Hemophilia B MedDRA version: 9.1 Level: LLT Classification code 10016077 Term: Factor IX deficiency
Conditions
Interventions
Trade Name: BeneFIX
Product Name: BeneFIX
Pharmaceutical Form: Powder and solvent for solution for injection
INN or Proposed INN: nonacog alfa
Current Sponsor code: rFIX
Concentration unit: IU interna
Sponsors
Wyeth Pharmaceuticals France
Eligibility
Sex/Gender
Male
Inclusion criteria
Inclusion criteria: · Moderately to severe hemophilia B patient (FIX activity == 150 ED to any FIX product · Male patients, aged >= 12 years · Absolute CD4 count >= 300/ml · Normal platelet count (>= 100 000/ml) · 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 =65 years) yes F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: · Any other known bleeding disorder in addition to hemophilia B · History of, or current detectable factor IX inhibitor (>= 0.6 BU by Bethesda inhibitor assay) · History of anaphylaxis to any coagulation factor IX · Patient with a known hypersensitivity to hamster protein · 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 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 judgement, makes participation in the study not advisable.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To collect data around the period of the conversion from pdFIX to BeneFIX in patients for whom a conversion to BeneFIX has already been decided by the investigator. The main following parameters will be collected : - a retrospective history of the bleedings in the 3-month period before the conversion - the recovery with the pdFIX (just before the conversion) - the recovery with BeneFIX (just after the conversion) - a prospective history of the bleedings in the 3-month period following the conversion - BeneFIX safety data after the conversion - convenience evaluation with pdFIX (retrospective) and BeneFIX (prospective);Secondary Objective: ;Primary end point(s): · Number / location of bleeding episode · Number of injections per bleeding episode · Factor IX consumption · Subjective evaluation of efficacy by the investigator and the patient | — |
Countries
France
Outcome results
None listed