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Completed

NCT Number: NCT01810666

Prophylaxis Versus on Demand Treatment for Children With Hemophilia A

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

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Key information

Conditions

Hemophilia A Blood Coagulation Disorders Blood Coagulation Disorders, Inherited Coagulation Protein Disorders Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Hematologic Diseases Hemic and Lymphatic Diseases Hemorrhagic Disorders

Age range

2 year–16 year

Sex eligibility

Male

Study type

Interventional

Phase

Phase 4

Primary location

Guangzhou, Guangdong, China

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Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

Recombinant Factor VIII (Kogenate FS, BAY14-2222)

Biological

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.

Primary outcomes

  1. 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 outcomes

  1. 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.

  2. 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.

Sponsors and collaborators

Lead sponsor

Bayer

Industry

Registry information

Official study title

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

Important dates

Study start
2013
Primary completion
2014
Study completion
2014
First posted
Mar 13, 2013
Registry last updated
May 19, 2015

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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