Wilson's DiseaseNCT05239858
International Wilson's Disease Patient Registry (iWilson Registry)
An observational study of Wilson's Disease, run by Orphalan at 16 sites in 7 countries.
Trial summary
Longitudinal, observational, non-interventional, standard of care Registry. Data will be collected from the routinely scheduled WD clinic visits at approximately 6-12 month intervals. Read more about this study
- Who is it for?
- Ages 12 years and older
- Conditions the study is for
- Wilson's Disease
- How many people
- 500 participants
- Where
- 16 sitesin 7 countries
- Study dates
- 29 Jun 2022to Dec 2027
- How long will I take part?
- 5 Years
- Phase
- Observational
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 12 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: The Registry aims to be as inclusive as possible. All patients with WD (adult and paediatric ≥12) who provide informed consent/assent can be included. They will be approached during regular follow up visits for ongoing care of their Wilson's Disease by their primary physician.
Who may be able to join
- Patient is able to provide, and has provided, written informed consent/assent
- Written documentation has been obtained in accordance with the relevant country and local privacy requirements, where applicable, including:
- For US sites: Authorization for Use and Release of Health Research Study Information
- For EU sites: Data Protection Consent
- All patients diagnosed with WD including pre-symptomatic individuals and individuals with co-morbidities/diagnoses
- Any treatments including prescribed and homeopathic/traditional therapies or naive patients on no therapy
Who cannot join
- 1. Refusal of informed consent by either patient or their legally acceptable guardian
Trial locations and contacts
16 sites in 7 countries. Contact the study team or a site near you to ask about taking part.
Maureen Richardson contact
16 sites in 7 countries
Germany4 sites, 4 recruiting
- Recruiting
Charite-Univeritatsmedizin Berlin Hospital
Berlin
Frank Tacke, Dr.
- Recruiting
Universitatsklinikum Dusseldorf
Düsseldorf
Christian Hartmann, Dr.
- Recruiting
Hannover Medical School (MHH)
Hanover
Heiner Wedemeyer, Dr.
- Recruiting
Universitätsklinikum Leipzig
Leipzig
Thomas Berg, Dr.
Spain4 sites, 4 recruiting
- Recruiting
University Hospital Clínic de Barcelona, C. de Villarroel
Barcelona
Xavier Forns, Dr.
- Recruiting
Hospital Universitario GC Dr Negrín
Las Palmas de Gran Canaria, Canary Islands
Quinones, Dr.
- Recruiting
Hospital Universitario Virgen del Rocío
Seville
Javier Ampuero, Dr.
- Recruiting
Hospital Universitario Y Politécnico La Fe
Valencia
Marina Berenguer, Dr.
France2 sites, 2 recruiting
- Recruiting
Hospices Civils de Lyon
Bron, Auvergne-Rhône-Alpes, 69677
Eduardo Couchonnal Bedoya
- Recruiting
HF Adolphe de Rothschild
Paris, Île-de-France Region
Aurelia Poujois
Poland2 sites, 2 recruiting
- Recruiting
Institute of Psychiatry and Neurology
Warsaw
Anna Czlonkowska, Dr.
- Recruiting
The Children's Memorial Health Institute
Warsaw
Piotrem Socha, Prof.
United Kingdom2 sites, 2 recruiting
- Recruiting
Leeds Teaching Hospitals NHS Trust
Leeds
Joanna Moore, Dr.
- Recruiting
Royal Free Hospital
London
Emmanouil Tsochatzis, Professor
Belgium1 site, 1 recruiting
- Recruiting
University Hospital Leuven
Leuven
David Cassiman, Dr.
Saudi Arabia1 site, 1 recruiting
- Recruiting
King Faisal Specialist Hospital in Riyadh
Riyāḑ, Riyadh Region, 11564
Saad Alghamdi
About this study
Longitudinal, observational, non-interventional, standard of care Registry. Data will be collected from the routinely scheduled WD clinic visits at approximately 6-12 month intervals. At enrolment, in addition to data from the clinic visit, retrospective data will be collected from the diagnostic evaluation and any relevant past medical history and a summary of WD medication history.
Read the detailed description
This is a retrospective/prospective observational registry without a control group. A standardised collection tool will be utilised to capture and compare data. The registry will use common data elements, guided by a literature review, and expert input from the multidisciplinary advisory board. The SF-12 Health Survey will be requested from patients aged 18 and over at entry to the Registry to provide a snapshot of baseline quality of life assessment in patients with WD. This will enhance generalisability (external validity) and comparability with clinical trial data.
The objectives of the registry are:
- Natural history of diagnosis and treated WD patients by documentation of observed clinical practice and patient experience
- Measure, and compare epidemiological and clinical aspects of Wilson Disease (WD) in the participating countries, thereby encouraging the identification of new standards for the management of WD
- To provide data for epidemiological research and identify groups of patients potentially eligible for multi-centre trials
- Describe clinical pathways, by documenting standardised examinations and tests used to monitor WD patients including tools to evaluate adherence and compliance to therapies using real-world outcomes.
- To be more inclusive (heterogeneous patient population; paediatric, adults, elderly) using much broader inclusion criteria and fewer exclusion criteria than commonly used in randomised trials.
- Longer follow-up compared to RCTs beneficial detecting delayed or long-term benefits or harm and surveillance for rare events of the disease itself or therapies
Patients will be seen as per their usual standard of care visits. Data points of interest for the Registry will be collected. These may include but not limited to: Medical history, Wilson's Disease Diagnosis and Medical History, Genetics, Liver Assessments, Fibrosis Stage, Liver Transplant, Neurological Assessments, Psychological Assessments, Clinical Global Impression of Change Scale (CGIC), Laboratory measurements incl. Urinary Copper Excretion and Free Copper (NCC), Physical Examination, Adverse Reactions and Serious Adverse Reactions documented by physicians
- Conditions
- Wilson's Disease
- Keywords
- trientine, d-Penicillamine, ATP7B, hepatolenticular degeneration, copper metabolism, zinc
- Medical subject headings
- Hepatolenticular Degeneration
What is being measured
The primary outcome is the main question the study is designed to answer.
Describe the natural history of treated WD
Describe the natural history of treated WD and to explore the clinical phenotype, geographic, ethnic and gender influences on the course of the condition.
Measured: 5 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted15 Feb 2022
- Study start29 Jun 2022
- Primary completionDec 2027 (estimated)
- Study completionDec 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Wilson's DiseaseStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 500 participants (estimated)
- Target duration
- 5 Years
Sponsor and investigators
- Lead sponsor
- Orphalan
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Ergomed
- Responsible party
- Sponsor
- Investigators
- Pramod Mistry, PhD, Study chair, Yale University Hospital, Conneticut
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05239858
- Sponsor's protocol ID
- ORPH-131-005
- Status last verified
- Aug 2026
- First submitted
- 4 Feb 2022
- Last updated
- 11 Aug 2026
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 11 Aug 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- International Wilson's Disease Patient Registry (iWilson Registry)
- ClinicalTrials.gov ID
- NCT05239858
- Acronym / sponsor ID
- ORPH-131-005
- Conditions
- Wilson's Disease
- Keywords
- trientine, d-Penicillamine, ATP7B, hepatolenticular degeneration, copper metabolism, zinc
- Study type
- Observational
- Recruitment status
- Recruiting
- Enrolment
- 500 participants (estimated)
- Lead sponsor
- OrphalanIndustry (pharma, biotech, devices)
- Collaborators
- Ergomed
- Study start
- 29 Jun 2022 (actual)
- Primary completion
- Dec 2027 (estimated)
- Study completion
- Dec 2027 (estimated)
- First posted
- 15 Feb 2022 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Longitudinal, observational, non-interventional, standard of care Registry. Data will be collected from the routinely scheduled WD clinic visits at approximately 6-12 month intervals. At enrolment, in addition to data from the clinic visit, retrospective data will be collected from the diagnostic evaluation and any relevant past medical history and a summary of WD medication history.
Detailed description
This is a retrospective/prospective observational registry without a control group. A standardised collection tool will be utilised to capture and compare data. The registry will use common data elements, guided by a literature review, and expert input from the multidisciplinary advisory board. The SF-12 Health Survey will be requested from patients aged 18 and over at entry to the Registry to provide a snapshot of baseline quality of life assessment in patients with WD. This will enhance generalisability (external validity) and comparability with clinical trial data.
The objectives of the registry are:
- Natural history of diagnosis and treated WD patients by documentation of observed clinical practice and patient experience
- Measure, and compare epidemiological and clinical aspects of Wilson Disease (WD) in the participating countries, thereby encouraging the identification of new standards for the management of WD
- To provide data for epidemiological research and identify groups of patients potentially eligible for multi-centre trials
- Describe clinical pathways, by documenting standardised examinations and tests used to monitor WD patients including tools to evaluate adherence and compliance to therapies using real-world outcomes.
- To be more inclusive (heterogeneous patient population; paediatric, adults, elderly) using much broader inclusion criteria and fewer exclusion criteria than commonly used in randomised trials.
- Longer follow-up compared to RCTs beneficial detecting delayed or long-term benefits or harm and surveillance for rare events of the disease itself or therapies
Patients will be seen as per their usual standard of care visits. Data points of interest for the Registry will be collected. These may include but not limited to: Medical history, Wilson's Disease Diagnosis and Medical History, Genetics, Liver Assessments, Fibrosis Stage, Liver Transplant, Neurological Assessments, Psychological Assessments, Clinical Global Impression of Change Scale (CGIC), Laboratory measurements incl. Urinary Copper Excretion and Free Copper (NCC), Physical Examination, Adverse Reactions and Serious Adverse Reactions documented by physicians
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Target follow-up
- 5 Years
Who may qualify
- Ages
- 12 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- The Registry aims to be as inclusive as possible. All patients with WD (adult and paediatric ≥12) who provide informed consent/assent can be included. They will be approached during regular follow up visits for ongoing care of their Wilson's Disease by their primary physician.
Key inclusion criteria (6 listed)
- Patient is able to provide, and has provided, written informed consent/assent
- Written documentation has been obtained in accordance with the relevant country and local privacy requirements, where applicable, including:
- All patients diagnosed with WD including pre-symptomatic individuals and individuals with co-morbidities/diagnoses
- Any treatments including prescribed and homeopathic/traditional therapies or naive patients on no therapy
Key exclusion criteria (1 listed)
- 1. Refusal of informed consent by either patient or their legally acceptable guardian
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
Describe the natural history of treated WD
Time frame: 5 years
Describe the natural history of treated WD and to explore the clinical phenotype, geographic, ethnic and gender influences on the course of the condition.
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 11 Aug 2026.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Maureen Richardson contact
Pramod Mistry, PhD study chair
Yale University Hospital, Conneticut
16 sites in 7 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Wilson's Disease, run by Orphalan at 16 sites in 7 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 1 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 11 Aug 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 16 sites in Germany, Spain, France, Poland, United Kingdom and 2 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Carla Bennett (+44 (0)7918380893, clinicaloperations@orphalan.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Describe the natural history of treated WD (time frame: 5 years). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 11 Aug 2026.