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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
Phase
First received
Last updated
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
Health authority
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 BicĂȘtre 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

Study Design


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 (BicĂȘtre 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 (BicĂȘtre Chiari and Syringomyelia score - BCS score) for evaluating functional severity of Chiari malformation and/or syringomyelia throughout the study (an average of 26 months)
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