International registry for Wilson's Disease patients

International Wilson's Disease Patient Registry (iWilson Registry)

This study is gathering information from people with Wilson's Disease during their regular doctor visits to better understand the disease and how patients are managing it.

Quick facts

• Study type: Observational
• Enrollment: 500 (estimated)
• Ages: 12 Years and up
• Sex: All
• Sponsor: Orphalan (Industry-sponsored)
• Locations: 16 sites (Leuven and 15 other locations)
• Trial ID: NCT05239858 on ClinicalTrials.gov

What this trial studies

This observational registry collects data from Wilson's Disease patients during routine clinic visits at 6-12 month intervals. It aims to document the natural history of the disease, measure clinical aspects, and provide a comprehensive overview of patient experiences and treatment adherence. The registry will utilize standardized tools for data collection and will include a diverse patient population, including children and adults. Additionally, it seeks to enhance the generalizability of findings by comparing real-world outcomes with clinical trial data.

Who should consider this trial

Good fit: Ideal candidates include all patients diagnosed with Wilson's Disease, including those who are pre-symptomatic or have co-morbidities.

Not a fit: Patients who refuse to provide informed consent will not benefit from this registry.

Why it matters

Potential benefit: If successful, this registry could lead to improved management standards and better treatment outcomes for patients with Wilson's Disease.

How similar studies have performed: Other observational registries have shown success in improving disease management and patient outcomes, indicating that this approach is promising.

Eligibility criteria

Inclusion Criteria:

1. Patient is able to provide, and has provided, written informed consent/assent
2. Written documentation has been obtained in accordance with the relevant country and local privacy requirements, where applicable, including:

   1. For US sites: Authorization for Use and Release of Health Research Study Information
   2. For EU sites: Data Protection Consent
3. All patients diagnosed with WD including pre-symptomatic individuals and individuals with co-morbidities/diagnoses
4. Any treatments including prescribed and homeopathic/traditional therapies or naive patients on no therapy

Exclusion Criteria:

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

Where this trial is running

Leuven and 15 other locations

• University Hospital Leuven — Leuven, Belgium (Recruiting)
• Hospices Civils de Lyon — Bron, Auvergne-Rhône-Alpes, 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, 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)

Study contacts

• Study coordinator: Carla Bennett

Conditions Wilson's Disease; trientine; d-Penicillamine; ATP7B; hepatolenticular degeneration; copper metabolism; zinc