CuidAME: Longitudinal Data Collection From Patients With Spinal Muscular Atrophy in Spain
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: Spinal Muscular Atrophy. Базовые параметры: Без ограничений · 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
- Испания
- Следующий шаг
- Сохраните исследование, покажите его лечащему врачу и уточните актуальный статус у исследовательского центра. Расходы, документы и поездка →
Не всё понятно в терминах? Прочитайте наш гид для пациентов →
Обзор
The goal of this observational study is to set up a longitudinal data collection study to perform a long-term follow-up of SMA patients in a real-world setting, and to standardise and homogenize the data collection process for SMA patient's data. Treated and untreated patients will be followed based on the routine clinical practice schedule.
Подробное описание
This is a retrospective, prospective, multicentre, non-randomized data collection study in Spain. The dataset collected is based on the TREAT-NMD SMA Core Dataset and the CuidAME steering committee consensus. The study collects pseudo-anonymized data, including data on genetic test results, clinical diagnoses, treatment, and outcome measures.
Patients will be followed based on the routine clinical practice schedule. Motor outcomes will be captured regularly, following the SMA standards of for care, for patients with a DMT every 6 months or at least once per year in patients without DMTs, for at least a 5-year follow-up period initially, which could be extended under agreement with funders of the study.
Participation in this study does not modify or influence any clinical decisions regarding the treatment the patient may receive, clinical assessments performed or frequency of visits.
The study will not collect data from patients participating in a clinical trial from the time they are enrolled. Data collected before the enrolment may be used, and data collection may continue after the end of the clinical trial. Data generated during a clinical trial could be used if written permission from the clinical trial sponsor is obtained.
Первичные конечные точки
- Motor and functional status (WHO) [Срок оценки: Yearly through study completion, an average of 5 years]
- Motor and functional status (CHOP INTEND) [Срок оценки: Yearly through study completion, an average of 5 years]
- Motor and functional status (HFMSE) [Срок оценки: Yearly through study completion, an average of 5 years]
- Motor and functional status (RULM) [Срок оценки: Yearly through study completion, an average of 5 years]
- Motor and functional status (6MWT) [Срок оценки: Yearly through study completion, an average of 5 years]
Вторичные конечные точки (5)
- Clinical SMA characteristics: mechanical ventilation [Срок оценки: Through study completion, an average of 5 years]
- Clinical SMA characteristics: external nutrition [Срок оценки: Yearly through study completion, an average of 5 years]
- Clinical SMA characteristics: scoliosis [Срок оценки: Yearly through study completion, an average of 5 years]
- Clinical SMA characteristics: neurodevelopmental developoment [Срок оценки: Yearly through study completion, an average of 5 years]
- Clinical SMA characteristics: treatment sequence [Срок оценки: Yearly through study completion, an average of 5 years]
Критерии участия
Критерии включения
- Genetically confirmed 5q SMA
- Patient or caregivers written informed consent has been obtained (except for paediatric patients deceased).
Критерии исключения
- Further types of SMA (non-5q SMA).
- Patient without legal capacity who cannot understand the nature, significance, and consequences of the project.
Критерии приведены из реестра в оригинале (на английском). Окончательную оценку соответствия проводит исследовательский центр.
Здоровые добровольцы: Нет
Дизайн исследования
- Модель наблюдения
- Когортное
Центры проведения
Испания · 1 центр
- Hospital Sant Joan de Déu — Esplugues de Llobregat
Идентификаторы
NCT: NCT07231549 · EOM-24-02