International Wilson's Disease Patient Registry (iWilson Registry)

NCT05239858 | Recruiting

• 1 other identifier: ORPH-131-005
• Study Type: observational
• Target: 500
• Locations: 7 countries
• Sites: 16

Brief 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. 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.

Conditions

Wilson's Disease

Keywords

trientine; d-Penicillamine; ATP7B; hepatolenticular degeneration; copper metabolism; zinc

Outcome Measures

Primary Outcomes (1)

• 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.

  5 years

Eligibility Criteria

• Age: 12 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

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.

You may qualify if:

• 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

You may not qualify if:

• \. Refusal of informed consent by either patient or their legally acceptable guardian

Sponsors & Collaborators

• Orphalan: lead
• Ergomed: collaborator

Study Sites (16)

University Hospital Leuven

Leuven, Belgium

RECRUITING

Hospices Civils de Lyon

Bron, Auvergne-Rhône-Alpes, 69677, France

RECRUITING

HF Adolphe de Rothschild

Paris, Île-de-France Region, France

RECRUITING

Charite-Univeritatsmedizin Berlin Hospital

Berlin, Germany

RECRUITING

Universitatsklinikum Dusseldorf

Düsseldorf, Germany

RECRUITING

Hannover Medical School (MHH)

Hanover, Germany

RECRUITING

Universitätsklinikum Leipzig

Leipzig, Germany

RECRUITING

Institute of Psychiatry and Neurology

Warsaw, Poland

RECRUITING

The Children's Memorial Health Institute

Warsaw, Poland

RECRUITING

King Faisal Specialist Hospital in Riyadh

Riyāḑ, Riyadh Region, 11564, Saudi Arabia

RECRUITING

Hospital Universitario GC Dr Negrín

Las Palmas de Gran Canaria, Canary Islands, Spain

RECRUITING

University Hospital Clínic de Barcelona, C. de Villarroel

Barcelona, Spain

RECRUITING

Hospital Universitario Virgen del Rocío

Seville, Spain

RECRUITING

Hospital Universitario Y Politécnico La Fe

Valencia, Spain

RECRUITING

Leeds Teaching Hospitals NHS Trust

Leeds, United Kingdom

RECRUITING

Royal Free Hospital

London, United Kingdom

RECRUITING

MeSH Terms

Conditions

Hepatolenticular Degeneration

Condition Hierarchy (Ancestors)

Liver Diseases; Digestive System Diseases; Basal Ganglia Diseases; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Brain Diseases, Metabolic, Inborn; Brain Diseases, Metabolic; Movement Disorders; Heredodegenerative Disorders, Nervous System; Neurodegenerative Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Metabolism, Inborn Errors; Metal Metabolism, Inborn Errors; Metabolic Diseases; Nutritional and Metabolic Diseases

Study Officials

• Pramod Mistry, PhD

  Yale University Hospital, Conneticut

  STUDY CHAIR

Central Study Contacts

Carla Bennett

CONTACT

+44 (0)7918380893; clinicaloperations@orphalan.com

Maureen Richardson

CONTACT

clinicaloperations@orphalan.com

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Target Duration: 5 Years
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: February 4, 2022
• First Posted: February 15, 2022
• Study Start: June 29, 2022
• Primary Completion (Estimated): December 1, 2027
• Study Completion (Estimated): December 1, 2027
• Last Updated: July 3, 2025

Record last verified: 2025-06

Locations

FR (2); BE (1); PL (2); SA (1); GB (2); DE (4); ES (4)