Monitoring of Anti-TFPI in Hemophilia
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- The protocol lists: blood sampling.
- Who it may be relevant to
- Registry conditions: Hemophilia, Rebalancing Agents, Prophylaxis. Basic parameters: from 18 years · All.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- France
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
Unsure about the terms? Read our patient guide →
Official title
Monitoring of Anti-TFPI in Hemophilia EUREKA
Overview
During the development of anti-TFPI antibodies, thrombin generation assay (TGA) was employed using both in vitro measurements (antibodies added to blood samples) and ex vivo approaches (blood samples from patients in phase II and III trials). While a significant improvement in thrombin generation was observed in all samples from patients with severe hemophilia, no correlation with clinical outcomes could be established. Notably, thrombin peak levels were consistently improved even in patients who experienced bleeding episodes. These measurements were conducted in platelet-poor plasma (PPP) with standard reagents, which may not adequately reflect the hemostatic efficacy of anti-TFPI antibodies given their mechanism of action. It is hypothesized that optimizing reagents and utilizing more appropriate biological materials could enhance TGA sensitivity, as previously demonstrated for monitoring emicizumab. The absence of a laboratory assay to monitor anti-TFPI (tissue factor pathway inhibitor) antibodies poses a significant challenge for managing patients in surgical settings and treating acute severe bleeding. This study aims to develop a reliable assay to evaluate the hemostatic efficacy of anti-TFPI antibodies and their combined procoagulant effect with factor concentrates (FVIII or FIX) or bypassing agents.
Interventions
- Biological blood sampling
One-time peripheral blood draw (10.8 mL total, 4 citrated tubes) collected during a routine clinical visit for thrombin generation testing on platelet-rich and platelet-poor plasma. No additional medical procedures or treatments are involved in the study.
Primary outcome measures
- Development of a Laboratory Assay to Assess Hemostatic Efficacy of Anti-TFPI Antibodies [Time frame: At time of sample analysis (single visit; Day 0)]
Secondary outcome measures (4)
- Analytical Sensitivity of TGA to Anti-TFPI Antibodies [Time frame: Day 0]
- Identification of Optimal TGA Parameters for Monitoring Anti-TFPI Effect [Time frame: Day 0]
- Correlation Between TGA Parameters and Clinical Use of Anti-TFPI Therapies [Time frame: Day 0]
- Detection of Hypercoagulability from Combined Therapy in TGA [Time frame: Day 0]
Eligibility criteria
- Inclusion Criteria :
- \- Male Patient
- 18 years old
- severe hemophilia patients A or B (FVIII < 1%,FIX<=2%)
- on prophylaxis with FVIII or IX concentrates after an adequate washout period of 48h for SHL FVIII molecules, at least 4 days for EHL-FVIII Fc and at least 10 days for EHL-FIX molecules.
- On prophylaxis with Marstacimab
- Willing to participate
- Capable of following protocol procedures under investigator appreciation
- Exclusion Criteria :
- \- patients refusing to provide 4 additional blood tubes for research
- Patient with an other coagulation disorder
- patients who received an injection of FVIII or FIX during the required washout period
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Observational model
- Case-only
Study locations
France · 2 centers
- Groupement hospitalier Est Hôpital Cardilogique Service d'hémostase clinique — Bron
- Centre de Référence Hémophilie et autres déficits rares en protéine de la coagulationCentr — Paris
Identifiers
NCT: NCT07101926 · 69HCL25_0342