An observational study in Syringomyelia and Chiari Malformation, sponsored by Assistance Publique - Hôpitaux de Paris. Not yet recruiting at 1 site in France. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2023-09-21.
Sponsored by Assistance Publique - Hôpitaux de Paris · Observational
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.
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.
33 studies on the registry are indexed under Arnold-Chiari Malformation; 11 are open to participants now.
This study's planned enrollment of 60 is below the median of 152 across 15 observational studies indexed under Arnold-Chiari Malformation.
Browse Arnold-Chiari Malformation studies →Assistance Publique - Hôpitaux de Paris is the lead sponsor of 3,505 studies on the registry; 1,006 are open to participants now.
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The development of a questionnaire for self-assessment of the clinical impact of a pathology necessarily requires the involvement of the patients concerned. In particular, it is important that the clinical heterogeneity of the patients concerned is represented. The study population will therefore be patients with Chiari malformation, whether or not they have associated syringomyelia, and whether or not they have foraminal syringomyelia. They will be recruited via neurosurgical centers belonging to the C-MAVEM (Chiari, syringomyelia and vertebro-medullary malformations) network of rare disease reference centers for Chiari malformations and syringomyelia, and with the help of the main patient association (APAISER).
Criteria common to all subjects included in the study
Patients Chiari only group:
Patients syringomyelia only group:
Patients Chiari with Syringomyelia group:
Exclusion Criteria:
development of the patient reported outcome: The strategy consists of four phases: 1. structured litterature review 2. Group of experts: identification of the main areas to be covered by the questionnaire in order to draw up the focus group moderation guide 3. focus groups: A discussion session will explore patient symptomatology, the functional impact of the disease, quality of life and the problems faced by patients from their point of view.
Other: focus groups
Once the initial version of the self-questionnaire has been developed, it will be tested on a group of 40 patients with either Chiari malformation with syringomyelia (n = 20), isolated Chiari malformation (n = 10) or isolated syringomyelia (n = 10), managed by the CRMR C-MAVEM at Bicêtre Hospital.
Other: questionnaire
the 3rd phase will be submitted to the Human Subjects Protection Review Board according to the progression of the first two phases.
Other: questionnaire
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.
developed questionnaire will be proposed to the patients in phase 2 and 3
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
Time frame: throughout the study (an average of 26 months)
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
Time frame: throughout the study (an average of 26 months)
This study is not yet recruiting, as verified in Jul 2023. You cannot join it, but the record below documents what was studied.
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Assistance Publique - Hôpitaux de Paris