What changed for Wilson Disease
A plain English summary of what recently moved in the public data Voxsanity tracks for Wilson Disease. Updated from government registries. Not medical advice.
How to read these changes: every figure on this page is the difference between two snapshots of what Voxsanity had tracked on each date. Voxsanity only began taking daily snapshots in July 2026 and is still ingesting the public registries, so a rise here is mostly our own coverage expanding rather than new research appearing. Read it as our tracked coverage growing, not as a claim about real-world research changing this fast: it overstates how much actually changed.
Comparing the two most recent snapshots, 27 July 2026 and 28 July 2026.
No tracked figures changed between these two snapshots. Trial and approval data is checked daily, so this page will update when something moves.
Newly subsidised on the PBS
No PBS-reviewed medicines are tracked for Wilson Disease yet, so new PBS listings cannot be reported for it. This is a gap in what Voxsanity has mapped, not a statement that no medicines are subsidised — check the PBS directly.
Past updates
Tracking began in July 2026. Month on month changes will appear here once a second month of data has been recorded. Until then, the comparison above shows the most recent movement.
Recent trend
Weekly, most recent weeks (fills out to a monthly trend as history grows).
Recent trial results
Trials tracked for Wilson Disease that have reported results to a public registry.
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
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NCT04537377 · results posted 28 January 2026
According to the results reported on ClinicalTrials.gov, this trial (NCT04537377) tested an investigational gene therapy called VTX-801, which was being studied as a potential treatment for Wilson's disease — a rare inherited condition in which copper builds up in the body. Four people took part in total: two received a lower dose (5E12 VG/kg) and two received a higher dose (1.5E13 VG/kg). The trial was measuring safety and tolerability (that is, what unwanted events occurred and how often), as well as several markers related to copper levels in the blood and urine. The study is listed as ongo...
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NCT06128954 · results posted 15 August 2025
According to the results reported on ClinicalTrials.gov, this trial (NCT06128954) enrolled 26 adults in total — 13 in each of two groups. All 26 participants completed the trial with no dropouts. The study compared two formulations of a medicine called triethylenetetramine (TETA), which is used in the context of Wilson's disease (a condition involving copper build-up in the body). One formulation was a once-daily tablet (referred to as "Treatment A"), and the other was an existing twice-daily tablet called Cuprior® ("Treatment B"). Because this was a "crossover" trial, each participant took bo...
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NCT05047523 · results posted 15 October 2024
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...
Read the full trial page · View reported results on ClinicalTrials.gov ↗
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NCT04573309 · results posted 27 October 2023
According to the results reported on ClinicalTrials.gov, this trial (NCT04573309) enrolled a total of nine participants, split into two groups: eight people in Cohort 1 and one person in Cohort 2. The trial was measuring how copper moves through the body — specifically, the difference between how much copper people took in through food and drink versus how much they passed out through urine and faeces (called "copper balance"). It also tracked a substance called molybdenum, which is a component of the study drug ALXN1840, and measured how it moved through the body as well. Seven of the eight p...
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NCT03403205 · results posted 10 October 2023
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...
Read the full trial page · View reported results on ClinicalTrials.gov ↗
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NCT04526210 · results posted 31 August 2023
According to the results reported on ClinicalTrials.gov, this trial (NCT04526210) enrolled 54 people in total — 27 in each of two groups, who received the treatments in different orders (a "crossover" design, meaning everyone took both treatments at different times, just in a different sequence). The trial was measuring how a drug called ALXN1840 might affect the way the body absorbs and processes bupropion hydrochloride (a medicine sometimes used for depression or to support quitting smoking). Specifically, it tracked how much bupropion — and a substance the body naturally converts it into, c...
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NCT04526197 · results posted 21 August 2023
According to the results reported on ClinicalTrials.gov, this trial enrolled 36 people in total — 18 in each of two treatment sequence groups. It was a crossover study, meaning participants took both treatments but in different orders. The trial was measuring how a drug called ALXN1840 (an investigational treatment) affected the way the body absorbs and processes celecoxib (an anti-inflammatory pain medicine), by looking at drug levels in the blood over time. Two people did not complete the trial — one from each group dropped out during the first period, and two more from one group dropped out...
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NCT04560816 · results posted 14 August 2023
According to the results reported on ClinicalTrials.gov, this trial (NCT04560816) enrolled 57 people in total, split across six groups who each received three treatments in a different order. The three treatments were ALXN1840 (the medicine being studied), a placebo (an inactive dummy treatment), and moxifloxacin (an antibiotic used as a comparison). The trial's main purpose was to measure whether ALXN1840 affects the heart's electrical activity — specifically a measurement called the QTcF interval, which is a way of looking at how long it takes the heart to "reset" between beats. A prolonged...
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NCT04422431 · results posted 3 July 2023
According to the results reported on ClinicalTrials.gov, this trial (NCT04422431) tested a single treatment called ALXN1840 in people with Wilson disease, a condition where the body builds up too much copper, particularly in the liver. The trial ran in two back-to-back stages: a 48-week treatment period and a 48-week extension period. Thirty-one participants started the treatment period, 26 of whom completed it. Twenty-five participants went on to start the extension period, and 21 of those completed it. The trial was primarily measuring changes in the amount of copper stored in the liver, and...
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NCT03539952 · results posted 20 April 2023
According to the results reported on ClinicalTrials.gov, this trial (NCT03539952) looked at a treatment for Wilson's disease — a condition where copper builds up in the body. The trial compared two copper-removing medicines: penicillamine (an existing treatment) and TETA 4HCl (the medicine being studied). A total of 77 people started the trial, all initially on penicillamine. After 12 weeks, 53 of those participants had completed that opening phase, and 53 were then randomly assigned to continue on either penicillamine (27 people) or switch to TETA 4HCl (26 people) for the remainder of the stu...
View reported results on ClinicalTrials.gov ↗ · Linked publication on PubMed ↗
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NCT02273596 · results posted 29 September 2021
According to the results reported on ClinicalTrials.gov, this trial enrolled 29 people, all of whom received the study drug ALXN1840, a treatment being investigated for Wilson's disease — a condition where the body builds up too much copper. The trial had a 24-week treatment period followed by a longer extension period. The main thing researchers were measuring was whether a specific form of copper in the blood, called non-ceruloplasmin-bound copper (NCC) — essentially the copper not attached to a particular carrier protein — reached a normal or near-normal level. Of the 29 who started, 22 com...
View reported results on ClinicalTrials.gov ↗ · Linked publication on PubMed ↗
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NCT02763215 · results posted 26 October 2020
According to the results reported on ClinicalTrials.gov, this trial (NCT02763215) enrolled 64 participants in total, all treated as a single group. Of those, 57 completed the study and 7 did not finish. The trial was looking at levels of a specific form of copper in the blood called non-ceruloplasmin-bound copper (NCC for short) — this is the portion of copper in the bloodstream that is not attached to a particular protein, and it can be elevated in people with Wilson's disease. The main thing the trial was measuring was whether participants' NCC levels either returned to a normal range, staye...
These are the results as reported to the registry, not medical advice. Verify independently with the trial site and discuss what they mean for you with your doctor.
What is coming
AI generated pipeline 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 NIH funding data, a moderate amount of public research investment has been directed toward Wilson Disease in the United States. However, the direction of that funding has been falling year on year, which suggests that government interest in this area may be cooling rather than growing. For patients, this trend is worth watching, as public funding often shapes how much new research act...
See the full Wilson Disease page
Not medical advice. Voxsanity republishes public government health data in plain English. These figures describe research and registry activity, not the suitability of any treatment for any person. Trial status can change; always verify current status directly with the trial site and confirm Australian availability with your doctor.