Clinical trial NCT01921855: Study of FVIIa Variant BAY86-6150 (B0189) in Subjects With Moderate or Severe Hemophilia Types A or B With or Without Inhibitors
Official title: A Phase I, Randomized, Double-blind, Placebo Controlled, Single Dose Escalation Study of FVIIa Variant BAY86-6150 (B0189) in Subjects With Moderate or Severe Hemophilia Types A or B With or Without Inhibitors
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE1
Conditions: Hemophilia A
Hemophilia B
Lead sponsor: Bayer
Sponsor class: INDUSTRY
First posted: 2013-08-13
Enrollment: 16
Sex: MALE
Minimum age (years): 18
Maximum age (years): 65
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: FACTORIAL
Primary purpose: TREATMENT

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.


Eligibility criteria: 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

Primary outcomes:
measure: Number of participants with adverse events as a measure of safety and tolerability
timeFrame: Up to Day 50
Secondary outcomes:
measure: Pharmacokinetic assessment, based on plasma concentration of BAY86-6150
timeFrame: 9 time points from pre-dosing on Day 1 up to 48 hours post-dosing
measure: Pharmacodynamic assessment, based on plasma hemostasis marker level
timeFrame: 9 time points from pre-dosing on Day 1 up to 48 hours post-dosing
measure: Immunogenicity assessment, based on anti-BAY86-6150 binding antibody levels
timeFrame: 3 time points from pre-dosing on Day 1 up to Day 50
Other outcomes:
Locations:
city: Warszawa
country: Poland
city: Bloemfontein
country: South Africa
state: Freestate
city: Johannesburg
country: South Africa
state: Gauteng
city: London
country: United Kingdom
Study officials:
affiliation: Bayer
name: Bayer Study Director
role: STUDY_DIRECTOR
References:
citation: Mahlangu JN, Coetzee MJ, Laffan M, Windyga J, Yee TT, Schroeder J, Haaning J, Siegel JE, Lemm G. Phase I, randomized, double-blind, placebo-controlled, single-dose escalation study of the recombinant factor VIIa variant BAY 86-6150 in hemophilia. J Thromb Haemost. 2012 May;10(5):773-80. doi: 10.1111/j.1538-7836.2012.04667.x.