Trial Outcomes & Findings for Study Comparing On-Demand Treatment With Two Prophylaxis Regimens Of BeneFIX In Patients With Severe Hemophilia B (NCT NCT00364182)
NCT ID: NCT00364182
Last Updated: 2011-10-04
Results Overview
Annualized bleed rate (ABR) or number of bleeds per year derived for each participant for each treatment regimen by using the following formula: ABR = number of bleeds / (days on treatment regimen / 365.25)
Recruitment status
COMPLETED
Study phase
PHASE3
Target enrollment
50 participants
Primary outcome timeframe
Baseline up to Week 56
Results posted on
2011-10-04
Participant Flow
3 participants who enrolled in the study were not randomized to either prophylaxis treatment regimen, but participated in the study during the first on demand treatment period
Participant milestones
| Measure |
Pre-Randomization
Participants were enrolled and received BeneFix (recombinant coagulation factor IX) as intravenous (IV) bolus infusion in the first on-demand (OD1) period but were never randomized.
|
BeneFIX OD1, Then 100 IU/kg, Then OD2, Then 50 IU/kg
BeneFIX (recombinant coagulation factor IX) on-demand for 16 weeks (OD1), followed by 100 international units per kilogram (IU/kg) once per week (QW) for 16 weeks prophylactically, followed by 8 weeks BeneFIX on-demand (OD2), followed by 50 IU/kg twice weekly (BW) for 16 weeks prophylactically. Dosage form: IV bolus infusion.
|
BeneFIX OD1 Then 50 IU/kg, Then OD2, Then 100 IU/kg
BeneFIX (recombinant coagulation factor IX) on-demand for 16 weeks (OD1), followed by 50 IU/kg BW for 16 weeks prophylactically, followed by 8 weeks BeneFIX on-demand (OD2), followed by 100 IU/kg QW for 16 weeks prophylactically. Dosage form: IV bolus infusion.
|
|---|---|---|---|
|
First Intervention
STARTED
|
3
|
22
|
25
|
|
First Intervention
COMPLETED
|
0
|
22
|
25
|
|
First Intervention
NOT COMPLETED
|
3
|
0
|
0
|
|
Second Intervention
STARTED
|
0
|
22
|
25
|
|
Second Intervention
Randomized
|
0
|
22
|
25
|
|
Second Intervention
COMPLETED
|
0
|
20
|
23
|
|
Second Intervention
NOT COMPLETED
|
0
|
2
|
2
|
|
Third Intervention
STARTED
|
0
|
20
|
23
|
|
Third Intervention
COMPLETED
|
0
|
19
|
22
|
|
Third Intervention
NOT COMPLETED
|
0
|
1
|
1
|
|
Fourth Intervention
STARTED
|
0
|
19
|
22
|
|
Fourth Intervention
COMPLETED
|
0
|
19
|
22
|
|
Fourth Intervention
NOT COMPLETED
|
0
|
0
|
0
|
Reasons for withdrawal
| Measure |
Pre-Randomization
Participants were enrolled and received BeneFix (recombinant coagulation factor IX) as intravenous (IV) bolus infusion in the first on-demand (OD1) period but were never randomized.
|
BeneFIX OD1, Then 100 IU/kg, Then OD2, Then 50 IU/kg
BeneFIX (recombinant coagulation factor IX) on-demand for 16 weeks (OD1), followed by 100 international units per kilogram (IU/kg) once per week (QW) for 16 weeks prophylactically, followed by 8 weeks BeneFIX on-demand (OD2), followed by 50 IU/kg twice weekly (BW) for 16 weeks prophylactically. Dosage form: IV bolus infusion.
|
BeneFIX OD1 Then 50 IU/kg, Then OD2, Then 100 IU/kg
BeneFIX (recombinant coagulation factor IX) on-demand for 16 weeks (OD1), followed by 50 IU/kg BW for 16 weeks prophylactically, followed by 8 weeks BeneFIX on-demand (OD2), followed by 100 IU/kg QW for 16 weeks prophylactically. Dosage form: IV bolus infusion.
|
|---|---|---|---|
|
First Intervention
Secondary prophylaxis
|
1
|
0
|
0
|
|
First Intervention
Withdrawal by Subject
|
2
|
0
|
0
|
|
Second Intervention
non-compliance
|
0
|
1
|
1
|
|
Second Intervention
Protocol Violation
|
0
|
1
|
0
|
|
Second Intervention
Adverse Event
|
0
|
0
|
1
|
|
Third Intervention
non-compliance
|
0
|
1
|
0
|
|
Third Intervention
Lost to Follow-up
|
0
|
0
|
1
|
Baseline Characteristics
Study Comparing On-Demand Treatment With Two Prophylaxis Regimens Of BeneFIX In Patients With Severe Hemophilia B
Baseline characteristics by cohort
| Measure |
Pre-Randomization
n=3 Participants
Participants were enrolled and received BeneFix (recombinant coagulation factor IX) as intravenous (IV) bolus infusion in the first on-demand (OD1) period but were never randomized.
|
BeneFIX OD1, Then 100 IU/kg, Then OD2, Then 50 IU/kg
n=22 Participants
BeneFIX (recombinant coagulation factor IX) on-demand for 16 weeks (OD1), followed by 100 international units per kilogram (IU/kg) once per week (QW) for 16 weeks prophylactically, followed by 8 weeks BeneFIX on-demand (OD2), followed by 50 IU/kg twice weekly (BW) for 16 weeks prophylactically. Dosage form: IV bolus infusion.
|
BeneFIX OD1 Then 50 IU/kg, Then OD2, Then 100 IU/kg
n=25 Participants
BeneFIX (recombinant coagulation factor IX) on-demand for 16 weeks (OD1), followed by 50 IU/kg BW for 16 weeks prophylactically, followed by 8 weeks BeneFIX on-demand (OD2), followed by 100 IU/kg QW for 16 weeks prophylactically. Dosage form: IV bolus infusion.
|
Total
n=50 Participants
Total of all reporting groups
|
|---|---|---|---|---|
|
Age Continuous
|
19.3 years
STANDARD_DEVIATION 4.5 • n=93 Participants
|
31.7 years
STANDARD_DEVIATION 13.4 • n=4 Participants
|
25.1 years
STANDARD_DEVIATION 14.4 • n=27 Participants
|
27.7 years
STANDARD_DEVIATION 13.9 • n=483 Participants
|
|
Sex: Female, Male
Female
|
0 Participants
n=93 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=27 Participants
|
0 Participants
n=483 Participants
|
|
Sex: Female, Male
Male
|
3 Participants
n=93 Participants
|
22 Participants
n=4 Participants
|
25 Participants
n=27 Participants
|
50 Participants
n=483 Participants
|
PRIMARY outcome
Timeframe: Baseline up to Week 56Population: Intention-to-treat (ITT) population: all enrolled participants
Annualized bleed rate (ABR) or number of bleeds per year derived for each participant for each treatment regimen by using the following formula: ABR = number of bleeds / (days on treatment regimen / 365.25)
Outcome measures
| Measure |
BeneFIX OD1
n=50 Participants
BeneFIX on-demand IV bolus infusion for 16 weeks (first intervention)
|
BeneFIX 100 IU/kg
n=44 Participants
BeneFIX 100 IU/kg IV bolus infusion QW for 16 weeks
|
BeneFIX 50 IU/kg
n=44 Participants
BeneFIX 50 IU/kg IV bolus infusion BW for 16 weeks
|
BeneFIX OD2
BeneFIX on-demand IV bolus infusion for 8 weeks (third intervention)
|
|---|---|---|---|---|
|
Annualized Number of Bleeding Episodes
|
35.1 episodes
Interval 28.8 to 41.4
|
4.6 episodes
Interval 2.1 to 7.2
|
2.6 episodes
Interval -0.1 to 5.3
|
—
|
SECONDARY outcome
Timeframe: 24 and 48 hours post-bleedPopulation: ITT
For each bleeding event, a diary was filled out that night and the subsequent night. Questions included: How long do you think you slept last night? Reported as average duration of sleep during study.
Outcome measures
| Measure |
BeneFIX OD1
n=50 Participants
BeneFIX on-demand IV bolus infusion for 16 weeks (first intervention)
|
BeneFIX 100 IU/kg
n=44 Participants
BeneFIX 100 IU/kg IV bolus infusion QW for 16 weeks
|
BeneFIX 50 IU/kg
n=44 Participants
BeneFIX 50 IU/kg IV bolus infusion BW for 16 weeks
|
BeneFIX OD2
n=43 Participants
BeneFIX on-demand IV bolus infusion for 8 weeks (third intervention)
|
|---|---|---|---|---|
|
Amount of Sleep Measured by Sleep Diary After Hemarthrosis
24 hours post-bleed
|
7.5 hours
Standard Deviation 2.0
|
7.3 hours
Standard Deviation 1.8
|
7.1 hours
Standard Deviation 1.6
|
7.4 hours
Standard Deviation 1.9
|
|
Amount of Sleep Measured by Sleep Diary After Hemarthrosis
48 hours post-bleed
|
7.9 hours
Standard Deviation 1.7
|
7.8 hours
Standard Deviation 1.6
|
7.5 hours
Standard Deviation 1.4
|
8.0 hours
Standard Deviation 1.7
|
SECONDARY outcome
Timeframe: 24 and 48 hours post-bleedPopulation: ITT
For each bleeding event, a diary was filled out that night and the subsequent night. Questions included: How would you describe the quality of your sleep last night? 1=Very Good, 2=Good, 3=Fair, 4=Poor, 5=Very Poor. Reported as quality of sleep during study.
Outcome measures
| Measure |
BeneFIX OD1
n=50 Participants
BeneFIX on-demand IV bolus infusion for 16 weeks (first intervention)
|
BeneFIX 100 IU/kg
n=44 Participants
BeneFIX 100 IU/kg IV bolus infusion QW for 16 weeks
|
BeneFIX 50 IU/kg
n=44 Participants
BeneFIX 50 IU/kg IV bolus infusion BW for 16 weeks
|
BeneFIX OD2
n=43 Participants
BeneFIX on-demand IV bolus infusion for 8 weeks (third intervention)
|
|---|---|---|---|---|
|
Quality of Sleep Measured by Sleep Diary After Hemarthrosis
24 hours post-bleed
|
2.4 Units on a scale
Standard Deviation 0.9
|
2.5 Units on a scale
Standard Deviation 0.9
|
2.3 Units on a scale
Standard Deviation 0.9
|
2.4 Units on a scale
Standard Deviation 0.9
|
|
Quality of Sleep Measured by Sleep Diary After Hemarthrosis
48 hours post-bleed
|
2.1 Units on a scale
Standard Deviation 0.7
|
2.1 Units on a scale
Standard Deviation 0.8
|
2.1 Units on a scale
Standard Deviation 0.7
|
2.1 Units on a scale
Standard Deviation 0.9
|
SECONDARY outcome
Timeframe: 24 and 48 hours post-bleedPopulation: ITT
For each bleeding event, a diary was filled out that night and the subsequent night and included a Brief Pain Inventory (BPI): self-reported scale that measured severity of pain experienced over the past 24 hours. Questions included How much pain right now? 0 (no pain) to 10 (pain as severe as you can imagine).
Outcome measures
| Measure |
BeneFIX OD1
n=50 Participants
BeneFIX on-demand IV bolus infusion for 16 weeks (first intervention)
|
BeneFIX 100 IU/kg
n=44 Participants
BeneFIX 100 IU/kg IV bolus infusion QW for 16 weeks
|
BeneFIX 50 IU/kg
n=44 Participants
BeneFIX 50 IU/kg IV bolus infusion BW for 16 weeks
|
BeneFIX OD2
n=43 Participants
BeneFIX on-demand IV bolus infusion for 8 weeks (third intervention)
|
|---|---|---|---|---|
|
Acute Pain After Hemarthrosis
24 hours post-bleed
|
2.8 units on a scale
Standard Deviation 2.1
|
2.8 units on a scale
Standard Deviation 2.0
|
3.1 units on a scale
Standard Deviation 2.2
|
3.0 units on a scale
Standard Deviation 2.1
|
|
Acute Pain After Hemarthrosis
48 hours post-bleed
|
2.3 units on a scale
Standard Deviation 1.6
|
1.8 units on a scale
Standard Deviation 1.2
|
2.7 units on a scale
Standard Deviation 1.5
|
4.1 units on a scale
Standard Deviation 2.4
|
SECONDARY outcome
Timeframe: 24 hours post-bleedPopulation: ITT
HRPQ: self-reported scale that measured for each bleed event, hours lost from work, school and housework because of hemophilia and its treatments. Mean and standard deviations calculated from measured values.
Outcome measures
| Measure |
BeneFIX OD1
n=50 Participants
BeneFIX on-demand IV bolus infusion for 16 weeks (first intervention)
|
BeneFIX 100 IU/kg
n=44 Participants
BeneFIX 100 IU/kg IV bolus infusion QW for 16 weeks
|
BeneFIX 50 IU/kg
n=44 Participants
BeneFIX 50 IU/kg IV bolus infusion BW for 16 weeks
|
BeneFIX OD2
n=43 Participants
BeneFIX on-demand IV bolus infusion for 8 weeks (third intervention)
|
|---|---|---|---|---|
|
Health-Related Productivity Questionnaire (HRPQ) Score: Hours Lost From Work or School at 24 Hours Post-bleed
Work missed
|
0.4 hours
Standard Deviation 1.6
|
1.2 hours
Standard Deviation 2.6
|
0.6 hours
Standard Deviation 1.9
|
0.6 hours
Standard Deviation 1.3
|
|
Health-Related Productivity Questionnaire (HRPQ) Score: Hours Lost From Work or School at 24 Hours Post-bleed
Housework couldn't do
|
0.7 hours
Standard Deviation 2.4
|
0.3 hours
Standard Deviation 0.8
|
0.4 hours
Standard Deviation 0.7
|
0.2 hours
Standard Deviation 0.5
|
|
Health-Related Productivity Questionnaire (HRPQ) Score: Hours Lost From Work or School at 24 Hours Post-bleed
Classwork couldn't do
|
0.8 hours
Standard Deviation 1.7
|
0.7 hours
Standard Deviation 2.2
|
0.4 hours
Standard Deviation 1.1
|
0.0 hours
Standard Deviation 0.0
|
SECONDARY outcome
Timeframe: 48 hours post-bleedPopulation: ITT
HRPQ: self-reported scale that measured for each bleed event, hours lost from work, school and housework because of hemophilia and its treatments. Mean and standard deviations calculated from measured values.
Outcome measures
| Measure |
BeneFIX OD1
n=50 Participants
BeneFIX on-demand IV bolus infusion for 16 weeks (first intervention)
|
BeneFIX 100 IU/kg
n=44 Participants
BeneFIX 100 IU/kg IV bolus infusion QW for 16 weeks
|
BeneFIX 50 IU/kg
n=44 Participants
BeneFIX 50 IU/kg IV bolus infusion BW for 16 weeks
|
BeneFIX OD2
n=43 Participants
BeneFIX on-demand IV bolus infusion for 8 weeks (third intervention)
|
|---|---|---|---|---|
|
HRPQ Score: Hours Lost From Work or School at 48 Hours Post-bleed
Work missed
|
0.2 hours
Standard Deviation 1.0
|
0.7 hours
Standard Deviation 2.1
|
0.1 hours
Standard Deviation 0.4
|
0.2 hours
Standard Deviation 0.5
|
|
HRPQ Score: Hours Lost From Work or School at 48 Hours Post-bleed
Housework couldn't do
|
0.5 hours
Standard Deviation 1.4
|
0.3 hours
Standard Deviation 0.7
|
0.0 hours
Standard Deviation 0.2
|
0.0 hours
Standard Deviation 0.0
|
|
HRPQ Score: Hours Lost From Work or School at 48 Hours Post-bleed
Classwork couldn't do
|
0.4 hours
Standard Deviation 1.2
|
0.7 hours
Standard Deviation 2.4
|
0.0 hours
Standard Deviation 0.0
|
1.7 hours
Standard Deviation 2.6
|
SECONDARY outcome
Timeframe: Weeks 16, 32, and 56Population: ITT; N=number of participants with evaluable data.
SF-36: standardized survey evaluating 8 aspects of functional health and well being: physical and social functioning, physical and emotional role limitations, bodily pain, general health, vitality, and mental health. The physical functioning domain score was an average of the individual physical functioning question scores across all time points, which was scaled 0-100 (100=highest level of functioning).
Outcome measures
| Measure |
BeneFIX OD1
n=40 Participants
BeneFIX on-demand IV bolus infusion for 16 weeks (first intervention)
|
BeneFIX 100 IU/kg
n=35 Participants
BeneFIX 100 IU/kg IV bolus infusion QW for 16 weeks
|
BeneFIX 50 IU/kg
n=34 Participants
BeneFIX 50 IU/kg IV bolus infusion BW for 16 weeks
|
BeneFIX OD2
BeneFIX on-demand IV bolus infusion for 8 weeks (third intervention)
|
|---|---|---|---|---|
|
36-Item Short-Form Health Survey (SF-36): Physical Functioning Domain
|
64 units on a scale
Standard Deviation 26
|
66 units on a scale
Standard Deviation 25
|
63 units on a scale
Standard Deviation 28
|
—
|
Adverse Events
BeneFIX OD1
Serious events: 1 serious events
Other events: 8 other events
Deaths: 0 deaths
BeneFIX 100 IU/kg
Serious events: 2 serious events
Other events: 8 other events
Deaths: 0 deaths
BeneFIX 50 IU/kg
Serious events: 2 serious events
Other events: 4 other events
Deaths: 0 deaths
BeneFIX OD2
Serious events: 0 serious events
Other events: 4 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
BeneFIX OD1
n=50 participants at risk
BeneFIX in an on-demand IV bolus infusion for 16 weeks (first intervention)
|
BeneFIX 100 IU/kg
n=44 participants at risk
BeneFIX 100 IU/kg IV bolus infusion QW for 16 weeks
|
BeneFIX 50 IU/kg
n=44 participants at risk
BeneFIX 50 IU/kg IV bolus infusion BW for 16 weeks
|
BeneFIX OD2
n=43 participants at risk
BeneFIX on-demand IV bolus infusion for 8 weeks (third intervention)
|
|---|---|---|---|---|
|
Musculoskeletal and connective tissue disorders
Arthropathy
|
0.00%
0/50
The same event may appear as both an adverse event (AE) and a serious AE (SAE). However, what is presented are distinct events. An event may be categorized as serious in 1 participant and as nonserious in another participant, or 1 participant may have experienced both a serious and nonserious event during the study.
|
0.00%
0/44
The same event may appear as both an adverse event (AE) and a serious AE (SAE). However, what is presented are distinct events. An event may be categorized as serious in 1 participant and as nonserious in another participant, or 1 participant may have experienced both a serious and nonserious event during the study.
|
2.3%
1/44
The same event may appear as both an adverse event (AE) and a serious AE (SAE). However, what is presented are distinct events. An event may be categorized as serious in 1 participant and as nonserious in another participant, or 1 participant may have experienced both a serious and nonserious event during the study.
|
0.00%
0/43
The same event may appear as both an adverse event (AE) and a serious AE (SAE). However, what is presented are distinct events. An event may be categorized as serious in 1 participant and as nonserious in another participant, or 1 participant may have experienced both a serious and nonserious event during the study.
|
|
Musculoskeletal and connective tissue disorders
Back pain
|
0.00%
0/50
The same event may appear as both an adverse event (AE) and a serious AE (SAE). However, what is presented are distinct events. An event may be categorized as serious in 1 participant and as nonserious in another participant, or 1 participant may have experienced both a serious and nonserious event during the study.
|
0.00%
0/44
The same event may appear as both an adverse event (AE) and a serious AE (SAE). However, what is presented are distinct events. An event may be categorized as serious in 1 participant and as nonserious in another participant, or 1 participant may have experienced both a serious and nonserious event during the study.
|
2.3%
1/44
The same event may appear as both an adverse event (AE) and a serious AE (SAE). However, what is presented are distinct events. An event may be categorized as serious in 1 participant and as nonserious in another participant, or 1 participant may have experienced both a serious and nonserious event during the study.
|
0.00%
0/43
The same event may appear as both an adverse event (AE) and a serious AE (SAE). However, what is presented are distinct events. An event may be categorized as serious in 1 participant and as nonserious in another participant, or 1 participant may have experienced both a serious and nonserious event during the study.
|
|
Injury, poisoning and procedural complications
Ulna fracture
|
2.0%
1/50
The same event may appear as both an adverse event (AE) and a serious AE (SAE). However, what is presented are distinct events. An event may be categorized as serious in 1 participant and as nonserious in another participant, or 1 participant may have experienced both a serious and nonserious event during the study.
|
0.00%
0/44
The same event may appear as both an adverse event (AE) and a serious AE (SAE). However, what is presented are distinct events. An event may be categorized as serious in 1 participant and as nonserious in another participant, or 1 participant may have experienced both a serious and nonserious event during the study.
|
0.00%
0/44
The same event may appear as both an adverse event (AE) and a serious AE (SAE). However, what is presented are distinct events. An event may be categorized as serious in 1 participant and as nonserious in another participant, or 1 participant may have experienced both a serious and nonserious event during the study.
|
0.00%
0/43
The same event may appear as both an adverse event (AE) and a serious AE (SAE). However, what is presented are distinct events. An event may be categorized as serious in 1 participant and as nonserious in another participant, or 1 participant may have experienced both a serious and nonserious event during the study.
|
|
Renal and urinary disorders
Calculus urinary
|
0.00%
0/50
The same event may appear as both an adverse event (AE) and a serious AE (SAE). However, what is presented are distinct events. An event may be categorized as serious in 1 participant and as nonserious in another participant, or 1 participant may have experienced both a serious and nonserious event during the study.
|
2.3%
1/44
The same event may appear as both an adverse event (AE) and a serious AE (SAE). However, what is presented are distinct events. An event may be categorized as serious in 1 participant and as nonserious in another participant, or 1 participant may have experienced both a serious and nonserious event during the study.
|
0.00%
0/44
The same event may appear as both an adverse event (AE) and a serious AE (SAE). However, what is presented are distinct events. An event may be categorized as serious in 1 participant and as nonserious in another participant, or 1 participant may have experienced both a serious and nonserious event during the study.
|
0.00%
0/43
The same event may appear as both an adverse event (AE) and a serious AE (SAE). However, what is presented are distinct events. An event may be categorized as serious in 1 participant and as nonserious in another participant, or 1 participant may have experienced both a serious and nonserious event during the study.
|
|
Renal and urinary disorders
Renal colic
|
0.00%
0/50
The same event may appear as both an adverse event (AE) and a serious AE (SAE). However, what is presented are distinct events. An event may be categorized as serious in 1 participant and as nonserious in another participant, or 1 participant may have experienced both a serious and nonserious event during the study.
|
2.3%
1/44
The same event may appear as both an adverse event (AE) and a serious AE (SAE). However, what is presented are distinct events. An event may be categorized as serious in 1 participant and as nonserious in another participant, or 1 participant may have experienced both a serious and nonserious event during the study.
|
0.00%
0/44
The same event may appear as both an adverse event (AE) and a serious AE (SAE). However, what is presented are distinct events. An event may be categorized as serious in 1 participant and as nonserious in another participant, or 1 participant may have experienced both a serious and nonserious event during the study.
|
0.00%
0/43
The same event may appear as both an adverse event (AE) and a serious AE (SAE). However, what is presented are distinct events. An event may be categorized as serious in 1 participant and as nonserious in another participant, or 1 participant may have experienced both a serious and nonserious event during the study.
|
|
Renal and urinary disorders
Testicular pain
|
0.00%
0/50
The same event may appear as both an adverse event (AE) and a serious AE (SAE). However, what is presented are distinct events. An event may be categorized as serious in 1 participant and as nonserious in another participant, or 1 participant may have experienced both a serious and nonserious event during the study.
|
0.00%
0/44
The same event may appear as both an adverse event (AE) and a serious AE (SAE). However, what is presented are distinct events. An event may be categorized as serious in 1 participant and as nonserious in another participant, or 1 participant may have experienced both a serious and nonserious event during the study.
|
2.3%
1/44
The same event may appear as both an adverse event (AE) and a serious AE (SAE). However, what is presented are distinct events. An event may be categorized as serious in 1 participant and as nonserious in another participant, or 1 participant may have experienced both a serious and nonserious event during the study.
|
0.00%
0/43
The same event may appear as both an adverse event (AE) and a serious AE (SAE). However, what is presented are distinct events. An event may be categorized as serious in 1 participant and as nonserious in another participant, or 1 participant may have experienced both a serious and nonserious event during the study.
|
|
Respiratory, thoracic and mediastinal disorders
Pneumothorax
|
0.00%
0/50
The same event may appear as both an adverse event (AE) and a serious AE (SAE). However, what is presented are distinct events. An event may be categorized as serious in 1 participant and as nonserious in another participant, or 1 participant may have experienced both a serious and nonserious event during the study.
|
2.3%
1/44
The same event may appear as both an adverse event (AE) and a serious AE (SAE). However, what is presented are distinct events. An event may be categorized as serious in 1 participant and as nonserious in another participant, or 1 participant may have experienced both a serious and nonserious event during the study.
|
0.00%
0/44
The same event may appear as both an adverse event (AE) and a serious AE (SAE). However, what is presented are distinct events. An event may be categorized as serious in 1 participant and as nonserious in another participant, or 1 participant may have experienced both a serious and nonserious event during the study.
|
0.00%
0/43
The same event may appear as both an adverse event (AE) and a serious AE (SAE). However, what is presented are distinct events. An event may be categorized as serious in 1 participant and as nonserious in another participant, or 1 participant may have experienced both a serious and nonserious event during the study.
|
Other adverse events
| Measure |
BeneFIX OD1
n=50 participants at risk
BeneFIX in an on-demand IV bolus infusion for 16 weeks (first intervention)
|
BeneFIX 100 IU/kg
n=44 participants at risk
BeneFIX 100 IU/kg IV bolus infusion QW for 16 weeks
|
BeneFIX 50 IU/kg
n=44 participants at risk
BeneFIX 50 IU/kg IV bolus infusion BW for 16 weeks
|
BeneFIX OD2
n=43 participants at risk
BeneFIX on-demand IV bolus infusion for 8 weeks (third intervention)
|
|---|---|---|---|---|
|
Nervous system disorders
Headache
|
6.0%
3/50
The same event may appear as both an adverse event (AE) and a serious AE (SAE). However, what is presented are distinct events. An event may be categorized as serious in 1 participant and as nonserious in another participant, or 1 participant may have experienced both a serious and nonserious event during the study.
|
13.6%
6/44
The same event may appear as both an adverse event (AE) and a serious AE (SAE). However, what is presented are distinct events. An event may be categorized as serious in 1 participant and as nonserious in another participant, or 1 participant may have experienced both a serious and nonserious event during the study.
|
4.5%
2/44
The same event may appear as both an adverse event (AE) and a serious AE (SAE). However, what is presented are distinct events. An event may be categorized as serious in 1 participant and as nonserious in another participant, or 1 participant may have experienced both a serious and nonserious event during the study.
|
4.7%
2/43
The same event may appear as both an adverse event (AE) and a serious AE (SAE). However, what is presented are distinct events. An event may be categorized as serious in 1 participant and as nonserious in another participant, or 1 participant may have experienced both a serious and nonserious event during the study.
|
|
Musculoskeletal and connective tissue disorders
Arthralgia
|
10.0%
5/50
The same event may appear as both an adverse event (AE) and a serious AE (SAE). However, what is presented are distinct events. An event may be categorized as serious in 1 participant and as nonserious in another participant, or 1 participant may have experienced both a serious and nonserious event during the study.
|
9.1%
4/44
The same event may appear as both an adverse event (AE) and a serious AE (SAE). However, what is presented are distinct events. An event may be categorized as serious in 1 participant and as nonserious in another participant, or 1 participant may have experienced both a serious and nonserious event during the study.
|
4.5%
2/44
The same event may appear as both an adverse event (AE) and a serious AE (SAE). However, what is presented are distinct events. An event may be categorized as serious in 1 participant and as nonserious in another participant, or 1 participant may have experienced both a serious and nonserious event during the study.
|
4.7%
2/43
The same event may appear as both an adverse event (AE) and a serious AE (SAE). However, what is presented are distinct events. An event may be categorized as serious in 1 participant and as nonserious in another participant, or 1 participant may have experienced both a serious and nonserious event during the study.
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee Pfizer has the right to review disclosures, requesting a delay of less than 60 days. Investigator will postpone single center publications until after disclosure of pooled data (all sites), less than 12 months from study completion/termination at all participating sites. Investigator may not disclose previously undisclosed confidential information other than study results.
- Publication restrictions are in place
Restriction type: OTHER