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Clinical Trial Details — Status: Recruiting

Administrative data

NCT number NCT05239858
Other study ID # ORPH-131-005
Secondary ID
Status Recruiting
Phase
First received
Last updated
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
Health authority
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

Study Design


Related Conditions & MeSH terms


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
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