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

Syringomyelia Clinical Trial

— BCS-score  

Official title:

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

Clinical Trial Details — Status: Not yet recruiting

Administrative data

• NCT number: NCT06011226
• Other study ID #: APHP220733
• Secondary ID: 2022-A01816-37
• Status: Not yet recruiting
• Start date: September 2023
• Est. completion date: December 2026

Study information

• Verified date: July 2023
• Source: Assistance Publique - Hôpitaux de Paris
• Contact: Steven KNAFO, MD
• Phone: 01 45 21 22 88
• Email: steven.knafo[@]aphp.fr
• Is FDA regulated: No
• Study type: Observational

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Not yet recruiting
• Enrollment: 60
• Est. completion date: December 2026
• Est. primary completion date: December 2025
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

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

Related Conditions & MeSH terms

• Arnold-Chiari Malformation
• Chiari Malformation
• Congenital Abnormalities
• Syringomyelia

Intervention

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

Locations

Country	Name	City	State
France	Hôpital Bicêtre - Service de Neurochirurgie - CRMR C-MAVEM	Le Kremlin-Bicêtre

Sponsors (2)

Lead Sponsor	Collaborator
Assistance Publique - Hôpitaux de Paris	Institut National de la Santé Et de la Recherche Médicale, France

Country where clinical trial is conducted

France, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	development of metrologic and psychometric features of a patient-reported outcome measure (Bicêtre Chiari and Syringomyelia score - BCS score)	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	throughout the study (an average of 26 months)
Primary	validation of metrologic and psychometric features of a patient-reported outcome	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	throughout the study (an average of 26 months)