Efficacy and Safety Study of Prophylactic Versus On-Demand Treatment With Feiba NF in Subjects With Hemophilia A or B and a High Titer Inhibitor

FEIBA NF: A Prospective, Open-label, Randomized, Parallel Study to Evaluate the Efficacy and Safety of Prophylactic Versus On-Demand Treatment in Subjects With Hemophilia A or B and a High Titer Inhibitor

The purpose of the study was to determine the efficacy, safety, and health-related quality of life benefits with FEIBA NF prophylactic treatment as compared with on-demand treatment.

Inclusion Criteria:

• Signed and dated informed consent form by the participant or the participant's legally authorized representative
• The participant is ≥ 4 to ≤ 65 years of age
• The participant has a Karnofsky performance score of ≥ 60
• Hemophilia A and B of any severity, with documented history of high-titer inhibitor (> 5 Bethesda unit (BU)) for at least 12 months; or, if inhibitor titer is ≤ 5 BU, and the participant is refractory with increased dosing of either factor VIII (FVIII) or factor IX (FIX), as demonstrated from the participant's medical history
• Currently being treated on an on-demand basis for treatment of bleeding episodes
• Adequate venous access, with or without central venous device
• ≥ 12 bleeding episodes requiring treatment with by-passing agents in the past 12 months, based on medical history
• Competent in-home treatment and infusion therapy
• Currently using bypassing agents (activated prothrombin complex concentrate (APCC) or recombinant activated factor VII (rFVIIa)) for treatment of bleeding episodes
• HCV-, either by antibody testing or polymerase chain reaction (PCR); or HCV+ with stable hepatic disease
• HIV-, or HIV+ with stable disease and CD4 count > 200 cells/mm3 at screening
• Female participant of childbearing potential, presents with a negative serum pregnancy test, and agrees to employ adequate birth control measures for the duration of the study

Exclusion Criteria:

• Currently receiving immune tolerance induction (ITI)
• Currently on regular prophylactic therapy to prevent bleeding episodes
• Clinically symptomatic liver disease (e.g. diagnosis of cirrhosis [confirmed by liver biopsy], portal vein hypertension, ascites, prothrombin time (PT) 5 seconds above upper limit of normal)
• Platelet count < 100,000/ml
• Planned elective surgery during participation in this study
• Participant is currently participating in another clinical study and has received an investigational product or device within 30 days prior to study entry
• Planned use of pegylated or non-pegylated alpha-interferon with or without ribavirin for HCV infected participants or planned use of a protease inhibitor for HIV infected participants. Participants currently taking any of these medications for a 30-day course are eligible.
• Clinically significant increase in D-dimer levels from historical baseline and/or associated with chronic liver disease or clinically evident thromboembolic event
• Known hypersensitivity to anti-inhibitor coagulant complexes (AICCs)
• Currently treated with a systemic immunomodulating drug
• Prior history of thromboembolic event: acute myocardial infarction, deep vein thrombosis, or pulmonary embolism
• Diagnosis of advanced atherosclerosis, malignancy and/or other diseases that may increase the participant's risk of thromboembolic complications
• Clinically significant medical, psychiatric, or cognitive illness, or recreational drug/alcohol use that, in the opinion of the investigator, would affect participant safety or compliance