Меню
Набор скоро начнётся NCT06011226

Development of a Patient-reported Outcome Measure for Chiari Malformation and Syringomyelia

— Наблюдательное Syringomyelia Chiari Malformation

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
В протоколе указаны: focus groups, questionnaire.
Кому может быть актуально
Состояния в реестре: Syringomyelia, Chiari Malformation. 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
Франция
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

Chiari malformation corresponds to the herniation of cerebellar tonsils into the foramen magnum resulting in obstruction of cerebrospinal fluid circulation, which may eventually lead to the formation of an intramedullary cavity called syringomyelia. Chiari and syringomyelia can be responsible of variable symptoms, based on which neurosurgeons might propose surgical treatment. Yet, there is no properly developped and validated patient reported outcome measure (PROM) to assess the clinical severity of Chiari malformation and/or syringomyelia. The lack of such evaluation tool is a major issue to determine the optimal therapeutic strategy and to achieve a standardized and reproducible follow-up.

Подробное описание

Chiari malformation corresponds to the herniation of cerebellar tonsils into the foramen magnum resulting in obstruction of cerebrospinal fluid circulation, which may eventually lead to the formation of an intramedullary cavity called syringomyelia.

Chiari and syringomyelia can be responsible of variable symptoms, based on which neurosurgeons might propose surgical treatment. Yet, there is no properly developped and validated patient reported outcome measure (PROM) to assess the clinical severity of Chiari malformation and/or syringomyelia. The lack of such evaluation tool is a major issue to determine the optimal therapeutic strategy and to achieve a standardized and reproducible follow-up. The project will consist of two successive phases: a survey design of the questionnaire with small selected groups of patients; a monocentric pilot study on a limited population; The purpose of the development study is to produce a first version of the PROM based on proposed items written by experts and tested with volunteer patients (n = 10-20) within the framework of " focus groups" followed by cognitive debriefings. These items will explore various dimensions of the functional impact of Chiari malformation and syringomyelia (pain, motor disability, sphincter disorders, quality of life, etc.). The pilot study will test this 1st version of the questionnaire on a limited population of patients (n = 40) within the CRMR C-MAVEM of Bicêtre hospital to produce a final version. This work will be supplemented subsequently by a multicenter study allowing to validate a simple and reproducible evaluation tool in order to ensure the follow-up of patients with a Chiari malformation and/or syringomyelia and to measure surgical outcome.

Вмешательства

  • Другое focus groups
    A dozen patients with Chiari malformation and syringomyelia (n = 4) or isolated Chiari malformation (n = 4) or isolated syringomyelia (n = 4) will be brought together in at least two focus groups. Each focus group will include 5 to 8 patients and will last approximately 1 to 2 hours. The focus groups will be recorded (audio only, no personal data will be collected during these sessions) and will be conducted in the presence of a neurosurgeon and a psychologist, who will moderate the session.
  • Другое questionnaire
    developed questionnaire will be proposed to the patients in phase 2 and 3

Первичные конечные точки

  • development of metrologic and psychometric features of a patient-reported outcome measure (Bicêtre Chiari and Syringomyelia score - BCS score) [Срок оценки: throughout the study (an average of 26 months)]
  • validation of metrologic and psychometric features of a patient-reported outcome [Срок оценки: throughout the study (an average of 26 months)]

Критерии участия

Критерии включения

Criteria common to all subjects included in the study

  • Age > 18 years
  • Subject understanding French Patients included in Phase I
  • Subject with signed informed consent Patients included in phase II
  • Patient not opposed to study participation

Patients Chiari only group:

  • Isolated descent of cerebellar tonsils > 5 mm below McRae's line (no associated syringomyelia)

Patients syringomyelia only group:

  • Presence of an intramedullary fluid cavity secondary to circulatory disturbance of cerebrospinal fluid of non foraminal origin (no Chiari)

Patients Chiari with Syringomyelia group:

  • Presence of Chiari malformation (tonsils > 5 mm below McRae's line) AND foraminal syringomyelia.

Критерии исключения

  • Insufficient command of French
  • Minor or protected adult (guardianship, curatorship, safeguard of justice)
  • Diagnosis of Chiari and/or syringomyelia not proven by an MRI scan
  • Pregnant or breast-feeding

Критерии приведены из реестра в оригинале (на английском). Окончательную оценку соответствия проводит исследовательский центр.

Здоровые добровольцы: Нет

Дизайн исследования

Модель наблюдения
Только случаи

Центры проведения

Франция · 1 центр
  • Hôpital Bicêtre - Service de Neurochirurgie - CRMR C-MAVEM — Le Kremlin-Bicêtre

Идентификаторы

NCT: NCT06011226 · APHP220733 · 2022-A01816-37

Первоисточники (государственные реестры)

Открыть это исследование на ClinicalTrials.gov ↗