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

Not yet recruiting

• Conditions: Syringomyelia; Chiari Malformation

• Registration Number: NCT06011226
• Lead Sponsor: Assistance Publique - Hôpitaux de Paris

Brief Summary

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.

Study Timeline

• Status Verified: 2023-07
• Study First Submitted: 2023-08-21
• Study First Submitted QC: 2023-08-21
• Study First Posted: 2023-08-25
• Study Start: 2023-09
• Last Update Submitted: 2023-09-18
• Last Update Posted: 2023-09-21
• Primary Completion: 2025-12
• Study Completion: 2026-12

Recruitment & Eligibility

• Status: NOT_YET_RECRUITING
• Sex: All
• Target Recruitment: 60

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

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
development of metrologic and psychometric features of a patient-reported outcome measure (Bicêtre Chiari and Syringomyelia score - BCS score)	throughout the study (an average of 26 months)	Development of metrologic and psychometric features of a patient-reported outcome measure (Bicêtre Chiari and Syringomyelia score - BCS score) for evaluating functional severity of Chiari malformation and/or syringomyelia
validation of metrologic and psychometric features of a patient-reported outcome	throughout the study (an average of 26 months)	validation of metrologic and psychometric features of a patient-reported outcome measure (Bicêtre Chiari and Syringomyelia score - BCS score) for evaluating functional severity of Chiari malformation and/or syringomyelia

Trial Locations

Locations (1)

Hôpital Bicêtre - Service de Neurochirurgie - CRMR C-MAVEM; 🇫🇷 Le Kremlin-Bicêtre, France