Reported trial results for Wilson Disease
Every Wilson Disease trial Voxsanity tracks that has reported results to a public registry, newest first. These trials have finished, so they are not recruiting. Not medical advice.
12 trials have reported 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
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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 ongoing, with no participants recorded as having formally completed it at the time of reporting. The reported data shows that across both dose groups, all four participants experienced at least one treatment-emergent adverse event (an unwanted health event that occurred after receiving the treatment). In the higher-dose group, one participant had a serious adverse event, while none were recorded in the lower-dose group. For the copper-related measurements, the reported data shows mixed changes from starting levels across both groups. For example, free copper in the blood showed a small increase in the lower-dose group and a small decrease in the higher-dose group. Total copper in the blood and 24-hour urinary copper figures also varied between the two groups, with some going up and some going down. A blood marker called ceruloplasmin activity showed a modest rise in the lower-dose group and a fall in the higher-dose group. Regarding "responder status" — a measure of whether a participant's body appeared to be processing copper differently — the reported data shows that none of the four participants were classified as responders, and all four were classified as insufficient-responders. It is worth noting that with only two participants in each group, the numbers reported here are averages across a very small number of people, so the reported data should be understood in that context. Where specific breakdown figures were not provided in the submitted results, those details were not reported rather than estimated here. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.
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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 both formulations at different times, allowing a direct comparison. The trial was primarily measuring how the medicine and its breakdown products moved through the bloodstream — a type of measurement called pharmacokinetics (PK). The reported data shows the following blood-level measurements. For the active medicine (TETA) itself, the once-daily formulation produced a total drug exposure over 24 hours (AUC24 — a measure of how much medicine was in the blood across that period) of approximately 12,668 h·ng/mL, compared with approximately 10,972 h·ng/mL for the twice-daily Cuprior®. The peak blood level (the highest concentration reached) was reported as approximately 3,436 ng/mL for the once-daily formulation and approximately 1,567 ng/mL for the twice-daily formulation. The time the medicine took to reach that peak was reported as roughly 0.9 hours for the once-daily tablet and roughly 1.9 hours for Cuprior®, while the time for the medicine to reduce to half its level in the blood was approximately 15.6 hours versus 8.6 hours respectively. Similar blood-level measurements were also reported for two breakdown products of the medicine (called MAT and DAT), with the twice-daily Cuprior® showing higher exposure figures for both breakdown products compared with the once-daily formulation. These numbers represent what was measured and recorded during the study period. The reported data shows differences in how the two formulations behaved in the bloodstream, but this summary does not draw any conclusions about what those differences mean in practice. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.
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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 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.
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 participants in Cohort 1 completed the study, and the single participant in Cohort 2 also completed it. The reported data shows that, for the primary outcome — average daily copper balance measured over the first eight days — Cohort 1 had a result of approximately 0.80 milligrams per day, and Cohort 2 had a result of approximately 0.31 milligrams per day. For the secondary outcomes, the reported data shows changes in copper balance from a starting point across different time periods; in Cohort 1 these change figures ranged from roughly −0.47 to −0.18 milligrams per day, while in Cohort 2 the figures ranged from approximately −0.21 to +0.19 milligrams per day. Copper amounts measured in food, drink, faeces, and urine were also reported, with daily intake figures hovering around 1.6–1.9 milligrams for Cohort 1 and 1.4–1.6 milligrams for Cohort 2. Separately, the trial tracked molybdenum balance and excretion; the reported daily molybdenum balance figures at steady state (the point where the amount going in roughly equals the amount going out) were approximately 1.36–2.76 milligrams per day for Cohort 1 and 2.11–3.66 milligrams per day for Cohort 2. It is worth noting that with only nine participants in total — including just one person in Cohort 2 — the reported numbers represent a very small group, and the data as submitted does not include measures of variability or statistical comparisons. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.
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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 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.
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, called hydroxybupropion — appeared in participants' blood. By the end of the study, 17 people in one sequence group and 20 in the other had completed the trial. The reported data shows the following blood-level measurements for bupropion on its own versus bupropion taken together with ALXN1840. The peak blood concentration of bupropion (the highest level detected) was reported as 98.64 ng/mL without ALXN1840 and 98.02 ng/mL with ALXN1840. The total amount of bupropion absorbed over time (measured two different ways) was reported as 1,021 and 1,049 units without ALXN1840, compared with 1,010 and 1,039 units with ALXN1840. For hydroxybupropion (the substance bupropion is converted into), the reported peak blood level was 286.5 ng/mL without ALXN1840 and 284.3 ng/mL with it, while the total absorption figures were approximately 11,900 and 11,960 units without ALXN1840, compared with 11,560 and 11,630 units with ALXN1840. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.
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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 during the second period. The reported data shows that the primary measurements related to celecoxib levels in the blood. The peak blood concentration of celecoxib (the highest level reached) was reported as 637.1 ng/mL when taken together with ALXN1840 (Treatment A), compared with 567.4 ng/mL when taken without it (Treatment B). The total exposure to celecoxib over time — measured two ways — was reported as 6,406 and 6,743 (in units of hours × ng/mL) with ALXN1840, compared with 6,482 and 6,869 without it. These figures represent how much of the drug was present in the bloodstream across the measurement period and projected beyond it. The reported data also shows secondary measurements looking at molybdenum levels in the blood — molybdenum being used as a way to track ALXN1840 in the body. When ALXN1840 was taken together with celecoxib, the peak molybdenum level was reported as 373.4 ng/mL in total blood and 82.44 ng/mL in a filtered portion of the blood plasma. The corresponding total exposure figures over time were reported as 19,240 and 1,796 (hours × ng/mL), and when projected to infinity, 20,910 and 2,305. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.
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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 QTcF interval can sometimes be a concern with medicines. Of the 57 people who started, 49 completed the study. The reported data shows that the primary measurement — the placebo-corrected change in the QTcF interval for ALXN1840 — produced values ranging from approximately −1.08 to +2.30 milliseconds (ms) across the different time points measured after dosing. The trial's pre-set benchmark was that if the upper limit of the statistical range (called the upper confidence bound) stayed below 10 ms at all time points, ALXN1840 would be considered not to have a notable effect on this heart measurement. The reported data also shows that moxifloxacin, used as a check that the measuring method was working reliably, produced QTcF changes of approximately 9.65, 11.04, and 11.37 ms at one, two, and three hours after dosing — figures above the 5 ms threshold the trial used to confirm the measurement method was functioning as intended. For the secondary measures, the reported changes in heart rate, PR interval, and QRS interval (other aspects of the heart's electrical activity) were all small and similar across the ALXN1840, placebo, and moxifloxacin groups, with no large differences noted in the reported numbers. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.
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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 also looked at changes in liver scarring (fibrosis) using several different scoring systems. The reported data shows that for the main outcome — the change in liver copper concentration after 48 weeks — the average change from the starting measurement was a reduction of 92.8 micrograms per gram of liver tissue. Regarding liver scarring, three different scoring systems were used. Each system groups participants by how much their scarring score changed (improved, stayed the same, or got worse) from the start of the trial to week 48, but the individual category breakdowns for each scoring scale involve multiple participant counts across different change categories, and a plain-language summary of every sub-group is not straightforward to present without the full category labels from the submitted data table. The reported data also shows changes in two markers measured directly from liver tissue samples: the average change in collagen content (a sign of scarring) was 8.54 percentage points, and the average change in a substance called alpha-SMA (another marker linked to scarring activity) was 1.60 percentage points. Whether these changes represent increases or decreases from the starting point is not clearly specified in the submitted data as provided. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.
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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 study. The trial then tracked participants through several further phases, with some continuing up to around two years. The reported data shows that the main thing being measured was a blood marker called "serum NCC" — a way of detecting a particular form of copper in the blood (measured in micrograms per litre). At 24 weeks after the random assignment, the reported average serum NCC was 46.5 µg/L for the penicillamine group and 58.7 µg/L for the TETA 4HCl group. The trial had set a pre-defined cut-off to judge whether TETA 4HCl performed comparably to penicillamine. For a secondary measure — the amount of copper passed out in urine over 24 hours — the reported average was 274.5 µg per 24 hours for the TETA 4HCl group and 510.8 µg per 24 hours for the penicillamine group. A clinician's rating of overall disease change (on a 7-point scale where 1 means "very much improved" and 7 means "very much worse") averaged 4.1 for the penicillamine group and 3.9 for the TETA 4HCl group, both sitting close to the "no change" mark of 4. 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 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 completed the initial 24-week period, and all 22 then entered the extension phase, though none had completed that longer phase at the time results were submitted. The reported data shows that 85.7% of participants (roughly 6 in every 7) achieved or maintained what the study defined as a normalised copper level during the trial. Among those who started with copper levels above the normal range, the reported data shows it took a median of around 147.5 days (about 21 weeks) for copper levels to reach the normal range on two consecutive measurements. The reported data also shows that the average NCC level fell by 2.56 units (micromoles per litre) from the start of the trial to week 24. For secondary measures looking at neurological and psychiatric symptoms, the reported data shows small reductions in scores on rating scales used to assess Wilson's disease-related neurological symptoms — for example, a combined neurological score dropped by an average of around 8 points out of a possible 218 — and small reductions across various psychiatric symptom categories. These numbers reflect averages across participants and a lower score on these scales was defined by the researchers as indicating fewer symptoms. 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 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, stayed in a normal range, or dropped by at least 25% over six months of treatment. The reported data shows that for the primary (main) goal — achieving or maintaining normal NCC levels, or reducing NCC by at least 25% within six months — around 51.6% of participants met that threshold. For a similar measurement taken at the last available check-up for each participant (which could be anywhere from 1 to 24 months into the study), the reported figure was 62.5%. The reported data also shows that average NCC levels in the blood were lower at later time points compared to the start of the study: the reported change was −0.60 units (micromoles per litre) at six months, −1.24 at 24 months, and −0.94 at the last recorded assessment. Among participants whose NCC was above the normal range when they enrolled, the reported median time to reaching a normal level was approximately 139.5 days. Other blood copper measurements — exchangeable copper and copper plasma ultrafiltrate — also showed reported reductions from baseline at most time points, though the size of those changes varied. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.
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Not medical advice. These are results as reported by the trial sponsor to a public registry, summarised in plain English. They describe what a trial recorded, not whether a treatment is suitable for any person. Verify anything important against the original registry record and discuss what it means for you with your doctor.