Study Evaluating BeneFIX in Patients With Haemophilia B, Previously Treated With Plasma Derived Factor IX
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government.
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The purpose of this study is to collect data around the period of the conversion from plasma-derived Factor IX (pdFIX) to BeneFIX. The main information collected will be: a retrospective history of the bleedings in the 3-month period before the conversion, the recovery with pdFIX just before the conversion and with BeneFIX just after the conversion, and a prospective history of the bleedings in the 3 month period following the conversion.
Condition or disease
Biological: Factor IX recovery
The switch to BeneFIX has already been decided by the investigator. Patients will be followed up to 3 months after the switch.
Number of Participants Reporting Efficacy [ Time Frame: 4 months ]
Clinical efficacy was measured by number/location of bleeding episodes, number of injections per bleeding, factor IX consumption, global assessment of efficacy by investigator and patient; biological efficacy (recovery) with BeneFIX was measured just after conversion.
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Ages Eligible for Study:
12 Years and older (Child, Adult, Older Adult)
Sexes Eligible for Study:
Accepts Healthy Volunteers:
Moderately to severe haemophilia B patient (FIX activity < or equal to 2%) for whom the switch from pdFIX to BeneFIX has already been decided by the investigator
Previously treated patients (PTP) with > or equal to 150 ED to any FIX product
Male patients, aged > or equal to 12 years
Absolute CD4 count > or equal to 300/microL
Normal platelet count (> or equal to 100 000/microL)
Patient is in a non-bleeding state and has not received any coagulation FIX within five (5) days of recovery
Written informed consent obtained prior to study entry (for patients aged < 18 years, parents' signature or subject legally acceptable representative obtained prior to study entry)
Any other known bleeding disorder in addition to haemophilia B
History of, or current detectable factor IX inhibitor (> or equal to 0.6 BU by Bethesda inhibitor assay)
History of anaphylaxis to any coagulation factor IX
Patient with a known hypersensitivity to hamster protein
Patient with a hypersensitivity to the active substance or to any of the excipients
Patient unable to be off FIX replacement therapy for at least 5 days without bleeding Patient with hepatic or renal impairment (ALT [SGPT] and AST [SGOT] > 5 x Upper Limit Normal (ULN), total bilirubin > 20mg/l, albumin < 25 g/l, prothrombin time > 1.25 x ULN, serum creatinine > 1.25 x ULN)
Treatment with any investigational drug or device within the past 30 days
Any condition that, in the Investigator's judgment, makes participation in the study not advisable