A Long-Term Observational Study of Patients With Fucosidosis
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: Fucosidosis. 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
- США, Индия, Нидерланды, Tunisia, Turkey (Türkiye) +1
- Следующий шаг
- Сохраните исследование, покажите его лечащему врачу и уточните актуальный статус у исследовательского центра. Расходы, документы и поездка →
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Официальное название
A Retrospective and Prospective Natural History Study of Patients With Fucosidosis
Обзор
The purpose of this observational research study is to learn more about the natural history of fucosidosis, its symptoms, and how it develops over time. This study intends to collect information from participants diagnosed with fucosidosis; however, this study does not include any medication or treatment other than the usual medical care provided to study participants. The information collected in this study will be used to help understand the disease characteristics of fucosidosis; with this information potentially being able to help design future studies and treatments for this disease. There is currently no approved treatment for patients with fucosidosis. The study consists of 2 parts: a) Part A - retrospective data collection, and b) Part B - prospective data collection.
Подробное описание
Time Perspective: Both retrospective (Part A) and prospective (Part B).
Enrollment: Part A is anticipated to enroll up to 57 participants for retrospective data collection. Part B is anticipated to enroll up to 31 participants for prospective data collection, the majority of whom are expected to be participants also enrolled in Part A.
Первичные конечные точки
- Evaluate the course of disease progression in individuals with fucosidosis who were/are untreated with any investigational products [Срок оценки: Baseline to up to 4 years]
Критерии участия
Inclusion Criteria (Part A):
- Confirmed diagnosis of fucosidosis
- Patient either with or without previous allogeneic hematopoietic stem cell transplant (HSCT) for treatment of fucosidosis
Exclusion Criteria (Part A):
- Patient/parent/caregiver not willing to consent to participate
- Patient deceased with no availability of appropriate historical consent, and patient's family/caregivers are either unable to be contacted, or refuse consent to data sharing
Inclusion Criteria (Part B):
- Patient is living
- Confirmed diagnosis of fucosidosis
- Patient either with or without previous allogeneic HSCT for treatment of fucosidosis
Exclusion Criteria (Part B):
- Patient/parent/caregiver not willing to consent to participate
- Current participation in any other interventional or therapeutic study (exception: patients who have previously received bone marrow / HSCT as treatment for fucosidosis are not excluded)
- Patients who, in the opinion of the site investigator, would be unable or unsuitable to participate in the demands of the study
Критерии приведены из реестра в оригинале (на английском). Окончательную оценку соответствия проводит исследовательский центр.
Здоровые добровольцы: Нет
Дизайн исследования
- Модель наблюдения
- Когортное
Центры проведения
Turkey (Türkiye) · 5 центров
- Cukurova University, Faculty of Medicine — Adana
- Istanbul University Cerrahpasa Medical Faculty Hospital — Istanbul
- Marmara University Pendik Training and Research Hospital — Istanbul
- Ege University, Faculty of Medicine — Izmir
- Dokuz Eylül University Hospital — Izmir
Великобритания · 5 центров
- Royal Belfast Hospital for Sick Children — Belfast
- Birmingham Women's and Children's NHS Foundation Trust — Birmingham
- National Institute for Health and Care Research Clinical Research Facility, Great Ormond S — London
- St. Mary's Hospital, Manchester University NHS Foundation Trust — Manchester
- Barnes Clinical Research Facility, Salford Royal Hospital, Northern Care Alliance NHS Foun — Salford
США · 2 центра
- Children's Hospital of Orange County — Orange
- University of Minnesota Health — Minneapolis
Tunisia · 2 центра
- La Rabta Hospital — Tunis
- National Institute Mongi-Ben Hamida of Neurology of Tunis — Tunis
Индия · 1 центр
- Indira Gandhi Institute of Child Health, Department of Pediatric Neurology — Bengaluru
Нидерланды · 1 центр
- Erasmus University Medical Center — Rotterdam
Идентификаторы
NCT: NCT07615400 · JR-471-NHX