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 (NCT05047523) looked at a drug called ALXN1840 compared to standard-of-care therapy in people with Wilson disease, a rare condition where the body builds up too much copper. The trial was divided into two groups of participants (Cohort 1 and Cohort 2) and had two phases: a 48-week main period and a 24-week extension period. In total, 40 people began the main period — 15 in Cohort 1 receiving ALXN1840, 16 in Cohort 1 receiving standard therapy, 4 in Cohort 2 receiving ALXN1840, and 5 in Cohort 2 receiving standard therapy. The trial was stopped early before it was fully completed. The primary thing the trial set out to measure was the change in a specific form of copper in the blood (called non-ceruloplasmin-bound copper, which is essentially the "free" copper not attached to a carrier protein) from the start of the trial to 48 weeks. However, the reported data shows that because the study was ended early, this measurement was not collected for any of the participant groups, so no results are available for the main outcome. For the secondary measures — things tracked alongside the main goal — the reported data shows that in Cohort 1, 13 out of 15 participants taking ALXN1840 and 13 out of 16 taking standard therapy experienced at least one adverse event (an unexpected medical occurrence during the study) during the main period; in Cohort 2, this was 3 out of 4 and 4 out of 5 respectively. The reported data also includes various blood-level measurements of copper and the study drug's active component (molybdenum) at different time points, with figures varying across groups, though the trial's early termination means these numbers are based on limited data. 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 Disease, confirmed by a scoring system called the Leipzig Score (score of 4 or higher).
- You have suitable veins that allow blood samples to be taken as required by the study.
- You are able to swallow the study medication (ALXN1840), which comes as tablets or mini-tablets.
- You are willing to avoid foods and drinks that are high in copper.
- You are willing and able to follow the birth control requirements set out in the study plan.
Who may not be able to join:
- You have severe liver scarring (cirrhosis) that has stopped working properly, or your liver disease severity score is above a certain level (confirm with trial site).
- Your Modified Nazer score (a measure of liver disease severity) is higher than 7 (confirm with trial site).
- You have had a significant bleed in your digestive system (such as your stomach or intestines) within the past 3 months.
- Your liver enzyme levels (a marker of liver inflammation) are too high, based on whether you have already been receiving treatment for Wilson Disease or not (confirm with trial site).
- You have severe neurological (brain or nervous system) symptoms that require tube feeding or intensive hospital care.
- Your blood count (specifically haemoglobin, which carries oxygen) is below the normal range for your age and sex.
- You have had a seizure (fit) within the 6 months before joining the trial.
- You have serious kidney failure, including being on dialysis or having very poor kidney function.
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, MD, PhD, 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
2 site(s) in Australia. Confirm current status and contact details directly with the trial site.
Primary endpoints
Percent Change From Baseline to Week 48 in Non-ceruloplasmin-bound Copper in Plasma
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.