The EUROSCA Natural History Study
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: Spinocerebellar Ataxia. Базовые параметры: от 18 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
- Австрия, Бельгия, Франция, Германия, Венгрия +5
- Следующий шаг
- Сохраните исследование, покажите его лечащему врачу и уточните актуальный статус у исследовательского центра. Расходы, документы и поездка →
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Обзор
The key goals of EUROSCA-NHS is to determine and compare the rate of disease progression in SCA1, SCA2, SCA3 and SCA6 including determination of the order and occurrence of non-ataxia symptoms, assessment of activities of daily living (ADL) and quality of life (QoL), and identification of predictors of disease progression and survival.
Подробное описание
The key goal of EUROSCA-NHS is to determine and compare the rate of disease progression in SCA1, SCA2, SCA3 and SCA6. To this end, a newly developed and validated ataxia scale (Scale for the Assessment and Rating of Ataxia, SARA) will be used. EUROSCA-NHS has a number of secondary aims including determination of the order and occurrence of non-ataxia symptoms, assessment of activities of daily living (ADL) and quality of life (QoL), and identification of predictors of disease progression and survival. Substudies will deal with the development of brain atrophy, as assessed by magnetic resonance imaging (MRI), progression of peripheral neuropathy, as assessed by nerve conduction studies, and specific clinical aspects of SCA.
Первичные конечные точки
- Scale for the assessment and rating of ataxia (SARA) [Срок оценки: Patients are first seen at a baseline visit, followed by annual visits for 3 years scheduled ± 3 months around the specified time point. After the initial 3 year observation period, visits are done at irregular intervals each time they went to hospital.]
Вторичные конечные точки (5)
- Disease stages [Срок оценки: Patients are first seen at a baseline visit, followed by annual visits for 3 years scheduled ± 3 months around the specified time point. After the initial 3 year observation period, visits are done at irregular intervals each time they went to hospital.]
- Inventory of non-ataxia signs (INAS) [Срок оценки: Patients are first seen at a baseline visit, followed by annual visits for 3 years scheduled ± 3 months around the specified time point. After the initial 3 year observation period, visits are done at irregular intervals each time they went to hospital.]
- UHDRS part IV [Срок оценки: Patients are first seen at a baseline visit, followed by annual visits for 3 years scheduled ± 3 months around the specified time point. After the initial 3 year observation period, visits are done at irregular intervals each time they went to hospital.]
- EQ-5D [Срок оценки: Patients are first seen at a baseline visit, followed by annual visits for 3 years scheduled ± 3 months around the specified time point. After the initial 3 year observation period, visits are done at irregular intervals each time they went to hospital.]
- PHQ-9 [Срок оценки: Patients are first seen at a baseline visit, followed by annual visits for 3 years scheduled ± 3 months around the specified time point. After the initial 3 year observation period, visits are done at irregular intervals each time they went to hospital.]
Критерии участия
Критерии включения
- Progressive, otherwise unexplained ataxia
- Positive genetic testing for SCA1, SCA2, SCA3, and SCA6
- Written informed consent by the patient or his legal agent
Критерии исключения
None.
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 · 5 центров
- Department of Neurology, St. Josef Hospital, University Hospital of Bochum — Bochum
- Department of Neurology, University of Bonn — Bonn
- Department of Neurology, University Clinic Essen, University of Duisburg-Essen — Essen
- Department of Neurology, University of Frankfurt — Frankfurt
- Department of Neurodegeneration and Hertie-Institute for Clinical Brain Research, Universi — Tübingen
Венгрия · 2 центра
- Department of Medical Genetics, University of Pecs — Pécs
- Department of Neurology, Zala County Hospital — Zalaegerszeg
Италия · 2 центра
- Fondazione-IRCCS Istituto Neurologico Carlo Besta — Milan
- Department of Neuroscience, Federico II University Naples — Naples
Австрия · 1 центр
- Department of Neurology, Medical University, Innsbruck — Innsbruck
Бельгия · 1 центр
- Université Libre de Bruxelles (ULB), Neurology Service - ULB Hôpital Erasme, ULB Laborator — Brussels
Франция · 1 центр
- Hôpital de la Pitié-Salpêtrière, Département de Génétique — Paris
Нидерланды · 1 центр
- Radboud University Medical Center, Department of Neurology, Donders Institute for Brain, C — Nijmegen
Польша · 1 центр
- Institute of Psychiatry and Neurology — Warsaw
Испания · 1 центр
- University Hospital Marqués de Valdecilla (IDIVAL), University of Cantabria — Santander
Великобритания · 1 центр
- Institute of Neurology — London
Публикации
- Diallo A, Jacobi H, Cook A, Labrum R, Durr A, Brice A, Charles P, Marelli C, Mariotti C, Nanetti L, Panzeri M, Rakowicz M, Sobanska A, Sulek A, Schmitz-Hubsch T, Schols L, Hengel H, Melegh B, Filla A, Antenora A, Infante J, Berciano J, van de Warrenburg BP, Timmann D, Boesch S, Pandolfo M, Schulz JB, Bauer P, Giunti P, Kang JS, Klockgether T, Tezenas du Montcel S. Survival in patients with spinoce PMID 29553382
- Jacobi H, du Montcel ST, Bauer P, Giunti P, Cook A, Labrum R, Parkinson MH, Durr A, Brice A, Charles P, Marelli C, Mariotti C, Nanetti L, Panzeri M, Rakowicz M, Sulek A, Sobanska A, Schmitz-Hubsch T, Schols L, Hengel H, Baliko L, Melegh B, Filla A, Antenora A, Infante J, Berciano J, van de Warrenburg BP, Timmann D, Szymanski S, Boesch S, Kang JS, Pandolfo M, Schulz JB, Molho S, Diallo A, Klockgeth PMID 26377379
Идентификаторы
NCT: NCT02440763 · 010/05