Development of a Patient-reported Outcome Measure for Chiari Malformation and Syringomyelia
Trial statusNot yet recruiting
Trial phaseNot listed
Trial typeObservational
Biological sexAll
Age18+
SponsorAssistance Publique - Hôpitaux de Paris
About this trial
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 criteria
This trial does not accept healthy volunteersQualifiers
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
Disqualifiers
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
Trial population
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).
Trial design
Design model
Case-only
Time perspective
Prospective
Treatments tested in this trial
focus groups
Other interventionA 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
Other interventiondeveloped questionnaire will be proposed to the patients in phase 2 and 3
Treatment groups
60 Participants
are divided into 3 treatment groupsGroup A: Phase 1: development of the questionnaire1 intervention
Group B: Phase 2: pilot phase1 intervention
Group C: Phase 3: national testing of the questionnaire1 intervention
Trial outcomes
Primary outcomes
1
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)2
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)Sponsors and contacts
Click on the lead sponsor to view all of their trials.
Assistance Publique - Hôpitaux de Paris
Lead sponsor
Institut National de la Santé Et de la Recherche Médicale, France
Collaborator
Source ClinicalTrials.gov
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