Recombinant Factor VIIa BI (rFVIIa BI) Treatment of Acute Bleeding Episodes Per an On-demand Regimen

This study has been completed.
Information provided by (Responsible Party):
Baxalta US Inc. Identifier:
First received: December 21, 2012
Last updated: November 18, 2015
Last verified: November 2015
The purpose of the study is to determine the efficacy and safety of rFVIIa BI as part of a six-month on-demand treatment regimen in hemophilia A or B subjects with inhibitors.

Condition Intervention Phase
Hemophilia A
Hemophilia B
Biological: Recombinant Factor VIIa BI (rFVIIa BI)
Phase 3

Study Type: Interventional
Study Design: Allocation: Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment

Resource links provided by NLM:

Further study details as provided by Baxalta US Inc.:

Primary Outcome Measures:
  • Bleeding episode treatment success [ Time Frame: within 12 hours of first dose ] [ Designated as safety issue: No ]
    No additional hemostatic product required within 12 hours of first dose other than the prescribed dosing regimen.

Secondary Outcome Measures:
  • Treatment response for each bleeding episode [ Time Frame: within 24 hours ] [ Designated as safety issue: No ]

    Participants shall rate the treatment of each bleeding episode. If treatment occurs under direct supervision of treating physician, then physician shall rate the response.

    Ratings based on a 4 point scale; Excellent, Good, Moderate, None.

  • Percentage of clinical responders (sustained bleeding control) for all acute bleeding episodes [ Time Frame: 24 hours post infusion ] [ Designated as safety issue: No ]
  • Safety and tolerability of treatment regimens by clinical assessment of adverse events (AEs) [ Time Frame: 6 months (throughout study period) ] [ Designated as safety issue: Yes ]
  • Inhibitor development to FVII [ Time Frame: 6 months (throughout study period) ] [ Designated as safety issue: Yes ]

Enrollment: 40
Study Start Date: February 2013
Study Completion Date: November 2014
Primary Completion Date: November 2014 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: ≤ 3 doses of 90 µg/kg rFVIIa BI Biological: Recombinant Factor VIIa BI (rFVIIa BI)
Administered approximately every 3 hours as an intravenous bolus injection on-demand
Experimental: One dose of 270 µg/kg rFVIIa BI Biological: Recombinant Factor VIIa BI (rFVIIa BI)
Administered as a single intravenous bolus injection on-demand


Ages Eligible for Study:   12 Years to 65 Years   (Child, Adult)
Genders Eligible for Study:   Male
Accepts Healthy Volunteers:   No

Main Inclusion Criteria:

  • Participant is male with hemophilia A or B with inhibitors, with a high titer (≥5 Bethesda unit (BU)) or a historical high anamnestic response.
  • Participant is 12 to 65 years old at the time of screening.
  • Participant is currently using or has used bypassing agents for treatment of bleeding episodes.
  • Participant has an annualized bleed rate of 5 or more bleeding episodes per year on average over the 2 years prior to the Screening visit.
  • Participant has a Karnofsky Performance Score ≥60.
  • Participant is hepatitis C virus negative (HCV-) either by antibody testing or polymerase chain reaction (PCR); or hepatitis C virus positive (HCV+) with stable hepatic disease.
  • Participant is human immunodeficiency virus negative (HIV-) or HIV+ with stable disease, CD4+ count ≥200 cells/mm^3 at screening.
  • Participant is willing and able to comply with the requirements of the protocol.

Main Exclusion Criteria:

  • Participant is not willing to go on an on-demand treatment scheme.
  • Participant is positive for a FVII inhibitor at screening.
  • Participant has clinically symptomatic liver disease.
  • Participant has a platelet count <100,000/µL.
  • The use of α-interferon with or without ribavirin is planned for an HCV-infected participant or the use of a protease inhibitor is planned for an HIV-infected participant.

    • Participants currently taking any of these medications for ≥30 days are eligible.
  • Participant has a known hypersensitivity to rFVIIa, hamster or murine proteins, or Tween 80.
  • Participant has a known history of being non-responsive to rFVIIa treatment of bleeding episodes.
  • Participant has a prior history of thromboembolic event or diagnosis of other diseases that may increase the participant's risk of thromboembolic complications.
  • Participant has participated in another clinical study involving an investigational product (IP) or investigational device within 30 days prior to enrollment or is scheduled to participate in another clinical study involving an IP or investigational device during the course of this study.
  • Participant is a family member or employee of the investigator.
  • Participant is scheduled for surgery during the study period.
  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 identifier: NCT01757405

United States, Florida
Health Point Medical Group "St Joseph's Children's Hospital"
Tampa, Florida, United States, 33607
Nara Medical University Hospital
Nara-Ken, Japan, 6348522
Tokyo Medical University Hospital
Tokyo, Japan, 1600023
Kracow Medical Center, LLC
Krakow, Poland, 31-501
Institute of Haematology and Transfusion Medicine, Clinic of Haemostatic Disorders and Internal Diseases
Warszawa, Poland, 02-776
Louis Turcanu Emergency Clinical Children´s Hospital
Timisoara, Romania, 300011
Russian Federation
Kirov Hematology and Blood Transfusion Research Institute under the Federal Medical and Biological Agency of Russia
Kirov, Russian Federation, 610027
Hematology Research Center under RAMS (State Institution), Department of Reconstructive Orthopedic Surgery for Hemophilia Patients
Moscow, Russian Federation, 125167
St. Petersburg City Healthcare Institution Municipal Policlinic # 37
St. Petersburg, Russian Federation, 195213
Clinic for Hematology of the Clinical Center of Serbia
Belgrade, Serbia, 11000
Hospital Teresa Herrera Materno Infantil del C.H.U.Carretera del Pasajes/nlaboratorio de hematología
A Coruña, Spain, 15006
University Hospital Virgen del Rocio
Sevilla, Spain, 41013
Tri-Service General Hospital (TSGH)
Taipei City, Taiwan, 11490
V.K. Gusak Institute of Urgent and Reconstructive Surgery within the Ukrainian National Academy of Medical Sciences, Hematology Department
Donetsk, Ukraine, 83045
Kyiv City Clinical Hospital #9, City Scientific-Practical Center for Diagnostics and Treatment of Patients with Hemostatic Pathlogies
Kiev, Ukraine, 79044
State Institution "Institute of Blood Pathology and Transfusion Medicine within the Ukrainian National Academy of Medical Sciences", Hematology Department
Lviv, Ukraine
Sponsors and Collaborators
Baxalta US Inc.
Study Director: Heinrich Farin, MD Baxter Healthcare Corporation
  More Information

Responsible Party: Baxalta US Inc. Identifier: NCT01757405     History of Changes
Other Study ID Numbers: 021101  2011-006294-26 
Study First Received: December 21, 2012
Last Updated: November 18, 2015
Health Authority: Belgium: Federal Agency for Medicinal Products and Health Products
India: Drugs Controller General of India
Japan: Pharmaceuticals and Medical Devices Agency
Poland: Ministry of Health
Romania: National Medicines Agency
Russia: FSI Scientific Center of Expertise of Medical Application
Serbia: Medicines and Medical Devices Agency
Spain: Spanish Agency of Medicines
Taiwan : Food and Drug Administration
Ukraine: State Pharmacological Center - Ministry of Health
United States: Food and Drug Administration

Keywords provided by Baxalta US Inc.:
with Factor VIII (FVIII) or Factor IX (FIX) inhibitors

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 processed this record on August 28, 2016