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

Bayer

Status and phase: Completed; Phase 1

Conditions: Hemophilia B; Hemophilia A

Treatments: Drug: Placebo; Drug: BAY Factor VII (BAY86-6150)

Study type: Interventional

Funder types: Industry

Identifiers

NCT01921855

13787

2008-000117-29 (EudraCT Number)

Details and patient eligibility

About

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.

Enrollment: 16 patients

Sex: Male

Ages: 18 to 65 years old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Factorial Assignment

Masking: Double Blind

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16 participants in 4 patient groups

BAY Factor VII (6.5 µg/kg) / Placebo

Experimental group

Description:

n = 4, randomized 3:1; 6.5 µg/kg BAY 86-6150 (B0189):Placebo

Treatment:

Drug: BAY Factor VII (BAY86-6150)

Drug: Placebo

Drug: BAY Factor VII (BAY86-6150)

Drug: BAY Factor VII (BAY86-6150)

Drug: BAY Factor VII (BAY86-6150)

BAY Factor VII (20 µg/kg) / Placebo

Experimental group

Description:

n = 4, randomized 3:1; 20 µg/kg BAY 86-6150 (B0189):Placebo

Treatment:

Drug: BAY Factor VII (BAY86-6150)

Drug: Placebo

Drug: BAY Factor VII (BAY86-6150)

Drug: BAY Factor VII (BAY86-6150)

Drug: BAY Factor VII (BAY86-6150)

BAY Factor VII (50 µg/kg) / Placebo

Experimental group

Description:

n = 4, randomized 3:1; 50 µg/kg BAY 86-6150 (B0189):Placebo

Treatment:

Drug: BAY Factor VII (BAY86-6150)

Drug: Placebo

Drug: BAY Factor VII (BAY86-6150)

Drug: BAY Factor VII (BAY86-6150)

Drug: BAY Factor VII (BAY86-6150)

BAY Factor VII (90 µg/kg) / Placebo

Experimental group

Description:

n = 4, randomized 3:1; 90 µg/kg BAY 86-6150 (B0189):Placebo

Treatment:

Drug: BAY Factor VII (BAY86-6150)

Drug: Placebo

Drug: BAY Factor VII (BAY86-6150)

Drug: BAY Factor VII (BAY86-6150)

Drug: BAY Factor VII (BAY86-6150)