Trial Outcomes & Findings for Prophylaxis Versus on Demand Treatment for Children With Hemophilia A (NCT NCT01810666)
NCT ID: NCT01810666
Last Updated: 2015-05-19
Results Overview
Annualized bleedings period 1 minus period 2 ITT analysis set.
Recruitment status
COMPLETED
Study phase
PHASE4
Target enrollment
30 participants
Primary outcome timeframe
Week 1-12 (on-demand treatment) and 13-24 (prophylactic treatment)
Results posted on
2015-05-19
Participant Flow
Subject recruitment period was between 20-Mar-2013 to 28-Jun-2013.
Of 33 participants who were screened for inclusion in the study, 30 were enrolled and received treatment.
Participant milestones
| Measure |
Rec. Factor VIII On-demand Followed by Prophylaxis
Participants received Recombinant Factor VIII (Rec. factor VIII) on-demand treatment for 12 weeks followed by a 12 weeks prophylaxis treatment phase. The dose and mode of prophylaxis treatment was 25 IU/Kg, 3 times/per week. The dose in on-demand treatment was decided by physician according to the package insert or the current standard of care.
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|---|---|
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Overall Study
STARTED
|
30
|
|
Overall Study
COMPLETED
|
30
|
|
Overall Study
NOT COMPLETED
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Prophylaxis Versus on Demand Treatment for Children With Hemophilia A
Baseline characteristics by cohort
| Measure |
Rec. Factor VIII On-demand Followed by Prophylaxis
n=30 Participants
Participants received Recombinant Factor VIII (Rec. factor VIII) on-demand treatment for 12 weeks followed by a 12 weeks prophylaxis treatment phase. The dose and mode of prophylaxis treatment was 25 IU/Kg, 3 times/per week. The dose in on-demand treatment was decided by physician according to the package insert or the current standard of care.
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|---|---|
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Age, Continuous
|
10 Years
STANDARD_DEVIATION 3.9 • n=5 Participants
|
|
Sex: Female, Male
Female
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0 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
30 Participants
n=5 Participants
|
|
Race/Ethnicity, Customized
Asian
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30 Participants
n=5 Participants
|
|
Race/Ethnicity, Customized
Others
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0 Participants
n=5 Participants
|
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Baseline weight
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41.42 Kg
STANDARD_DEVIATION 19.02 • n=5 Participants
|
|
Baseline body mass index
|
18.65 Kg/m2
STANDARD_DEVIATION 4.16 • n=5 Participants
|
|
Target Joint for Bleeds
No
|
8 Participants
n=5 Participants
|
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Target Joint for Bleeds
Yes
|
22 Participants
n=5 Participants
|
|
Number of Target Joint
0
|
8 Participants
n=5 Participants
|
|
Number of Target Joint
1
|
16 Participants
n=5 Participants
|
|
Number of Target Joint
2
|
6 Participants
n=5 Participants
|
|
Prior FVIII Treatment On-demand
|
30 Participants
n=5 Participants
|
|
Number of Bleeds in Last 3 Months
|
12.4 Bleeds
STANDARD_DEVIATION 8.71 • n=5 Participants
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|
Number of Joint Bleeds in Last 3 Months
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8.73 Bleeds
STANDARD_DEVIATION 8.07 • n=5 Participants
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PRIMARY outcome
Timeframe: Week 1-12 (on-demand treatment) and 13-24 (prophylactic treatment)Annualized bleedings period 1 minus period 2 ITT analysis set.
Outcome measures
| Measure |
Rec. Factor VIII On-demand Followed by Prophylaxis
n=30 Participants
Participants received Recombinant Factor VIII (Rec. factor VIII) on-demand treatment for 12 weeks followed by a 12 weeks prophylaxis treatment phase. The dose and mode of prophylaxis treatment was 25 IU/Kg, 3 times/per week. The dose in on-demand treatment was decided by physician according to the package insert or the current standard of care.
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|---|---|
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Difference of Annualized Number of All Bleeds Between On-demand and Prophylaxis Period
|
56.00 Bleeds
Interval 48.8 to 62.54
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SECONDARY outcome
Timeframe: Week 1-12 (on-demand treatment) and 13-24 (prophylactic treatment)Annualized joint bleedings period 1 minus period 2 ITT analysis set.
Outcome measures
| Measure |
Rec. Factor VIII On-demand Followed by Prophylaxis
n=30 Participants
Participants received Recombinant Factor VIII (Rec. factor VIII) on-demand treatment for 12 weeks followed by a 12 weeks prophylaxis treatment phase. The dose and mode of prophylaxis treatment was 25 IU/Kg, 3 times/per week. The dose in on-demand treatment was decided by physician according to the package insert or the current standard of care.
|
|---|---|
|
Difference of Annualized Number of Joint Bleeds Between On-demand and Prophylaxis Period
|
37.71 Bleeds
Interval 30.42 to 46.02
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SECONDARY outcome
Timeframe: From baseline to Week 12 (on-demand treatment) and Week 24 (prophylactic treatment)Hemophilia Joint Health Score(HJHS) ranges from 0 to 124. Higher values in the HJHS represent worse situation for the subject. 2-sided Hodges Lehmann estimates for median 95% CI HJHS values difference of changes ITT analysis set.
Outcome measures
| Measure |
Rec. Factor VIII On-demand Followed by Prophylaxis
n=30 Participants
Participants received Recombinant Factor VIII (Rec. factor VIII) on-demand treatment for 12 weeks followed by a 12 weeks prophylaxis treatment phase. The dose and mode of prophylaxis treatment was 25 IU/Kg, 3 times/per week. The dose in on-demand treatment was decided by physician according to the package insert or the current standard of care.
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|---|---|
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Difference of Intra-individual Change of Joint Function During Each Period Assessed by the Hemophilia Joint Health Score Between On-demand and Prophylaxis Period
|
-3.00 Scores on scale
Interval -5.0 to 0.0
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Adverse Events
Rec. Factor VIII On-demand Followed by Prophylaxis
Serious events: 0 serious events
Other events: 2 other events
Deaths: 0 deaths
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
Rec. Factor VIII On-demand Followed by Prophylaxis
n=30 participants at risk
Participants received Recombinant Factor VIII (Rec. factor VIII) on-demand treatment for 12 weeks followed by a 12 weeks prophylaxis treatment phase. The dose and mode of prophylaxis treatment was 25 IU/Kg, 3 times/per week. The dose in on-demand treatment was decided by physician according to the package insert or the current standard of care.
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|---|---|
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General disorders
Pyrexia
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6.7%
2/30 • Number of events 2 • From first treatment through 3 days after the last dose of study drug, up to 6 months
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Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place
Restriction type: GT60