Hemophilia A; Hemophilia B
Conditions
Brief summary
The primary objective of this study is to assess the pharmacokinetics in patients with severe hemophilia. The secondary objective is to assess the pharmacodynamics of BAY1093884 based on tissue factor pathway inhibitor activity
Interventions
DRUGBAY1093884
0.3 mg/kg given intravenously
Sponsors
Bayer
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
BASIC_SCIENCE
Masking
NONE
Eligibility
Sex/Gender
MALE
Age
18 Years to 65 Years
Healthy volunteers
No
Inclusion criteria
* Males with severe congenital hemophilia A or B defined as \<1% FVIII or \<2% FIX concentration by measurement at the time of screening or from reliable prior documentation (e.g., measurement in other clinical Bayer trials, or diagnostic genetic testing) * Male with any inhibitor titer at screening or prior to screening at any time from medical records. Subjects may be receiving a bypassing agent (rFVIIa; NovoSeven and/or aPCC; FEIBA) for treatment. * Age: 18 to 65 years at screening * BMI: 18 to 29.9 kg/m2
Exclusion criteria
* Subjects with known bleeding disorders (such as von Willebrand factor \[vWF\] deficiency, FXI deficiency, platelet disorders, or known acquired or inherited thrombophilia etc.) other than congenital Hemophilia A or B with or without inhibitors * History of angina pectoris or treatment for angina pectoris * History of coronary and/or peripheral atherosclerotic disease, congestive heart failure, disseminated intravascular coagulopathy, or stage 2 hypertension defined as systolic blood pressure (SBP) ≥160 mmHg or diastolic blood pressure (DBP) ≥100 mmHg even if controlled * History of thrombophlebitis, venous/arterial thromboembolic diseases (particularly deep vein thrombosis, pulmonary embolism, stroke, myocardial infarction, cerebrovascular accident, ischemic heart disease, transient ischemic attack) * Known or suspected hypersensitivity of the immune system, history of anaphylactic reaction, known severe allergies, non-allergic drug reactions, or multiple drug allergies * Subjects with inhibitors treated with FEIBA, who are not willing to accept rFVIIa (NovoSeven) for the treatment of any bleeds occurring either between screening and dosing or after study drug administration, and until the end of the study.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| AUC (0-tlast) of BAY1093884 in plasma | Up to 15 days after drug administration | Area under the concentration vs. time curve from time 0 to the last data point \> LLOQ |
| AUC(0-tlast)/D of BAY1093884 in plasma | Up to 15 days after drug administration | AUC(0-tlast) divided by dose |
| Cmax of BAY1093884 in plasma | Up to 15 days after drug administration | Maximum observed drug concentration in measured matrix after single dose administration |
| Cmax/D of BAY1093884 in plasma | Up to 15 days after drug administration | Cmax divided by dose |
Secondary
| Measure | Time frame |
|---|---|
| Tissue factor plasma inhibitor activity: effect of BAY1093884 to inhibit the anticoagulatory activity of plasma TFPI as assessed by a chromogenic assay | Up to 15 days after drug administration |
Countries
Israel
Outcome results
None listed