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Not yet recruiting NCT06011226

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

— Observational Syringomyelia Chiari Malformation

For patients and families

In plain language

An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.

What is being studied
The protocol lists: focus groups, questionnaire.
Who it may be relevant to
Registry conditions: Syringomyelia, Chiari Malformation. Basic parameters: from 18 years · All.
What needs checking
Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
Where it takes place
France
Next step
Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →

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Overview

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.

Detailed description

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.

Interventions

  • Other 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.
  • Other questionnaire
    developed questionnaire will be proposed to the patients in phase 2 and 3

Primary outcome measures

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

Eligibility criteria

Inclusion criteria

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.

Exclusion criteria

  • 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

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: No

Study design

Observational model
Case-only

Study locations

France · 1 center
  • Hôpital Bicêtre - Service de Neurochirurgie - CRMR C-MAVEM — Le Kremlin-Bicêtre

Identifiers

NCT: NCT06011226 · APHP220733 · 2022-A01816-37

Primary sources (government registries)

View this study on ClinicalTrials.gov ↗