Clinical trial NCT03481946: A Study to Assess Pharmacokinetics and Pharmacodynamics Following Administration of BAY1093884 in Patients With Severe Hemophilia
Official title: A Phase 1 Study to Assess Pharmacokinetics and Pharmacodynamics Following Administration of BAY1093884 in Patients With Severe Hemophilia
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE1
Conditions: Hemophilia A; Hemophilia B
Lead sponsor: Bayer
Sponsor class: INDUSTRY
First posted: 2018-03-29
Enrollment: 6
Sex: MALE
Minimum age (years): 18
Maximum age (years): 65
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: BASIC_SCIENCE

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


Eligibility criteria: 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.

Primary outcomes:
measure: AUC (0-tlast) of BAY1093884 in plasma
timeFrame: Up to 15 days after drug administration
measure: AUC(0-tlast)/D of BAY1093884 in plasma
timeFrame: Up to 15 days after drug administration
measure: Cmax of BAY1093884 in plasma
timeFrame: Up to 15 days after drug administration
measure: Cmax/D of BAY1093884 in plasma
timeFrame: Up to 15 days after drug administration
Secondary outcomes:
measure: Tissue factor plasma inhibitor activity: effect of BAY1093884 to inhibit the anticoagulatory activity of plasma TFPI as assessed by a chromogenic assay
timeFrame: Up to 15 days after drug administration
Other outcomes:
Locations:
city: Ramat Gan
country: Israel
facility: Chaim Sheba Medical Center
Study officials:
References: