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

NCT06011226 | Not Yet Recruiting

• 2 other identifiers: APHP2207332022-A01816-37
• Study Type: observational
• Target: 60
• Locations: 1 country
• Sites: 1

Brief 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.

Conditions

Syringomyelia; Chiari Malformation

Keywords

Syringomyelia; Chiari Malformation; patient-reported outcome measure

Outcome Measures

Primary Outcomes (2)

• 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)

• 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)

Study Arms (3)

Phase 1: development of the questionnaire

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

Phase 2: pilot phase

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

Phase 3: national testing of the 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

Interventions

focus groups OTHER

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.

Phase 1: development of the questionnaire

questionnaire OTHER

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

Phase 2: pilot phase; Phase 3: national testing of the questionnaire

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)
• Sampling Method: Probability Sample

Study 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).

You may qualify if:

• 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.

You may not qualify if:

• 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

Sponsors & Collaborators

• Assistance Publique - Hôpitaux de Paris: lead
• Institut National de la Santé Et de la Recherche Médicale, France: collaborator

Study Sites (1)

Hôpital Bicêtre - Service de Neurochirurgie - CRMR C-MAVEM

Le Kremlin-Bicêtre, 94270, France

Location

MeSH Terms

Conditions

Syringomyelia; Arnold-Chiari Malformation

Interventions

Focus Groups; Surveys and Questionnaires

Condition Hierarchy (Ancestors)

Spinal Cord Diseases; Central Nervous System Diseases; Nervous System Diseases; Neural Tube Defects; Nervous System Malformations; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Intervention Hierarchy (Ancestors)

Data Collection; Epidemiologic Methods; Investigative Techniques; Health Care Evaluation Mechanisms; Quality of Health Care; Health Care Quality, Access, and Evaluation; Public Health; Environment and Public Health

Study Officials

• Steven KNAFO

  Assistance Publique - Hôpitaux de Paris, Hôpital Bicêtre

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Steven KNAFO, MD

CONTACT

01 45 21 22 88; steven.knafo@aphp.fr

Study Design

• Study Type: observational
• Observational Model: CASE ONLY
• Time Perspective: PROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: August 21, 2023
• First Posted: August 25, 2023
• Study Start: September 1, 2023
• Primary Completion: December 1, 2025
• Study Completion (Estimated): December 1, 2026
• Last Updated: September 21, 2023

Record last verified: 2023-07

Locations

FR (1)