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

Completed · Phase 3

Conditions studied: Hemophilia A or B With Inhibitors, Hemophilia A, Hemophilia B

In brief

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.

Key facts

Study ID

NCT00851721

Run by

Baxalta now part of Shire

People needed

52

Starts

2009-03-31

Expected to finish

2012-10-17

Last updated by the study team

2021-05-19

Who can join

Age: 4 and older, up to 65. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 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

You may not qualify if…

• 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

Where it is running

• Study site — Chicago, Illinois, United States
• Study site — Cleveland, Ohio, United States
• Study site — Rio de Janeiro, Rio de Janeiro, Brazil
• Study site — São Paulo, São Paulo, Brazil
• Study site — Sofia, Bulgaria
• Study site — Zagreb, Croatia
• Study site — Kanagawa, Japan
• Study site — Nara, Japan
• Study site — Wellington, New Zealand
• Study site — Krakow, Poland
• Study site — Warsaw, Poland
• Study site — Bucharest, Romania
• Study site — Timișoara, Romania
• Study site — Kirov, Russia
• Study site — Moscow, Russia
• Study site — Yekaterinburg, Russia
• Study site — Lviv, Ukraine

Full record on ClinicalTrials.gov

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