease — a condition where the body accumulates too much copper. Participants were split into two cohorts based on whether they had mainly neurological (nerve and brain-related) or hepatic (liver-related) symptoms. Within each cohort, people were randomly assigned to receive either the investigational medicine ALXN1840 or a standard-of-care (SoC) therapy — meaning the treatments already commonly used for Wilson disease. In total, 105 people started in Cohort 1 on ALXN1840, 56 in Cohort 1 on SoC, 37 in Cohort 2 on ALXN1840, and 16 in Cohort 2 on SoC, across a 48-week main study period followed by a longer extension phase of up to 60 months. The trial's main focus was measuring levels of a particular form of copper in the blood — called non-ceruloplasmin-bound copper (essentially the "free" copper not attached to a carrier protein) — over the 48 weeks. The reported data shows that for the primary measure — the average daily level of free copper in the blood over 48 weeks — the ALXN1840 groups recorded higher values than the SoC groups. Specifically, the reported figures were 2.50 units for Cohort 1 on ALXN1840 versus 0.87 units for Cohort 1 on SoC, and 4.76 units for Cohort 2 on ALXN1840 versus 0.96 units for Cohort 2 on SoC (measured in micromoles per litre, averaged across hours). For the secondary measures, changes in neurological symptom scores (rated on a scale where lower numbers mean improvement) were also reported at 48 weeks. In Cohort 1, the ALXN1840 group's neurological examination score changed by −2.24 points and the SoC group by −1.59 points. In Cohort 2, the ALXN1840 group changed by −2.06 points while the SoC group's score increased by +1.55 points. Self-reported daily functioning scores showed small changes across all groups. The reported data also shows that 89 out of 104 participants who received ALXN1840 in Cohort 1, 41 out of 56 in the Cohort 1 SoC group, 30 out of 33 in Cohort 2 on ALXN1840, and 12 out of 14 in the Cohort 2 SoC group experienced at least one adverse event (an unwanted medical occurrence during the study period) — though the data does not specify the nature or severity of those events in this summary. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.
View full results on ClinicalTrials.gov ↗
These are the results as reported to ClinicalTrials.gov, not medical advice. Verify independently with the trial site and discuss what they mean for you with your doctor.
Phase 3 Wilson Disease Trial, Terminated
Who may be able to join
AI generated eligibility summary. Written by an AI model from the official source data and checked on a sample basis. It can contain mistakes, so confirm anything important against the original source. How we use AI
Who might be able to join this trial:
- You have been diagnosed with Wilson's disease, confirmed by a scoring system called the Leipzig Score (a score of 4 or higher)
- If you are a woman of childbearing age and are sexually active with a male partner, you are willing to use highly effective birth control starting at least 6 weeks before the trial begins and for 28 days after your last dose of study medication
- If you are a man and are sexually active with a female partner, you are willing to use highly effective birth control from the first day of the trial and for 90 days after your last dose of study medication
Who may not be able to join:
- You have severe liver scarring (cirrhosis) where the liver is no longer able to function properly
- Your liver disease severity score (called a MELD score) is higher than 13 (confirm with trial site)
- Your liver disease score on another measure (called the Modified Nazer score) is higher than 7 (confirm with trial site)
- You have had a significant bleeding episode in your digestive system (such as stomach or intestines) in the past 3 months
- Your liver enzyme levels (called ALT) are too high — the exact limit depends on how long you have already been treated for Wilson's disease (confirm with trial site)
- You have severe neurological symptoms requiring tube feeding or intensive hospital care
- Your red blood cell count is too low (a hemoglobin level below 9 grams per deciliter)
- You have had seizures in the 6 months before joining the trial
- You are pregnant, planning to become pregnant, or are currently breastfeeding
- You have an active hepatitis B or hepatitis C infection, or you are HIV positive
- You have previously been treated with a medication called tetrathiomolybdate
- You have very severe kidney disease requiring dialysis, or your kidneys are not filtering blood well enough (confirm with trial site)
Important: Always verify eligibility with the trial site directly before applying.
Based on publicly available eligibility criteria from ClinicalTrials.gov. Verify directly with the trial site before acting. This is not medical advice.
Contact this trial
Principal Investigator: Eugene S. Swenson, M.D., Ph.D., Alexion Pharmaceuticals, Inc.
Australian sites
Contact details sourced from ClinicalTrials.gov. Verify directly with the trial site before attending.
GP referral letter
Print a one-page summary to share with your doctor.
Trial details
Where this trial is recruiting
4 site(s) in Australia. Confirm current status and contact details directly with the trial site.
Primary endpoints
Daily Mean Area Under The Effect-time Curve (AUEC) of Directly Measured Non-ceruloplasmin-bound Copper (dNCC) From 0 to 48 Weeks (dNCC AUEC0-48W)
Can't join this trial?
Data last synced from ClinicalTrials.gov: 28 July 2026. Trial status can change. Always verify current status directly with the trial site before making any decision.