Hemophilia A
Conditions
Keywords
Hemophilia A, bleeding disorder
Brief summary
This study will examine the efficacy and safety of ReFacto AF in patients with severe and moderately severe hemophilia A undergoing elective major surgery when administered by either bolus injections (BI) or continuous infusion (CI).
Interventions
BIOLOGICALReFacto AF
Sponsors
Wyeth is now a wholly owned subsidiary of Pfizer
Study design
Allocation
NON_RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
MALE
Age
12 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* Male, at least 12 years old, with severe hemophilia A undergoing major elective major surgery requiring FVIII replacement for at least 6 days. Negative FVIII inhibitor test at screening and no past history of inhibitor. * Previously treated with at least 150 exposure days to any Factor VIII product * Normal hepatic and renal function tests and no other bleeding disorder
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Number of Patients Who Achieved Hemostatic Efficacy After Surgery | 6 weeks | Efficacy at the end of surgery as determined by the investigator and/or the surgeon using the 4 point ordinal scale (Excellent, Good, Moderate or None). Excellent: Achieved hemostatic comparable to non-hemophilic patients. Good: Prolonged time to hemostasis (with somewhat increased bleeding compared to non-hemophilic patients. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Number of Patients Who Achieved Hemostatic Efficacy at Hospital Discharge | 6 weeks | Assessment of hemostatic efficacy by the investigator at the day of discharge from the hospital using the 4 point ordinal scale (Excellent, Good, Moderate or None). Excellent: Achieved hemostatic comparable to non-hemophilic patients. Good: Prolonged time to hemostasis (with somewhat increased bleeding compared to non-hemophilic patients. |
Countries
Australia, Austria, Hungary, New Zealand, Poland, Romania, Sweden, United States
Participant flow
Recruitment details
Patients were recruited worldwide from April 2006 to April 2008.
Pre-assignment details
Patients were screened for a maximum of 42 days.
Participants by arm
| Arm | Count |
|---|---|
| Bolus Injection Approximately 50 IU/kg Refacto AF for 6 consecutive days | 22 |
| Continuous Infusion Approximately 50 IU/Kg Refacto AF then continuously | 8 |
| Total | 30 |
Withdrawals & dropouts
| Period | Reason | FG000 | FG001 |
|---|---|---|---|
| Overall Study | Protocol Violation | 1 | 2 |
| Overall Study | Withdrawal by Subject | 1 | 1 |
Baseline characteristics
| Characteristic | Bolus Injection | Continuous Infusion | Total |
|---|---|---|---|
| Age Continuous | 35.05 years STANDARD_DEVIATION 9.51 | 38.13 years STANDARD_DEVIATION 8.54 | 35.87 years STANDARD_DEVIATION 9.22 |
| Sex: Female, Male Female | 0 Participants | 0 Participants | 0.0 Participants |
| Sex: Female, Male Male | 22 Participants | 8 Participants | 30.0 Participants |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk |
|---|---|---|
| deaths Total, all-cause mortality | — / — | — / — |
| other Total, other adverse events | 19 / — | 7 / — |
| serious Total, serious adverse events | 4 / — | 1 / — |
Outcome results
Primary
Number of Patients Who Achieved Hemostatic Efficacy After Surgery
Efficacy at the end of surgery as determined by the investigator and/or the surgeon using the 4 point ordinal scale (Excellent, Good, Moderate or None). Excellent: Achieved hemostatic comparable to non-hemophilic patients. Good: Prolonged time to hemostasis (with somewhat increased bleeding compared to non-hemophilic patients.
Time frame: 6 weeks
Population: The analysis population is the efficacy evaluable at visit three.
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| Bolus Injection | Number of Patients Who Achieved Hemostatic Efficacy After Surgery | Excellent | 15 patients |
| Bolus Injection | Number of Patients Who Achieved Hemostatic Efficacy After Surgery | Good | 5 patients |
| Bolus Injection | Number of Patients Who Achieved Hemostatic Efficacy After Surgery | Moderate | 0 patients |
| Bolus Injection | Number of Patients Who Achieved Hemostatic Efficacy After Surgery | None | 0 patients |
| Continuous Infusion | Number of Patients Who Achieved Hemostatic Efficacy After Surgery | None | 0 patients |
| Continuous Infusion | Number of Patients Who Achieved Hemostatic Efficacy After Surgery | Excellent | 3 patients |
| Continuous Infusion | Number of Patients Who Achieved Hemostatic Efficacy After Surgery | Moderate | 0 patients |
| Continuous Infusion | Number of Patients Who Achieved Hemostatic Efficacy After Surgery | Good | 2 patients |
Secondary
Number of Patients Who Achieved Hemostatic Efficacy at Hospital Discharge
Assessment of hemostatic efficacy by the investigator at the day of discharge from the hospital using the 4 point ordinal scale (Excellent, Good, Moderate or None). Excellent: Achieved hemostatic comparable to non-hemophilic patients. Good: Prolonged time to hemostasis (with somewhat increased bleeding compared to non-hemophilic patients.
Time frame: 6 weeks
Population: The analysis population is the efficacy evaluable at visit five.
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| Bolus Injection | Number of Patients Who Achieved Hemostatic Efficacy at Hospital Discharge | None | 0 patients |
| Bolus Injection | Number of Patients Who Achieved Hemostatic Efficacy at Hospital Discharge | Excellent | 18 patients |
| Bolus Injection | Number of Patients Who Achieved Hemostatic Efficacy at Hospital Discharge | Good | 2 patients |
| Bolus Injection | Number of Patients Who Achieved Hemostatic Efficacy at Hospital Discharge | Moderate | 0 patients |
| Continuous Infusion | Number of Patients Who Achieved Hemostatic Efficacy at Hospital Discharge | Excellent | 5 patients |
| Continuous Infusion | Number of Patients Who Achieved Hemostatic Efficacy at Hospital Discharge | None | 0 patients |
| Continuous Infusion | Number of Patients Who Achieved Hemostatic Efficacy at Hospital Discharge | Moderate | 0 patients |
| Continuous Infusion | Number of Patients Who Achieved Hemostatic Efficacy at Hospital Discharge | Good | 0 patients |