Status:
COMPLETED
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
Lead Sponsor:
Baxalta now part of Shire
Conditions:
Hemophilia A or B With Inhibitors
Hemophilia A
Eligibility:
All Genders
4-65 years
Phase:
PHASE3
Brief 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
- 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
- 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
Key Trial Info
Start Date :
March 31 2009
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
October 17 2012
Estimated Enrollment :
52 Patients enrolled
Trial Details
Trial ID
NCT00851721
Start Date
March 31 2009
End Date
October 17 2012
Last Update
May 19 2021
Active Locations (17)
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1
Chicago, Illinois, United States
2
Cleveland, Ohio, United States
3
Rio de Janeiro, Rio de Janeiro, Brazil
4
São Paulo, São Paulo, Brazil