Wilson Disease
Plain English clinical trial information, updated daily from ClinicalTrials.gov and public government registries. Not medical advice.
How these figures are counted
- Trials on record is every trial tagged to this condition at any stage, except those withdrawn before starting or terminated early. Most are completed rather than ongoing, so this is a measure of research history, not of current activity. It is also smaller than the sitewide "trials tracked" figure, which does include withdrawn and terminated trials.
- Recruiting now counts only trials whose registry status is "Recruiting". Trials listed as not yet recruiting or enrolling by invitation are excluded, so this understates how many may open to new participants soon.
- Australian sites counts trials counted under "Recruiting now" that list at least one Australian site. A site being listed does not mean it is currently enrolling, and Australian trials registered only on ANZCTR are not counted yet, so this understates local availability.
- Phase 3 trials counts trials counted under "Recruiting now" that are at Phase 3.
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Voxsanity currently covers 103 conditions. We are expanding coverage regularly.
Understanding Wilson Disease
Wilson disease is a rare inherited condition in which the body is unable to properly process copper. Normally, the liver filters excess copper and removes it from the body, but in Wilson disease this process does not work correctly, causing copper to build up slowly over time in the liver, brain, and other organs. It is caused by a change in a specific gene, meaning a person is born with it, though symptoms often do not appear until copper has accumulated over a number of years. It is not contagious and is not caused by anything a person did or did not do.
In Australia, Wilson disease is considered rare, affecting roughly 1 in 30,000 people, which means there are likely only a few thousand Australians living with the condition at any one time. It tends to be diagnosed in children, teenagers, and young adults, though it can be identified at any age. Because it is uncommon, getting a correct diagnosis sometimes takes time, and people are often referred to specialists such as hepatologists or neurologists for assessment and ongoing care.
Treatment for Wilson disease generally focuses on reducing the amount of copper in the body and preventing further build-up. Doctors typically use medications that help the body remove excess copper or block copper from being absorbed through food. People with the condition are also usually given guidance around dietary copper intake. Treatment is generally lifelong, and with consistent management many people are able to live full and active lives. If you have questions about what the right approach looks like for your specific situation, your treating medical team is the best source of guidance.
This overview is an AI generated summary written from public medical sources and checked on a sample basis. It is general information, not medical advice or a diagnosis. Always speak with your doctor about your situation. How we use AI
Can you access treatment for Wilson Disease in Australia right now? Here's what's publicly known.
- No PBS-subsidised medicines are currently mapped for Wilson Disease on Voxsanity. Your doctor or pharmacist can advise on what may be available.
- No trials currently recruiting at Australian sites right now. Worldwide trials may still be an option. See the trial list below.
Information on this site does not constitute medical advice. Data is sourced from publicly available government registries. Always consult your doctor before making any health decisions. Trial status should be verified directly with the trial site.
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Organisations with the most recruiting trials for Wilson Disease.
Where trials are running
Clinical trial sites for Wilson Disease by country. Australia is highlighted.
Clinical trials for Wilson Disease
Showing 21 of 21 trials
The plain English eligibility text on each trial below is an AI generated summary of the official criteria, checked on a sample basis. How we use AI
🇦🇺 NCT03403205 · Alexion Pharmaceuticals, Inc. Phase 3 · 22 countries
**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...
🇦🇺 NCT05047523 · Alexion Pharmaceuticals, Inc. Phase 3 · 7 countries
**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), whi...
NCT03131427 · Beijing Friendship Hospital Not Applicable · 1 country
Who might be able to join this trial: - You have been diagnosed, or possibly diagnosed, with a genetic or inherited liver condition, such as Wilson's disease, hereditary hemochromatosis, inherited high bilirubin conditions, inherited cholestatic liver disease, or another genetic/metabolic liver disease. Who may not be...
NCT03334292 · Yale University Not Applicable · 3 countries
Who might be able to join this trial: - You have been diagnosed with Wilson's Disease (WD) - You are able and willing to sign a consent form agreeing to take part (for those under 18, a parent or guardian must give permission, and the child may also need to give their agreement depending on their age and local rules)...
NCT04012658 · Wan-Jin Chen Not Applicable · 1 country
Who might be able to join this trial: - People who have been diagnosed with Wilson's Disease through genetic testing - People who carry the gene for Wilson's Disease but do not have symptoms - Family members of someone who has Wilson's Disease or carries the gene for it - Healthy volunteers who are not related to anyon...
NCT04880356 · Fondazione I.R.C.C.S. Istituto Neurologico Carlo Besta Not Applicable · 1 country
Who might be able to join this trial: - You are 18 years old or older - You have been diagnosed with a very rare inherited (genetic) disease that affects the nervous system and causes it to break down or affects how the body processes energy - You have a neurological (brain or nervous system) disease that has not yet b...
NCT04965545 · Second Affiliated Hospital, School of Medicine, Zhejiang University Not Applicable · 1 country
Who might be able to join this trial: - You have been diagnosed with Wilson's disease through genetic testing Who may not be able to join: - You are unwilling or unable to attend follow-up appointments as part of the trial Important: Always verify eligibility with the trial site directly before applying.
NCT04965558 · Second Affiliated Hospital, School of Medicine, Zhejiang University Not Applicable · 1 country
Who might be able to join this trial: - You have been diagnosed with Wilson's Disease through genetic testing Who may not be able to join: - You are unwilling or unable to attend follow-up appointments or check-ins as part of the trial Important: Always verify eligibility with the trial site directly before applying.
NCT05183165 · Fondation Ophtalmologique Adolphe de Rothschild Not Applicable · 1 country
Who might be able to join this trial: - You are 18 years old or older - You have been diagnosed with Wilson's disease, confirmed using a standard scoring system called the Leipzig score - You are currently pregnant, at any stage of pregnancy - You are willing and able to give your consent to take part in the study - Yo...
NCT05231876 · Fondation Ophtalmologique Adolphe de Rothschild Not Applicable · 1 country
Who might be able to join this trial: - You have been diagnosed with Wilson disease Who may not be able to join: - You (or your legal representative, if applicable) are not able or willing to provide written consent to take part in the trial Important: Always verify eligibility with the trial site directly before app...
NCT05239858 · Orphalan Not Applicable · 7 countries
Who might be able to join this trial: - You must be willing and able to sign a consent form agreeing to take part in the study - You must agree to allow your health information to be used for research purposes (the exact paperwork depends on your country) - You must have been diagnosed with Wilson's disease, including...
NCT05444127 · Fondation Ophtalmologique Adolphe de Rothschild Not Applicable · 1 country
**Who might be able to join this trial:** - You are willing to give your consent to take part in the study - You are covered by a social security (health insurance) scheme - *If you are in the patient group:* You have been diagnosed with Wilson's disease - *If you are in the comparison group (healthy volunteers):* You...
NCT05493605 · Fondation Ophtalmologique Adolphe de Rothschild Not Applicable · 1 country
**Who might be able to join this trial:** - You are an adult who has been diagnosed with Wilson's disease, confirmed by a specialist scoring system called the Leipzig score (with a score of 4 or higher) - You are willing and able to give your consent to take part, or your legal guardian/representative can give consent...
NCT06430359 · Hospices Civils de Lyon Not Applicable · 1 country
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 above 4). - You are between 6 and 70 years old. - You are able to collect all of your urine over a 24-hour period for testing. - You are currently being treated with one...
NCT06466291 · Asociación Española para el Estudio del Hígado Not Applicable · 1 country
Who might be able to join this trial: - You have been officially diagnosed with Wilson Disease. Who may not be able to join: - You are not willing to sign the consent form agreeing to take part in the study. Important: Always verify eligibility with the trial site directly before applying.
NCT06573723 · Hospital Italiano de Buenos Aires Not Applicable · 1 country
**Who might be able to join this trial:** - You have been diagnosed (either through medical examination or genetic/molecular testing) with one of these rare conditions: Amyloidosis, Sarcoidosis, Phacomatosis, Pheochromocytoma, Paraganglioma, Von Hippel-Lindau Disease, Immunoglobulin G4-Related Disease, Demyelinating Di...
NCT06650319 · Chaohui Yu Early Phase 1 · 1 country
**Who might be able to join this trial:** - People who are able to fully understand what the study involves, including its purpose, methods, and possible side effects, and who are willing to take part and sign a consent form. - People who have been diagnosed with Wilson Disease. - People whose Wilson Disease has been...
NCT06858670 · Hanoi Medical University Not Applicable · 1 country
Who might be able to join this trial: - You are currently undergoing orthodontic (braces) treatment on the front/outer side of your teeth - You have a specific tooth position issue where your lower back molars (second molars) on both sides of your mouth are tilted inward toward your tongue more than normal - You are wi...
NCT06945081 · Centre Hospitalier Universitaire de Saint Etienne Not Applicable · 1 country
**Who might be able to join this trial:** **For people with Wilson's disease:** - You are 12 years old or older - You have been diagnosed with Wilson's disease confirmed by genetic testing - You are being cared for at the Wilson's Disease Reference Center - You are currently being treated with a medication called D-Pe...
NCT07240896 · DSciLab Co., Ltd. Early Phase 1 · 1 country
**Who might be able to join this trial:** - You are 18 years of age or older (any gender) - You have been diagnosed with Wilson's Disease according to specific medical guidelines, with a diagnosis confirmed at least one year before joining the study, and a particular protein in your blood (ceruloplasmin) is at a very...
NCT07301216 · Yale University Not Applicable · 1 country
**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 are willing and able to sign a consent form agreeing to take part in the study - You are willing to follow all study requirements, including collec...
Advocacy and support in Australia
Organisations that can offer information, support, or connection for Wilson Disease. Where more than one is listed, they serve different needs or regions, so choose whichever fits your situation.
There's no dedicated Australian organisation for Wilson disease. Rare Voices Australia is the best starting point for general rare disease support.
This is a starting point, not medical advice or an endorsement. Contact details change; search the organisation name to find its current website.