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

Conditions: Hemophilia A or B With Inhibitors; Hemophilia A; Hemophilia B

Summary: 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.


Eligibility criteria:
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

Keywords: hemophilia a factor viii inhibitor factor ix inhibitor hemophilia b