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

Condition(s) studied

Investigational drug(s) / intervention(s)

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

Study 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.

Eligibility

Primary outcome measure(s)

• 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 sites (1)

More Assistance Publique - Hôpitaux de Paris trials in France

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