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

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Bayer
ClinicalTrials.gov Identifier:
NCT01921855
First received: July 10, 2013
Last updated: August 20, 2014
Last verified: August 2014
  Purpose

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.


Condition Intervention Phase
Hemophilia A
Hemophilia B
Drug: BAY Factor VII (BAY86-6150)
Drug: Placebo
Phase 1

Study Type: Interventional
Study Design: Allocation: Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Factorial Assignment
Masking: Double Blind (Subject, Investigator)
Primary Purpose: Treatment
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

Resource links provided by NLM:


Further study details as provided by Bayer:

Primary Outcome Measures:
  • Number of participants with adverse events as a measure of safety and tolerability [ Time Frame: Up to Day 50 ] [ Designated as safety issue: Yes ]

Secondary Outcome Measures:
  • Pharmacokinetic assessment, based on plasma concentration of BAY86-6150 [ Time Frame: 9 time points from pre-dosing on Day 1 up to 48 hours post-dosing ] [ Designated as safety issue: No ]
  • Pharmacodynamic assessment, based on plasma hemostasis marker level [ Time Frame: 9 time points from pre-dosing on Day 1 up to 48 hours post-dosing ] [ Designated as safety issue: No ]
  • Immunogenicity assessment, based on anti-BAY86-6150 binding antibody levels [ Time Frame: 3 time points from pre-dosing on Day 1 up to Day 50 ] [ Designated as safety issue: Yes ]

Enrollment: 16
Study Start Date: January 2009
Study Completion Date: December 2009
Primary Completion Date: December 2009 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: BAY Factor VII (6.5 µg/kg) / Placebo
n = 4, randomized 3:1; 6.5 µg/kg BAY 86-6150 (B0189):Placebo
Drug: BAY 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.
Drug: Placebo
Placebo will be administered as a slow intravenous (i.v.) administration over a period of 2-5 minutes (min) on Study Day 1.
Experimental: BAY Factor VII (20 µg/kg) / Placebo
n = 4, randomized 3:1; 20 µg/kg BAY 86-6150 (B0189):Placebo
Drug: BAY Factor VII (BAY86-6150)
BAY Factor VII (BAY86-6150), 20 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.
Drug: Placebo
Placebo will be administered as a slow intravenous (i.v.) administration over a period of 2-5 minutes (min) on Study Day 1.
Experimental: BAY Factor VII (50 µg/kg) / Placebo
n = 4, randomized 3:1; 50 µg/kg BAY 86-6150 (B0189):Placebo
Drug: BAY Factor VII (BAY86-6150)
BAY Factor VII (BAY86-6150), 50 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.
Drug: Placebo
Placebo will be administered as a slow intravenous (i.v.) administration over a period of 2-5 minutes (min) on Study Day 1.
Experimental: BAY Factor VII (90 µg/kg) / Placebo
n = 4, randomized 3:1; 90 µg/kg BAY 86-6150 (B0189):Placebo
Drug: BAY Factor VII (BAY86-6150)
BAY Factor VII (BAY86-6150), 90 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.
Drug: Placebo
Placebo will be administered as a slow intravenous (i.v.) administration over a period of 2-5 minutes (min) on Study Day 1.

  Eligibility

Ages Eligible for Study:   18 Years to 65 Years
Genders Eligible for Study:   Male
Accepts Healthy Volunteers:   No
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
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01921855

Locations
Poland
Warszawa, Poland, 02-776
South Africa
Bloemfontein, Freestate, South Africa, 9300
Johannesburg, Gauteng, South Africa, 2193
United Kingdom
London, United Kingdom, W12 0HS
Sponsors and Collaborators
Bayer
Investigators
Study Director: Bayer Study Director Bayer
  More Information

Additional Information:
Publications:
Responsible Party: Bayer
ClinicalTrials.gov Identifier: NCT01921855     History of Changes
Other Study ID Numbers: 13787, 2008-000117-29
Study First Received: July 10, 2013
Last Updated: August 20, 2014
Health Authority: South Africa: Medicines Control Council
United Kingdom: Medicines and Healthcare Products Regulatory Agency
Poland: Office for Registration of Medicinal Products, Medical Devices and Biocidal Products

Additional relevant MeSH terms:
Hemophilia A
Hemophilia B
Blood Coagulation Disorders, Inherited
Blood Coagulation Disorders
Hematologic Diseases
Coagulation Protein Disorders
Hemorrhagic Disorders
Genetic Diseases, Inborn
Genetic Diseases, X-Linked

ClinicalTrials.gov processed this record on October 02, 2014