The purpose of the study is to determine the efficacy, safety, and health-related quality of life benefits with FEIBA NF prophylactic treatment as compared with on-demand treatment. The study will be conducted globally and will consist of two arms. Eligible subjects will be randomized to undergo a 12-month period of either prophylactic or on-demand therapy.
(High-Titer) Factor IX Inhibitor, (High-Titer) Factor VIII Inhibitor, Hemophilia A, Hemophilia B
Eligibility:
Genders Eligible for Study: Both
Minimum Age Requirement: 4 Years
Maximum Age Requirement: 65 Years
Inclusion Criteria:
• Signed and dated informed consent form by the subject or the subject's legally authorized representative • The subject is >= 4 to <= 65 years of age • The subject has a Karnofsky performance score of >= 60 • Hemophilia A and B of any severity, with documented history of high-titer inhibitor (> 5 BU) for at least 12 months; or, if inhibitor titer is <= 5 BU, and the subject is refractory with increased dosing of either FVIII or FIX, as demonstrated from the subject'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 (APCCs or rFVIIa) for treatment of bleeding episodes • HCV-, either by antibody testing or PCR; or HCV+ with stable hepatic disease • HIV-, or HIV+ with stable disease and CD4 count > 200 cells/mm3 at screening
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 • Subject 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 subjects or planned use of a protease inhibitor for HIV infected subjects. Subjects currently taking any of these medications for a 30-day course are eligible. • D-dimer levels twice the upper level of normal • Known hypersensitivity to 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 subject'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 subject safety or compliance
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