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About
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.
Full 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:
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
Enrollment
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Volunteers
Inclusion criteria
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
Exclusion criteria
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Central trial contact
Frank Verheggen
Data sourced from clinicaltrials.gov
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