Impact of Von Willebrand Factor-platelet Aggregates in Patients With Type 2B Disease

Not Applicable

• Conditions: Von Willebrand Disease, Type 2B

• Registration Number: NCT05015244
• Lead Sponsor: University Hospital, Caen

Brief Summary

The working hypothesis is that patients with Von Willebrand Disease-type 2B may have neurological symptoms due to the chronic formation of platelets/Von Willebrand Factor aggregates. Interestingly, several patients present nonspecific neurological symptoms (nystagmus, headaches, memory disorder, ...) which may be associated with cerebral microangiopathy and chronic microvessel inflammation secondary to Von Willebrand Disease-type 2B due to chronic exposure to Von Willebrand Factor-platelets complexes.

Detailed Description

Investigator will use an extensive series of neuropsychological tests that cover virtually all cognitive domains. A measurement of global cognitive function will be assessed by the Mini Mental State Examination (MMSE). The verbal memory function will be assessed by the Rey Auditory Verbal Learning Test (RAVLT). Visuospatial memory will be administered by the Rey's Complex Figure Test (RCFT). To evaluate speed of mental processes, Stroop test (three subtasks), the Paper and Pencil Memory Scanning Task (four subtasks), the Symbol-Digit Substitution Task will be used. To evaluate attention, the verbal series attention test (VSAT) will be used. To register subjective cognitive failures investigator will administer the Cognitive failures questionnaire (CFQ). The neurological evaluation will be completed with 3T (Tesla) Magnetic Resonance Imaging (MRI) with different MRI sequences including T1 3D SPACE, FLAIR 3D , 3D SWI (Susceptibility weighted imaging) and 2D EPI (Echo Planar Imaging) to address the presence of cerebral small vessel disease. The presence of each of the four MRI markers for cerebral small vessel disease (white matter hyperintensities, lacunes, cerebral microbleeds and perivascular spaces will be addressed.

Study Timeline

• Study Start: 2019-06-01
• Study First Submitted: 2021-07-20
• Status Verified: 2021-08
• Study First Submitted QC: 2021-08-19
• Study First Posted: 2021-08-20
• Last Update Submitted: 2022-08-23
• Last Update Posted: 2022-08-24
• Primary Completion: 2023-12
• Study Completion: 2023-12

Recruitment & Eligibility

• Status: UNKNOWN
• Sex: All
• Target Recruitment: 5

Inclusion Criteria

• patients with type 2B disease
• neurological signs
• signature of inform consent

Exclusion Criteria

• contraindication for MRI
• Pregnant women
• other hemorrhagic disease

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Primary Outcome Measures

Name	Time	Method
Rate of cerebral lesions in patients with type 2B disease	at the inclusion	Analysis of MRI sequences and % of patients with cerebral lesions

Secondary Outcome Measures

Name	Time	Method
Verbal memory function evaluation	at the inclusion	Rey Auditory Verbal Learning Test (RAVLT) will be used
Attention evaluation	at the inclusion	Verbal series attention test (VSAT) will be used
Subjective cognitive failures evaluation	at the inclusion	Cognitive failures questionnaire (CFQ) will be used
Global cognitive function evaluation	at the inclusion	Mini Mental State Examination (MMSE) will be used
Speed of mental processes evaluation	at the inclusion	Stroop test will be used
Visuospatial memory evaluation	at the inclusion	Rey's Complex Figure Test (RCFT) will be used

Trial Locations

Locations (1)

University Hospital; 🇫🇷 Caen, Calvados, France