International Wilson's Disease Patient Registry (iWilson Registry)

Wilson's Disease Clinical Trial

Official title:

International Wilson's Disease Patient Registry (iWilson Registry)

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT05239858
• Other study ID #: ORPH-131-005
• Status: Recruiting
• Start date: June 29, 2022
• Est. completion date: December 2027

Study information

• Verified date: April 2024
• Source: Orphalan
• Contact: Frank Verheggen
• Phone: +31625241264
• Email: frank.verheggen[@]orphalan.com
• Is FDA regulated: No
• Study type: Observational [Patient Registry]

Clinical 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. 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.

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:

1. Natural history of diagnosis and treated WD patients by documentation of observed clinical practice and patient experience

2. 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

3. To provide data for epidemiological research and identify groups of patients potentially eligible for multi-centre trials

4. 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.

5. 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.

6. 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

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 500
• Est. completion date: December 2027
• Est. primary completion date: December 2027
• Accepts healthy volunteers: No
• Gender: All
• Age group: 12 Years and older

Eligibility

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

Related Conditions & MeSH terms

• Wilson's Disease

Locations

Country	Name	City	State
Belgium	University Hospital Leuven	Leuven
France	Hospices Civils de Lyon	Bron	Auvergne-Rhone-Alpes
France	HF Adolphe de Rothschild	Paris	Île-de-France
Germany	Charite-Univeritatsmedizin Berlin Hospital	Berlin
Germany	Universitatsklinikum Dusseldorf	Düsseldorf
Germany	Hannover Medical School (MHH)	Hannover
Germany	Universitätsklinikum Leipzig	Leipzig
Poland	Institute of Psychiatry and Neurology	Warsaw
Poland	The Children's Memorial Health Institute	Warsaw
Saudi Arabia	King Faisal Specialist Hospital in Riyadh	Riya?	Ar Riya?
Spain	University Hospital Clínic de Barcelona, C. de Villarroel	Barcelona
Spain	Hospital Universitario GC Dr Negrín	Las Palmas De Gran Canaria	Canary Islands
Spain	Hospital Universitario Virgen del Rocío	Sevilla
Spain	Hospital Universitario Y Politécnico La Fe	Valencia
United Kingdom	Leeds Teaching Hospitals NHS Trust	Leeds
United Kingdom	Royal Free Hospital	London

Sponsors (2)

Lead Sponsor	Collaborator
Orphalan	Ergomed

Countries where clinical trial is conducted

Belgium,  France,  Germany,  Poland,  Saudi Arabia,  Spain,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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