Hemophilia A
Conditions
Brief summary
Comparison of the effect of three times a week prophylaxis on all bleeds with on-demand treatment for children with severe Hemophilia A.
Interventions
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.
Sponsors
Bayer
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
MALE
Age
2 Years to 16 Years
Healthy volunteers
No
Inclusion criteria
* Male, aged 2-16yrs * Severe hemophilia A (\<1% FVIII:C \[Blood Clotting Factor VIII:C\] ) * Minimum of at least 50 documented ED (exposure day) prior to enrolment * No measurable inhibitor activity at baseline and history of FVIII inhibitor antibody formation * Parents or legal guardians document, sign, and date informed consent
Exclusion criteria
* Another bleeding disease that is different from hemophilia A * Known hypersensitivity to the active substance, mouse or hamster protein * Thrombocytopenia (platelet count \<100 000/mm3) based on previous medical records
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Difference of Annualized Number of All Bleeds Between On-demand and Prophylaxis Period | Week 1-12 (on-demand treatment) and 13-24 (prophylactic treatment) | Annualized bleedings period 1 minus period 2 ITT analysis set. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Difference of Annualized Number of Joint Bleeds Between On-demand and Prophylaxis Period | Week 1-12 (on-demand treatment) and 13-24 (prophylactic treatment) | Annualized joint bleedings period 1 minus period 2 ITT analysis set. |
| Difference of Intra-individual Change of Joint Function During Each Period Assessed by the Hemophilia Joint Health Score Between On-demand and Prophylaxis Period | 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. |
Countries
China
Participant flow
Recruitment details
Subject recruitment period was between 20-Mar-2013 to 28-Jun-2013.
Pre-assignment details
Of 33 participants who were screened for inclusion in the study, 30 were enrolled and received treatment.
Participants by arm
| Arm | Count |
|---|---|
| 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. | 30 |
| Total | 30 |
Baseline characteristics
| Characteristic | Rec. Factor VIII On-demand Followed by Prophylaxis |
|---|---|
| Age, Continuous | 10 Years STANDARD_DEVIATION 3.9 |
| Baseline body mass index | 18.65 Kg/m2 STANDARD_DEVIATION 4.16 |
| Baseline weight | 41.42 Kg STANDARD_DEVIATION 19.02 |
| Number of Bleeds in Last 3 Months | 12.4 Bleeds STANDARD_DEVIATION 8.71 |
| Number of Joint Bleeds in Last 3 Months | 8.73 Bleeds STANDARD_DEVIATION 8.07 |
| Number of Target Joint 0 | 8 Participants |
| Number of Target Joint 1 | 16 Participants |
| Number of Target Joint 2 | 6 Participants |
| Prior FVIII Treatment On-demand | 30 Participants |
| Race/Ethnicity, Customized Asian | 30 Participants |
| Race/Ethnicity, Customized Others | 0 Participants |
| Sex: Female, Male Female | 0 Participants |
| Sex: Female, Male Male | 30 Participants |
| Target Joint for Bleeds No | 8 Participants |
| Target Joint for Bleeds Yes | 22 Participants |
Adverse events
| Event type | EG000 affected / at risk |
|---|---|
| deaths Total, all-cause mortality | — / — |
| other Total, other adverse events | 2 / 30 |
| serious Total, serious adverse events | 0 / 30 |
Outcome results
Primary
Difference of Annualized Number of All Bleeds Between On-demand and Prophylaxis Period
Annualized bleedings period 1 minus period 2 ITT analysis set.
Time frame: Week 1-12 (on-demand treatment) and 13-24 (prophylactic treatment)
| Arm | Measure | Value (MEDIAN) |
|---|---|---|
| Rec. Factor VIII On-demand Followed by Prophylaxis | Difference of Annualized Number of All Bleeds Between On-demand and Prophylaxis Period | 56.00 Bleeds |
Secondary
Difference of Annualized Number of Joint Bleeds Between On-demand and Prophylaxis Period
Annualized joint bleedings period 1 minus period 2 ITT analysis set.
Time frame: Week 1-12 (on-demand treatment) and 13-24 (prophylactic treatment)
| Arm | Measure | Value (MEDIAN) |
|---|---|---|
| Rec. Factor VIII On-demand Followed by Prophylaxis | Difference of Annualized Number of Joint Bleeds Between On-demand and Prophylaxis Period | 37.71 Bleeds |
Secondary
Difference of Intra-individual Change of Joint Function During Each Period Assessed by the Hemophilia Joint Health Score Between On-demand and Prophylaxis Period
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.
Time frame: From baseline to Week 12 (on-demand treatment) and Week 24 (prophylactic treatment)
| Arm | Measure | Value (MEDIAN) |
|---|---|---|
| Rec. Factor VIII On-demand Followed by Prophylaxis | 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 |