Impact of Von Willebrand Factor-platelet Aggregates in Patients With Type 2B Disease (Von2B)

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.

Study Overview

• Status: Recruiting
• Conditions: Von Willebrand Disease, Type 2B
• Intervention / Treatment: Procedure: 3T (Tesla) Magnetic Resonance Imaging (MRI)

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 Type: Interventional
• Enrollment (Anticipated): 5
• Phase: Not Applicable

Contacts and Locations

Study Locations

• France
  • Calvados
    • Caen, Calvados, France, 14000
      • Recruiting
      • University Hospital
      • Contact: Frederic VARNIER; Phone Number: +33 0231063106; Email: directiongenerale@chu-caen.fr

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years to 70 years (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

Inclusion Criteria:

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

Exclusion Criteria:

• contraindication for MRI
• Pregnant women
• other hemorrhagic disease

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Basic Science
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Von Willebrand Disease Type 2B patient with neurological symptoms; evaluation of patients using 3T (Tesla) Magnetic Resonance Imaging (MRI) and series of neuropsychological tests that cover virtually all cognitive domains.	Procedure: 3T (Tesla) Magnetic Resonance Imaging (MRI); Neurological evaluation using MRI and neuropsychological tests; Other Names: Series of neuropsychological tests that cover virtually all cognitive domains

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Rate of cerebral lesions in patients with type 2B disease	Analysis of MRI sequences and % of patients with cerebral lesions	at the inclusion

Secondary Outcome Measures

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

Collaborators and Investigators

• Sponsor: University Hospital, Caen

Study record dates

Study Major Dates

• Study Start (Actual): June 1, 2019
• Primary Completion (Anticipated): December 1, 2023
• Study Completion (Anticipated): December 1, 2023

Study Registration Dates

• First Submitted: July 20, 2021
• First Submitted That Met QC Criteria: August 19, 2021
• First Posted (Actual): August 20, 2021

Study Record Updates

• Last Update Posted (Actual): August 24, 2022
• Last Update Submitted That Met QC Criteria: August 23, 2022
• Last Verified: August 1, 2021

More Information

Terms related to this study

• Keywords: VWF; Type 2B von willebrand disease
• Additional Relevant MeSH Terms: Hematologic Diseases; Blood Coagulation Disorders, Inherited; Coagulation Protein Disorders; Hemorrhagic Disorders; Genetic Diseases, Inborn; Blood Coagulation Disorders; Blood Platelet Disorders; Von Willebrand Diseases; Von Willebrand Disease, Type 2
• Other Study ID Numbers: 2018-A03005-50

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: UNDECIDED

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No