International Wilson's Disease Patient Registry (iWilson Registry)
Ориентир для пациента и семьи
Простыми словами
Автоматическая сводка по структурированным данным реестра. Она помогает сориентироваться, но не заменяет официальный протокол или оценку врача.
- Что изучают
- Это наблюдательное исследование: исследуемое лечение участникам по протоколу не назначают.
- Кому может быть актуально
- Состояния в реестре: Wilson's Disease. Базовые параметры: от 12 лет · Все.
- Что важно проверить
- Возраст, диагноз и пол — только базовые ориентиры. Предыдущее лечение, анализы и другие обязательные условия указаны ниже в критериях участия.
- Где проводится
- Бельгия, Франция, Германия, Польша, Саудовская Аравия +2
- Следующий шаг
- Сохраните исследование, покажите его лечащему врачу и уточните актуальный статус у исследовательского центра. Расходы, документы и поездка →
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Обзор
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.
Подробное описание
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
Первичные конечные точки
- Describe the natural history of treated WD [Срок оценки: 5 years]
Критерии участия
Критерии включения
- 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
Критерии исключения
1\. Refusal of informed consent by either patient or their legally acceptable guardian
Критерии приведены из реестра в оригинале (на английском). Окончательную оценку соответствия проводит исследовательский центр.
Здоровые добровольцы: Нет
Дизайн исследования
- Модель наблюдения
- Когортное
Центры проведения
Германия · 4 центра
- Charite-Univeritatsmedizin Berlin Hospital — Berlin
- Universitatsklinikum Dusseldorf — Düsseldorf
- Hannover Medical School (MHH) — Hanover
- Universitätsklinikum Leipzig — Leipzig
Испания · 4 центра
- Hospital Universitario GC Dr Negrín — Las Palmas de Gran Canaria
- University Hospital Clínic de Barcelona, C. de Villarroel — Barcelona
- Hospital Universitario Virgen del Rocío — Seville
- Hospital Universitario Y Politécnico La Fe — Valencia
Франция · 2 центра
- Hospices Civils de Lyon — Bron
- HF Adolphe de Rothschild — Paris
Польша · 2 центра
- Institute of Psychiatry and Neurology — Warsaw
- The Children's Memorial Health Institute — Warsaw
Великобритания · 2 центра
- Leeds Teaching Hospitals NHS Trust — Leeds
- Royal Free Hospital — London
Бельгия · 1 центр
- University Hospital Leuven — Leuven
Саудовская Аравия · 1 центр
- King Faisal Specialist Hospital in Riyadh — Riyāḑ
Идентификаторы
NCT: NCT05239858 · ORPH-131-005