An observational study in Hemophilia A With Inhibitor and Hemophilia A, sponsored by Enzyre B.V.. Completed at 1 site in Poland. Open to male participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2024-04-10.
Sponsored by Enzyre B.V. · Observational
The goal of this observational study is to assess if the version A of the HemA EnzySystem, a novel portable coagulation testing platform, can be used in patients with hemophilia A treated with Factor VIII Bypassing Agent (FEIBA). The main question[s] it aims to answer are:
Participants are asked to fill in a questionnaire regarding their general health and hemophilia treatment. Subsequently, blood will be drawn from the patients before, and at 30, 120, and 240 minutes after FEIBA administration. Whole blood is immediately tested using the Version A HemA EnzySystem, and plasma is generated for testing with the Ceveron s100 (Technoclone). Leftover samples are frozen for later additional coagulation testing.
Rationale: Hemophilia is an X-linked hemostatic bleeding disorder characterized by a lack of coagulation factor VIII activity (HemA) or factor IX activity (HemB). Spontaneous major bleeding in joints and muscles frequently occur when factor activity levels are low. Currently, there is a growing evidence that the bleeding phenotype of hemophilia A patients is not only reflected by the actual factor VIII activity level, as there is a large variety in bleeding among patients with similar factor VIII activity.
Currently, patients with severe Hemophilia A are treated with either FVIII containing products or Emicizumab. Initially emicizumab was described for patients who developed inhibitors as inhibitors do not interfere with emicizumab. Despite, bleeding complications still may occur and therefore bypassing therapies are still of great importance. Unfortunately, monitoring treatment with these drugs is a challenge.
Thrombin generation profile may be an additional tool in hemophilia patients to differentiate between bleeding phenotype, to guide prophylactic replacement therapy and adjust bypassing therapy, especially in patients treated with emicizumab. Based on recent studies, thrombin generation as a global hemostasis assay offers an opportunity to assess the hemostatic capacity of patients, and therefore has much potential for monitoring therapy. To facilitate this, a hand-held in vitro diagnostic medical device able to simultaneously measure multiple disease biomarkers with a single drop of blood is currently in development, focusing on simultaneous measurements of Factor VIII activity and Thrombin generation.
Objective: The primary objective of this study is to demonstrate that the version A of the HemA EnzySystem can record thrombin generation within a time frame of 60 min in fresh whole blood samples of patients with hemophilia A treated with Factor VIII Bypassing Agent (FEIBA). As secondary objective, the study results will be validated with a Thrombin Generation Assay (TGA) measured in freshly obtained non frozen plasma.
Study design: This is a cross-sectional observational study. All participants are asked to fill a questionnaire prior to blood collection of four tubes. Blood sample measurements will be conducted within a two-hour time frame using the version A of the HemA EnzySystem and the preparation of plasma using a standard TGA assay. Any remaining blood material will be processed and stored.
Study population: The study population consists of 6 patients treated with FEIBA. In total, at least 24 samples are tested, four for each patient (t=0, 30, 120 and 240 minutes).
866 studies on the registry are indexed under Hemophilia A; 137 are open to participants now.
This study's enrollment of 6 is below the median of 80 across 314 observational studies indexed under Hemophilia A.
Browse Hemophilia A studies →Enzyre B.V. is the lead sponsor of 2 studies on the registry; none are open to participants now.
Counted across the registry records on this site, refreshed daily.
Hemophilia A patients at the Institute of Hematology and Transfusion Medicine who are on prophylactic or on-demand FEIBA treatment.
Exclusion Criteria:
NSAIDs; antimicrobial medication; thyroid inhibitors or SSRI's;
Diagnostic Test: Thrombin generation assay (EnzySystem HemA version A - whole blood) · Diagnostic Test: Thrombin generation assay (Ceveron s100 (Technoclone) - fresh plasma) · Diagnostic Test: Additional coagulation tests
Whole blood obtained by venipuncture is immediately tested with the EnzySystem HemA version A), before and 0, 30, 120 and 240 minutes after administration of FEIBA.
Whole blood is centrifuged to obtain plasma. The fresh plasma is tested using the Ceveron s100.
Plasma samples are frozen for later coagulation testing. The following tests are performed: Factor VIII activity von Willebrand Factor antigen levels von Willebrand Factor ristocetin activity levels Prothrombin Fragment 1+2 levels ADAMTS13 activity FVIII antigen levels Nijmegen Hemostasis Assay
Time between venipuncture and TGA result
The primary objective of this study is to demonstrate that the EnzySystem Thrombin Generation assay can record thrombin generation within a time frame of 60 min. Fresh whole blood samples of patients with hemophilia A on prophylactic or on-demand FEIBA treatment are used and tested in a near-patient setting at the Institute of Hematology and Transfusion Medicine (IHiT)
Time frame: 2 hours
Agreement between TGA obtained with the EnzySystem HemA version A and conventional TGA results
Outcome of the TGAlum measured with the version A of the HemA EnzySystem will be compared to the reference TGA which is the standard method of testing at the IHiT to determine patients coagulation status. The reference TGA will be evaluated using the Ceveron by Technoclone
Time frame: 6 hours
Plan to share: No
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This study is completed, as verified in Apr 2024. You cannot join it, but the record below documents what was studied.
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Enzyre B.V.