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

NCT01921855 | Completed Phase 1 DMC

• 2 other identifiers: 137872008-000117-29
• Study Type: interventional
• Target: 16
• Locations: 3 countries
• Sites: 4

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.

Conditions

Hemophilia A; Hemophilia B

Outcome Measures

Primary Outcomes (1)

• Number of participants with adverse events as a measure of safety and tolerability

  Up to Day 50

Secondary Outcomes (3)

• 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

Study Arms (4)

BAY Factor VII (6.5 µg/kg) / Placebo

EXPERIMENTAL

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

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

BAY Factor VII (20 µg/kg) / Placebo

EXPERIMENTAL

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

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

BAY Factor VII (50 µg/kg) / Placebo

EXPERIMENTAL

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

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

BAY Factor VII (90 µg/kg) / Placebo

EXPERIMENTAL

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

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

Interventions

BAY Factor VII (BAY86-6150) DRUG

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.

BAY Factor VII (6.5 µg/kg) / Placebo

Placebo DRUG

Placebo will be administered as a slow intravenous (i.v.) administration over a period of 2-5 minutes (min) on Study Day 1.

BAY Factor VII (20 µg/kg) / Placebo; BAY Factor VII (50 µg/kg) / Placebo; BAY Factor VII (6.5 µg/kg) / Placebo; BAY Factor VII (90 µg/kg) / Placebo

Eligibility Criteria

• Age: 18 Years - 65 Years
• Sex: male
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Bayer: lead

Study Sites (4)

Unknown Facility

Warsaw, 02-776, Poland

Location

Unknown Facility

Bloemfontein, Freestate, 9300, South Africa

Location

Unknown Facility

Johannesburg, Gauteng, 2193, South Africa

Location

Unknown Facility

London, W12 0HS, United Kingdom

Location

Related Publications (1)

• 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.

  PMID: 22353395 RESULT

MeSH Terms

Conditions

Hemophilia A; Hemophilia B

Condition Hierarchy (Ancestors)

Blood Coagulation Disorders, Inherited; Blood Coagulation Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Coagulation Protein Disorders; Hemorrhagic Disorders; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Genetic Diseases, X-Linked

Study Officials

• Bayer Study Director

  Bayer

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: DOUBLE
• Who Masked: PARTICIPANT, INVESTIGATOR
• Purpose: TREATMENT
• Intervention Model: FACTORIAL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: July 10, 2013
• First Posted: August 13, 2013
• Study Start: January 1, 2009
• Primary Completion: December 1, 2009
• Study Completion: December 1, 2009
• Last Updated: August 21, 2014

Record last verified: 2014-08

Locations

ZA (2); PL (1); GB (1)