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

Baxalta

Status and phase

Completed

Phase 3

Conditions

Hemophilia B

Hemophilia A or B With Inhibitors

Hemophilia A

Treatments

Biological: Factor VIII Inhibitor Bypassing Activity (nanofiltered, vapor heat-treated)

Study type

Interventional

Funder types

Industry

Identifiers

NCT00851721

2008-003855-65 (EudraCT Number)

090701

Details and patient eligibility

About

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.

Enrollment

52 patients

Sex

All

Ages

4 to 65 years old

Volunteers

No Healthy Volunteers

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