Trial Outcomes & Findings for Study Evaluating B-Domain Deleted Recombinant Factor VIII (BDDrFVIII, ReFacto AF) in Patients With Hemophilia A Undergoing Elective Major Surgery (NCT NCT00243659)
NCT ID: NCT00243659
Last Updated: 2011-06-03
Results Overview
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.
Recruitment status
COMPLETED
Study phase
PHASE3
Target enrollment
30 participants
Primary outcome timeframe
6 weeks
Results posted on
2011-06-03
Participant Flow
Patients were recruited worldwide from April 2006 to April 2008.
Patients were screened for a maximum of 42 days.
Participant milestones
| Measure |
Bolus Injection
Approximately 50 IU/kg Refacto AF for 6 consecutive days
|
Continuous Infusion
Approximately 50 IU/Kg Refacto AF then continuously
|
|---|---|---|
|
Overall Study
STARTED
|
22
|
8
|
|
Overall Study
COMPLETED
|
20
|
5
|
|
Overall Study
NOT COMPLETED
|
2
|
3
|
Reasons for withdrawal
| Measure |
Bolus Injection
Approximately 50 IU/kg Refacto AF for 6 consecutive days
|
Continuous Infusion
Approximately 50 IU/Kg Refacto AF then continuously
|
|---|---|---|
|
Overall Study
Withdrawal by Subject
|
1
|
1
|
|
Overall Study
Protocol Violation
|
1
|
2
|
Baseline Characteristics
Study Evaluating B-Domain Deleted Recombinant Factor VIII (BDDrFVIII, ReFacto AF) in Patients With Hemophilia A Undergoing Elective Major Surgery
Baseline characteristics by cohort
| Measure |
Bolus Injection
n=22 Participants
Approximately 50 IU/kg Refacto AF for 6 consecutive days
|
Continuous Infusion
n=8 Participants
Approximately 50 IU/Kg Refacto AF then continuously
|
Total
n=30 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age Continuous
|
35.05 years
STANDARD_DEVIATION 9.51 • n=93 Participants
|
38.13 years
STANDARD_DEVIATION 8.54 • n=4 Participants
|
35.87 years
STANDARD_DEVIATION 9.22 • n=27 Participants
|
|
Sex: Female, Male
Female
|
0 Participants
n=93 Participants
|
0 Participants
n=4 Participants
|
0.0 Participants
n=27 Participants
|
|
Sex: Female, Male
Male
|
22 Participants
n=93 Participants
|
8 Participants
n=4 Participants
|
30.0 Participants
n=27 Participants
|
PRIMARY outcome
Timeframe: 6 weeksPopulation: The analysis population is the efficacy evaluable at visit three.
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.
Outcome measures
| Measure |
Bolus Injection
n=20 Participants
Approximately 50 IU/kg Refacto AF for 6 consecutive days
|
Continuous Infusion
n=5 Participants
Approximately 50 IU/Kg Refacto AF then continuously
|
|---|---|---|
|
Number of Patients Who Achieved Hemostatic Efficacy After Surgery
Excellent
|
15 patients
|
3 patients
|
|
Number of Patients Who Achieved Hemostatic Efficacy After Surgery
Good
|
5 patients
|
2 patients
|
|
Number of Patients Who Achieved Hemostatic Efficacy After Surgery
Moderate
|
0 patients
|
0 patients
|
|
Number of Patients Who Achieved Hemostatic Efficacy After Surgery
None
|
0 patients
|
0 patients
|
SECONDARY outcome
Timeframe: 6 weeksPopulation: The analysis population is the efficacy evaluable at visit five.
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.
Outcome measures
| Measure |
Bolus Injection
n=20 Participants
Approximately 50 IU/kg Refacto AF for 6 consecutive days
|
Continuous Infusion
n=5 Participants
Approximately 50 IU/Kg Refacto AF then continuously
|
|---|---|---|
|
Number of Patients Who Achieved Hemostatic Efficacy at Hospital Discharge
Excellent
|
18 patients
|
5 patients
|
|
Number of Patients Who Achieved Hemostatic Efficacy at Hospital Discharge
Good
|
2 patients
|
0 patients
|
|
Number of Patients Who Achieved Hemostatic Efficacy at Hospital Discharge
Moderate
|
0 patients
|
0 patients
|
|
Number of Patients Who Achieved Hemostatic Efficacy at Hospital Discharge
None
|
0 patients
|
0 patients
|
Adverse Events
Bolus Injection
Serious events: 4 serious events
Other events: 19 other events
Deaths: 0 deaths
Continuous Infusion
Serious events: 1 serious events
Other events: 7 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Bolus Injection
Approximately 50 IU/kg Refacto AF for 6 consecutive days
|
Continuous Infusion
Approximately 50 IU/Kg Refacto AF then continuously
|
|---|---|---|
|
General disorders
Fever
|
0.00%
0/22
|
12.5%
1/8
|
|
General disorders
Accidental injury
|
4.5%
1/22
|
0.00%
0/8
|
|
General disorders
Cellulitis
|
4.5%
1/22
|
0.00%
0/8
|
|
Cardiac disorders
Hemorrhage
|
0.00%
0/22
|
12.5%
1/8
|
|
Blood and lymphatic system disorders
Factor VIII inhibition
|
9.1%
2/22
|
0.00%
0/8
|
|
Respiratory, thoracic and mediastinal disorders
Cough increased
|
0.00%
0/22
|
12.5%
1/8
|
Other adverse events
| Measure |
Bolus Injection
Approximately 50 IU/kg Refacto AF for 6 consecutive days
|
Continuous Infusion
Approximately 50 IU/Kg Refacto AF then continuously
|
|---|---|---|
|
General disorders
Accidental injury
|
13.6%
3/22
|
0.00%
0/8
|
|
General disorders
Asthenia
|
0.00%
0/22
|
12.5%
1/8
|
|
General disorders
Back pain
|
4.5%
1/22
|
0.00%
0/8
|
|
General disorders
Cellulitis
|
4.5%
1/22
|
0.00%
0/8
|
|
General disorders
Chills
|
9.1%
2/22
|
0.00%
0/8
|
|
General disorders
Fever
|
31.8%
7/22
|
75.0%
6/8
|
|
General disorders
Flu syndrome
|
13.6%
3/22
|
0.00%
0/8
|
|
General disorders
Headache
|
13.6%
3/22
|
12.5%
1/8
|
|
General disorders
Injection
|
22.7%
5/22
|
0.00%
0/8
|
|
General disorders
Non-specified drug reaction
|
0.00%
0/22
|
12.5%
1/8
|
|
General disorders
Pain
|
9.1%
2/22
|
75.0%
6/8
|
|
Cardiac disorders
Hemorrhage
|
0.00%
0/22
|
12.5%
1/8
|
|
Cardiac disorders
Hypertension
|
9.1%
2/22
|
12.5%
1/8
|
|
Cardiac disorders
Hypotension
|
4.5%
1/22
|
0.00%
0/8
|
|
Cardiac disorders
Hypovolemia
|
0.00%
0/22
|
12.5%
1/8
|
|
Cardiac disorders
Pallor
|
0.00%
0/22
|
12.5%
1/8
|
|
Cardiac disorders
Tachycardia
|
0.00%
0/22
|
12.5%
1/8
|
|
Gastrointestinal disorders
Abdominal distension
|
0.00%
0/22
|
12.5%
1/8
|
|
Gastrointestinal disorders
Constipation
|
4.5%
1/22
|
0.00%
0/8
|
|
Gastrointestinal disorders
Diarrhea
|
4.5%
1/22
|
0.00%
0/8
|
|
Gastrointestinal disorders
Jaundice
|
0.00%
0/22
|
12.5%
1/8
|
|
Gastrointestinal disorders
Mouth ulceration
|
4.5%
1/22
|
0.00%
0/8
|
|
Gastrointestinal disorders
Nausea
|
13.6%
3/22
|
12.5%
1/8
|
|
Gastrointestinal disorders
Vomiting
|
4.5%
1/22
|
12.5%
1/8
|
|
Blood and lymphatic system disorders
Anemia
|
36.4%
8/22
|
12.5%
1/8
|
|
Blood and lymphatic system disorders
Leukocytosis
|
0.00%
0/22
|
12.5%
1/8
|
|
Blood and lymphatic system disorders
Thrombocythemia
|
9.1%
2/22
|
12.5%
1/8
|
|
Metabolism and nutrition disorders
Bilirubinemia
|
0.00%
0/22
|
12.5%
1/8
|
|
Metabolism and nutrition disorders
Peripheral edema
|
4.5%
1/22
|
12.5%
1/8
|
|
Musculoskeletal and connective tissue disorders
Arthralgia
|
4.5%
1/22
|
0.00%
0/8
|
|
Musculoskeletal and connective tissue disorders
Plantar fascitis
|
4.5%
1/22
|
0.00%
0/8
|
|
Nervous system disorders
Hypesthesia
|
4.5%
1/22
|
0.00%
0/8
|
|
Nervous system disorders
Memory impairment
|
0.00%
0/22
|
12.5%
1/8
|
|
Nervous system disorders
Paresthesia
|
4.5%
1/22
|
0.00%
0/8
|
|
Respiratory, thoracic and mediastinal disorders
Cough increased
|
0.00%
0/22
|
12.5%
1/8
|
|
Reproductive system and breast disorders
Rhinitis
|
4.5%
1/22
|
0.00%
0/8
|
|
Respiratory, thoracic and mediastinal disorders
Upper respiratory infection
|
13.6%
3/22
|
0.00%
0/8
|
|
Skin and subcutaneous tissue disorders
Rash
|
4.5%
1/22
|
0.00%
0/8
|
|
Skin and subcutaneous tissue disorders
Vesiculobullous rash
|
0.00%
0/22
|
25.0%
2/8
|
|
Eye disorders
Abnormal vision
|
0.00%
0/22
|
12.5%
1/8
|
|
Ear and labyrinth disorders
Ear disorder
|
4.5%
1/22
|
0.00%
0/8
|
|
Renal and urinary disorders
Hematuria
|
4.5%
1/22
|
0.00%
0/8
|
|
Renal and urinary disorders
Kidney calculus
|
4.5%
1/22
|
0.00%
0/8
|
|
Renal and urinary disorders
Oliguria
|
4.5%
1/22
|
0.00%
0/8
|
|
Renal and urinary disorders
Scrotal edema
|
0.00%
0/22
|
12.5%
1/8
|
|
Injury, poisoning and procedural complications
Location reaction to procedure
|
13.6%
3/22
|
12.5%
1/8
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee The PIs agreed to allow the sponsor 60 days to review and require changes to presentations or publications but only to protect confidential information and intellectual property, and for the sponsor to file a patent application, as applicable. The PIs also agreed for data to be presented first as a joint, multi-center publication.
- Publication restrictions are in place
Restriction type: OTHER