Hemophilia A, Hemophilia B
Conditions
Brief summary
This is the first in humans study of BAY86-6150 (B0189) in non-bleeding subjects with moderate or severe congenital hemophilia A or B with or without inhibitors. This is a randomized, double-blind, placebo-controlled, single-dose, dose escalation study. It is designed to investigate the safety, tolerability, potential immunogenicity, pharmacokinetic and pharmacodynamic profile of BAY86-6150 (B0189) and to determine a dose or range of doses to be examined in subsequent studies.
Interventions
DRUGBAY Factor VII (BAY86-6150)
BAY Factor VII (BAY86-6150), 6.5 µg/kg body weight, will be administered as a slow intravenous (i.v.) administration over a period of 2-5 minutes (min) on Study Day 1.
DRUGPlacebo
Placebo will be administered as a slow intravenous (i.v.) administration over a period of 2-5 minutes (min) on Study Day 1.
Sponsors
Bayer
Study design
Allocation
RANDOMIZED
Intervention model
FACTORIAL
Primary purpose
TREATMENT
Masking
DOUBLE (Subject, Investigator)
Eligibility
Sex/Gender
MALE
Age
18 Years to 65 Years
Healthy volunteers
No
Inclusion criteria
* History of moderate or severe congenital hemophilia A or B with or without inhibitors to Factor VIII (FVIII) or Factor IX (FIX) * Male subjects 18-65 years of age inclusive * Able to dismiss factor replacement therapy during the course of the study unless required for the treatment of an acute bleeding episode * Written informed consent * Willing and able to comply with the requirements of the protocol * Have adequate venous access * Willing to use an effective method of contraception until Day 30 of their study participation
Exclusion criteria
* Received factor replacement therapy or treatment with any other procoagulant therapeutics, or any antifibrinolytic agents, including blood products, at anytime within 5 days prior to administration of investigational medicinal product (IMP) * Planned administration of factor replacement therapy or treatment with any other procoagulant therapeutics or any antifibrinolytic agents, including blood products, at anytime during the study period * Acute bleeding episode or any ongoing bleeding episode at any time within 7 days prior to administration IMP * Clinically relevant coagulation disorder other than congenital hemophilia A or B * History of angina or receiving treatment for angina * History of coronary atherosclerotic disease, disseminated intravascular coagulopathy, or stage 2 hypertension defined as systolic blood pressure (SBP) \>/= 160 mmHg or diastolic blood pressure (DBP) \>/= 90 mmHg * History of transient ischemic attack, stroke, myocardial infarction, coronary artery disease, congestive heart failure, or thromboembolic event * Active infection on day of IMP administration or septicemia at any time within 30 days prior to administration of IMP
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Number of participants with adverse events as a measure of safety and tolerability | Up to Day 50 |
Secondary
| Measure | Time frame |
|---|---|
| Pharmacokinetic assessment, based on plasma concentration of BAY86-6150 | 9 time points from pre-dosing on Day 1 up to 48 hours post-dosing |
| Pharmacodynamic assessment, based on plasma hemostasis marker level | 9 time points from pre-dosing on Day 1 up to 48 hours post-dosing |
| Immunogenicity assessment, based on anti-BAY86-6150 binding antibody levels | 3 time points from pre-dosing on Day 1 up to Day 50 |
Countries
Poland, South Africa, United Kingdom
Outcome results
None listed