Targeting TFPI With Concizumab to Improve Haemostasis in Glanzmann Thrombasthenia Patients: an in… (NCT06234813) | Clinical Trial Compass
CompletedNot Applicable
Targeting TFPI With Concizumab to Improve Haemostasis in Glanzmann Thrombasthenia Patients: an in Vitro Study
France20 participantsStarted 2023-04-06
Plain-language summary
Glanzmann thrombasthenia is a rare genetic disorder caused by the absence or the dysfunction of the main receptor present on the surface of platelets, integrin αIIbβ3 or GPIIb-IIIa.
The lack of this protein on the surface of platelets no longer allows these blood cells to bind to each other. This binding corresponds to the process of platelet aggregation.
Generally, local measures will control nasal and superficial bleeding whereas platelet transfusions are used to control or prevent life-threatening.
The main complication of this treatment is the risk of developing anti-αIIbβ3 antibodies directed against the absent protein and platelet transfusion therapy can become ineffective.
Activated recombinant factor VII (rFVIIa) provides an alternative treatment for GT patients who develop such antibodies. However, this therapy has a short duration of efficacy, requiring repeated intravenous administrations every 2 to 3 hours.
There is a new treatment, Concizumab, which has not yet been marketed. This treatment acts on TFPI (tissue factor pathway inhibitor). TFPI is a protein that occurs naturally in the body and prevents blood cells from binding to each other.
Concizumab works by blocking TFPI, which may allow sufficient clotting to prevent bleeding.
This treatment could replace recombinant activated factor VII (rFVIIa) because it has the advantage of a much longer duration of efficacy (about 3 days) and is administered subcutaneously.
Who can participate
Age range
18 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion Criteria:
Glanzmann Thrombasthenia Group:
* Patient ≥18 years old
* Patient with a clear diagnosis of Glanzmann Thrombasthenia (GT), whatever the subtype of disease
* Affiliated person or beneficiary of a social security scheme.
* Free, informed and written consent signed by the participant, and the investigator (at the latest on the day of inclusion and before any examination required by the research)
Control Group:
* Healthy donor ≥ 18 years old
* Healthy donor, without haemorrhagic ant thrombotic medical history
* Person should not work in the investigator's department.
* Affiliated person or beneficiary of a social security scheme
* Free, informed and written consent signed by the participant, and the investigator (at the latest on the day of inclusion and before any examination required by the research)
Exclusion Criteria:
For both patient groups:
* Patient who has taken aspirin or a nonsteroidal anti-inflammatory medication within the previous 10 days
* Patient who has received a platelet transfusion or recombinant activated factor VII hemostatic treatment within the previous 7 days
* Patient who participated in another interventional study involving a drug within 30 days of entering this protocol
* Psychiatric, social or behavioral condition judged to be non-compatible with the respect of the protocol
* Adult protected by the law
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
Effects of mixing concizumab compared with the main bleed treatment options for persons with GT