Prophylaxis Versus on Demand Treatment for Children With Hemophilia A

Study Overview
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Brief Summary
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Comparison of the effect of three times a week prophylaxis on all bleeds with on-demand treatment for children with severe Hemophilia A.

Official Title
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Routine Prophylaxis Treatment Versus On-demand Treatment for Children With Severe Hemophilia A: Comparison of All Bleeding Events in Chinese Hemophilia Patients

Conditions
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Hemophilia A

Intervention / Treatment
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* Biological: Recombinant Factor VIII (Kogenate FS, BAY14-2222)


Participation Criteria
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Eligibility Criteria
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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

Ages Eligible for Study
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Minimum Age: 2 Years
Maximum Age: 16 Years

Sexes Eligible for Study
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Male

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Recombinant Factor VIII<br> | Biological: Recombinant Factor VIII (Kogenate FS, BAY14-2222)<br>* 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.<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Difference of Annualized Number of All Bleeds Between On-demand and Prophylaxis Period | Annualized bleedings period 1 minus period 2 ITT analysis set. | Week 1-12 (on-demand treatment) and 13-24 (prophylactic treatment) | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Difference of Annualized Number of Joint Bleeds Between On-demand and Prophylaxis Period | Annualized joint bleedings period 1 minus period 2 ITT analysis set. | Week 1-12 (on-demand treatment) and 13-24 (prophylactic treatment) | 
| 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. | From baseline to Week 12 (on-demand treatment) and Week 24 (prophylactic treatment) |