Hemophilia B
Conditions
Brief summary
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.
Detailed description
The switch to BeneFIX has already been decided by the investigator. Patients will be followed up to 3 months after the switch.
Interventions
BIOLOGICALFactor IX recovery
Sponsors
Wyeth is now a wholly owned subsidiary of Pfizer
Study design
Allocation
NON_RANDOMIZED
Intervention model
SINGLE_GROUP
Masking
NONE
Eligibility
Sex/Gender
MALE
Age
12 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* 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)
Exclusion criteria
* 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
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Number of Participants Reporting Efficacy | 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. |
Countries
France
Participant flow
Recruitment details
Patients were recruited in France from May 2008 to January 2009.
Pre-assignment details
Before enrollment there was up to a 14 day screening period and the investigator reviewed the subject's medical history and medications to ensure that the subject was in good health and met all of the inclusion criteria and none of the exclusion criteria.
Participants by arm
| Arm | Count |
|---|---|
| BeneFIX Plasma-derived FIX recovery with a dose of 50 ± 5 IU/kg before the conversion, BeneFIX recovery with a dose of 50 ± 5 IU/kg after the conversion, treatment with BeneFIX during the next 3 months. | 1 |
| Total | 1 |
Baseline characteristics
| Characteristic | BeneFIX |
|---|---|
| Age Continuous | 27.0 years STANDARD_DEVIATION 0 |
| Sex: Female, Male Female | 0 Participants |
| Sex: Female, Male Male | 1 Participants |
Adverse events
| Event type | EG000 affected / at risk |
|---|---|
| deaths Total, all-cause mortality | — / — |
| other Total, other adverse events | 1 / 1 |
| serious Total, serious adverse events | 1 / 1 |
Outcome results
Primary
Number of Participants Reporting Efficacy
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.
Time frame: 4 months
Population: Intent to Treat (ITT) population. Due to low number of subjects that completed, no descriptive statistics for the efficacy endpoints are provided.