Von Willebrand Disease, Type 2BVon2BNCT05015244
Impact of Von Willebrand Factor-platelet Aggregates in Patients With Type 2B Disease
A study testing 3T (Tesla) Magnetic Resonance Imaging (MRI) in people with Von Willebrand Disease, Type 2B, run by University Hospital, Caen at 1 site in France.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 to 70 years
- Conditions the study is for
- Von Willebrand Disease, Type 2B
- What is being tested?
- 3T (Tesla) Magnetic Resonance Imaging (MRI)
- Type of treatment
- Surgery or procedure
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 5 participants
- Where
- 1 siteFrance
- Study dates
- 1 Jun 2019to Dec 2023
- Phase
- Interventional
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 70 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- patients with type 2B disease
- neurological signs
- signature of inform consent
Who cannot join
- contraindication for MRI
- Pregnant women
- other hemorrhagic disease
Trial locations and contacts
1 site in 1 country.
France1 site, 1 recruiting
- Recruiting
University Hospital
Caen, Calvados, 14000
Frederic VARNIER, 0231063106 ext. +33, directiongenerale@chu-caen.fr
About this study
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.
Read the 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.
- Conditions
- Von Willebrand Disease, Type 2B
- Being tested
- 3T (Tesla) Magnetic Resonance Imaging (MRI)
- Keywords
- VWF, Type 2B von willebrand disease
- Medical subject headings
- von Willebrand Disease, Type 2
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
3T (Tesla) Magnetic Resonance Imaging (MRI)Procedure or surgery
Also called Series of neuropsychological tests that cover virtually all cognitive domains
Neurological evaluation using MRI and neuropsychological tests
| Study group | Role | What they receive |
|---|---|---|
| Von Willebrand Disease Type 2B patient with neurological symptoms | Experimental | evaluation of patients using 3T (Tesla) Magnetic Resonance Imaging (MRI) and series of neuropsychological tests that cover virtually all cognitive domains. Receives: Procedure: 3T (Tesla) Magnetic Resonance Imaging (MRI) |
What is being measured
The primary outcome is the main question the study is designed to answer.
Rate of cerebral lesions in patients with type 2B disease
Analysis of MRI sequences and % of patients with cerebral lesions
Measured: at the inclusion
Secondary outcomes (6)
Global cognitive function evaluation
Mini Mental State Examination (MMSE) will be used
Measured: at the inclusion
Verbal memory function evaluation
Rey Auditory Verbal Learning Test (RAVLT) will be used
Measured: at the inclusion
Visuospatial memory evaluation
Rey's Complex Figure Test (RCFT) will be used
Measured: at the inclusion
Speed of mental processes evaluation
Stroop test will be used
Measured: at the inclusion
Attention evaluation
Verbal series attention test (VSAT) will be used
Measured: at the inclusion
Subjective cognitive failures evaluation
Cognitive failures questionnaire (CFQ) will be used
Measured: at the inclusion
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted20 Aug 2021
- Study start1 Jun 2019
- Primary completionDec 2023 (estimated)
- Study completionDec 2023 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Von Willebrand Disease, Type 2BStudies of the same condition, open and under way.
- More trials run by University Hospital, CaenThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Basic science
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 5 participants (estimated)
Sponsor and investigators
- Lead sponsor
- University Hospital, Caen
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05015244
- Sponsor's protocol ID
- 2018-A03005-50
- Acronym
- Von2B
- Status last verified
- Aug 2021
- First submitted
- 20 Jul 2021
- Last updated
- 24 Aug 2022
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Study at a glance
From the registry record, last updated by the sponsor on 24 Aug 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Impact of Von Willebrand Factor-platelet Aggregates in Patients With Type 2B Disease
- ClinicalTrials.gov ID
- NCT05015244
- Acronym / sponsor ID
- Von2B / 2018-A03005-50
- Conditions
- Von Willebrand Disease, Type 2B
- Keywords
- VWF, Type 2B von willebrand disease
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 5 participants (estimated)
- Lead sponsor
- University Hospital, CaenUniversity, hospital or other
- Study start
- 1 Jun 2019 (actual)
- Primary completion
- Dec 2023 (estimated)
- Study completion
- Dec 2023 (estimated)
- First posted
- 20 Aug 2021 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Primary purpose
- Basic science
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Von Willebrand Disease Type 2B patient with neurological symptoms | Experimental | Procedure: 3T (Tesla) Magnetic Resonance Imaging (MRI)evaluation of patients using 3T (Tesla) Magnetic Resonance Imaging (MRI) and series of neuropsychological tests that cover virtually all cognitive domains. |
Who may qualify
- Ages
- 18 to 70 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (3 listed)
- patients with type 2B disease
- neurological signs
- signature of inform consent
Key exclusion criteria (3 listed)
- contraindication for MRI
- Pregnant women
- other hemorrhagic disease
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| 3T (Tesla) Magnetic Resonance Imaging (MRI)Also: Series of neuropsychological tests that cover virtually all cognitive domainsNeurological evaluation using MRI and neuropsychological tests | Procedure or surgery | Von Willebrand Disease Type 2B patient with neurological symptoms |
Primary outcome
Rate of cerebral lesions in patients with type 2B disease
Time frame: at the inclusion
Analysis of MRI sequences and % of patients with cerebral lesions
6 secondary outcomes
Global cognitive function evaluation
Time frame: at the inclusion
Verbal memory function evaluation
Time frame: at the inclusion
Visuospatial memory evaluation
Time frame: at the inclusion
Speed of mental processes evaluation
Time frame: at the inclusion
Attention evaluation
Time frame: at the inclusion
Subjective cognitive failures evaluation
Time frame: at the inclusion
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 24 Aug 2022.
- Individual participant dataSharing planned: Undecided
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The Severe Von Willebrand Disease (sVWD) Patient Registry
VWD - Von Willebrand's Disease
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Efficacy and Safety of BT200 (Rondaptivon Pegol) in Patients With Type 2B Von Willebrand Disease
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing 3T (Tesla) Magnetic Resonance Imaging (MRI) in people with Von Willebrand Disease, Type 2B, run by University Hospital, Caen at 1 site in France.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 3 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 24 Aug 2022. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in France. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Each site lists its own contact: choose the nearest one under Trial locations. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Rate of cerebral lesions in patients with type 2B disease (time frame: at the inclusion). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 24 Aug 2022.