Hemophilia A or B and a high titer inhibitor MedDRA version: 9.1 Level: LLT Classification code 10056492 Term: Haemophilia A with anti factor VIII MedDRA version: 9.1 Level: LLT Classification code 10056494 Term: Haemophilia B with anti factor IX
Conditions
Interventions
Trade Name: FEIBA
Pharmaceutical Form: Powder and solvent for solution for injection
INN or Proposed INN: Factor VIII Inhibitor Bypassing Agent
Concentr
Sponsors
Baxter Innovations GmbH
Eligibility
Sex/Gender
All
Inclusion criteria
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 Karnofsky performance score of = 60 - Hemophilia A and B of any severity, with documented history of high-titer inhibitor (>5BU) 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 Are the trial subjects under 18? yes Number of subjects for this age range: F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range F.1.3 Elderly (>=65 years) no F.1.3.1 Number of subjects for this age range
Exclusion criteria
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, [liver biopsy confirmed], 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 and non-pegylated alpha-interferon with or without ribavarin 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, 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
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Primary end point(s): A reduction in the annualized bleed rate among subjects receiving prophylactic treatment as compared to those treated on-demand.;Main Objective: To demonstrate that the annualized rate of all types of bleeds in subjects on the prophylaxis arm is less than that of the subjects on the on-demand arm.; Secondary Objective: Efficacy: - To characterize the bleeding episodes requiring treatment between the 2 treatment regimens. - To determine the efficacy of FEIBA NF for the control of bleeding episodes. Safety: - To evaluate the safety of prophylactic treatment versus on-demand treatment with FEIBA NF. Pharmacoeconomic and Quality of Life Parameters: - To determine differences in HRQoL between prophylactic and on-demand treatment regimens. - To determine pharmacoeconomic differences between prophylactic and on-demand treatment regimens. Exploratory Objective: - Evaluation of Thrombin Generation Assay (TGA) parameters for hemostatic efficacy correlation. | — |
Countries
Bulgaria, France, Italy, Poland
Outcome results
None listed