Prophylaxis Versus on Demand Treatment for Children With Hemophilia A

Phase 4

Completed

• Conditions: Hemophilia A
• Interventions: Biological: Recombinant Factor VIII (Kogenate FS, BAY14-2222)

• Registration Number: NCT01810666
• Lead Sponsor: Bayer

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.

Detailed Description

Not available

Study Timeline

• Study Start: 2013-03
• Study First Submitted: 2013-03-12
• Study First Submitted QC: 2013-03-12
• Study First Posted: 2013-03-13
• Primary Completion: 2014-01
• Study Completion: 2014-01
• Results First Submitted: 2014-12-22
• Results First Submitted QC: 2014-12-22
• Results First Posted: 2015-01-05
• Status Verified: 2015-04
• Last Update Submitted: 2015-04-30
• Last Update Posted: 2015-05-19

Recruitment & Eligibility

• Status: COMPLETED
• Sex: Male
• Target Recruitment: 30

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

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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

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Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Recombinant Factor VIII	Recombinant Factor VIII (Kogenate FS, BAY14-2222)	-

Primary Outcome Measures

Name	Time	Method
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 Outcome Measures

Name	Time	Method
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.
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.