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

A PHASE 3, PROSPECTIVE, OPEN-LABEL, RANDOMIZED STUDY TO EVALUATE SAFETY AND EFFICACY OF RECOMBINANT ACTIVATED FVII BI (rFVIIa BI) IN THE TREATMENT OF ACUTE BLEEDING EPISODES PER AN ON-DEMAND REGIMEN IN PATIENTS WITH HEMOPHILIA A OR B WITH INHIBITORS

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.

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.