Study of FVIIa Variant BAY86-6150 (B0189) in Subjects With Moderate or Severe Hemophilia Types A or B With or Without Inhibitors

Phase 1

Completed

• Conditions: Hemophilia A; Hemophilia B
• Interventions: Drug: BAY Factor VII (BAY86-6150); Drug: Placebo

• Registration Number: NCT01921855
• Lead Sponsor: Bayer

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.

Detailed Description

Not available

Study Timeline

• Study Start: 2009-01
• Primary Completion: 2009-12
• Study Completion: 2009-12
• Study First Submitted: 2013-07-10
• Study First Submitted QC: 2013-08-09
• Study First Posted: 2013-08-13
• Status Verified: 2014-08
• Last Update Submitted: 2014-08-20
• Last Update Posted: 2014-08-21

Recruitment & Eligibility

• Status: COMPLETED
• Sex: Male
• Target Recruitment: 16

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

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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

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Study & Design

• Study Type: INTERVENTIONAL
• Study Design: FACTORIAL

Arm && Interventions

Group	Intervention	Description
BAY Factor VII (50 µg/kg) / Placebo	BAY Factor VII (BAY86-6150)	n = 4, randomized 3:1; 50 µg/kg BAY 86-6150 (B0189):Placebo
BAY Factor VII (50 µg/kg) / Placebo	Placebo	n = 4, randomized 3:1; 50 µg/kg BAY 86-6150 (B0189):Placebo
BAY Factor VII (90 µg/kg) / Placebo	Placebo	n = 4, randomized 3:1; 90 µg/kg BAY 86-6150 (B0189):Placebo
BAY Factor VII (6.5 µg/kg) / Placebo	Placebo	n = 4, randomized 3:1; 6.5 µg/kg BAY 86-6150 (B0189):Placebo
BAY Factor VII (6.5 µg/kg) / Placebo	BAY Factor VII (BAY86-6150)	n = 4, randomized 3:1; 6.5 µg/kg BAY 86-6150 (B0189):Placebo
BAY Factor VII (90 µg/kg) / Placebo	BAY Factor VII (BAY86-6150)	n = 4, randomized 3:1; 90 µg/kg BAY 86-6150 (B0189):Placebo
BAY Factor VII (20 µg/kg) / Placebo	BAY Factor VII (BAY86-6150)	n = 4, randomized 3:1; 20 µg/kg BAY 86-6150 (B0189):Placebo
BAY Factor VII (20 µg/kg) / Placebo	Placebo	n = 4, randomized 3:1; 20 µg/kg BAY 86-6150 (B0189):Placebo

Primary Outcome Measures

Name	Time	Method
Number of participants with adverse events as a measure of safety and tolerability	Up to Day 50

Secondary Outcome Measures

Name	Time	Method
Immunogenicity assessment, based on anti-BAY86-6150 binding antibody levels	3 time points from pre-dosing on Day 1 up to Day 50
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