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Prophylaxis Versus on Demand Treatment for Children With Hemophilia A

This study has been completed.
Information provided by (Responsible Party):
Bayer Identifier:
First received: March 12, 2013
Last updated: April 30, 2015
Last verified: April 2015
Comparison of the effect of three times a week prophylaxis on all bleeds with on-demand treatment for children with severe Hemophilia A.

Condition Intervention Phase
Hemophilia A
Biological: Recombinant Factor VIII (Kogenate FS, BAY14-2222)
Phase 4

Study Type: Interventional
Study Design: Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: Routine Prophylaxis Treatment Versus On-demand Treatment for Children With Severe Hemophilia A: Comparison of All Bleeding Events in Chinese Hemophilia Patients

Resource links provided by NLM:

Further study details as provided by Bayer:

Primary Outcome Measures:
  • Difference of Annualized Number of All Bleeds Between On-demand and Prophylaxis Period [ Time Frame: Week 1-12 (on-demand treatment) and 13-24 (prophylactic treatment) ]
    Annualized bleedings period 1 minus period 2 ITT analysis set.

Secondary Outcome Measures:
  • Difference of Annualized Number of Joint Bleeds Between On-demand and Prophylaxis Period [ Time Frame: 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 [ Time Frame: 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.

Enrollment: 30
Study Start Date: March 2013
Study Completion Date: January 2014
Primary Completion Date: January 2014 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Recombinant Factor VIII Biological: Recombinant Factor VIII (Kogenate FS, BAY14-2222)
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.


Ages Eligible for Study:   2 Years to 16 Years   (Child)
Sexes Eligible for Study:   Male
Accepts 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
  Contacts and Locations
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Please refer to this study by its identifier: NCT01810666

China, Guangdong
Guangzhou, Guangdong, China, 510515
China, Hubei
Wuhan, Hubei, China, 430022
Beijing, China, 100730
Beijing, China
Tianjin, China
Sponsors and Collaborators
Study Director: Bayer Study Director Bayer
  More Information

Additional Information:
Responsible Party: Bayer Identifier: NCT01810666     History of Changes
Other Study ID Numbers: 16287
2014-001362-10 ( EudraCT Number )
Study First Received: March 12, 2013
Results First Received: December 22, 2014
Last Updated: April 30, 2015

Additional relevant MeSH terms:
Hemophilia A
Blood Coagulation Disorders, Inherited
Blood Coagulation Disorders
Hematologic Diseases
Coagulation Protein Disorders
Hemorrhagic Disorders
Genetic Diseases, Inborn
Factor VIII
Coagulants processed this record on May 24, 2017