Prophylaxis Versus on Demand Treatment for Children With Hemophilia A

April 30, 2015 updated by: Bayer

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

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

Study Overview

Status

Completed

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Actual)

30

Phase

  • Phase 4

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • China
      • Beijing, China
      • Beijing, China, 100730
      • Tianjin, China
    • Guangdong
      • Guangzhou, Guangdong, China, 510515
    • Hubei
      • Wuhan, Hubei, China, 430022

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

2 years to 16 years (Child)

Accepts Healthy Volunteers

No

Genders Eligible for Study

Male

Description

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
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.

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
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.
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
Time Frame: Week 1-12 (on-demand treatment) and 13-24 (prophylactic treatment)
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
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.
From baseline to Week 12 (on-demand treatment) and Week 24 (prophylactic treatment)

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Bayer

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

Helpful Links

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

March 1, 2013

Primary Completion (Actual)

January 1, 2014

Study Completion (Actual)

January 1, 2014

Study Registration Dates

First Submitted

March 12, 2013

First Submitted That Met QC Criteria

March 12, 2013

First Posted (Estimate)

March 13, 2013

Study Record Updates

Last Update Posted (Estimate)

May 19, 2015

Last Update Submitted That Met QC Criteria

April 30, 2015

Last Verified

April 1, 2015

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • 16287
  • 2014-001362-10 (EudraCT Number)

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

Clinical Trials on Hemophilia A

Clinical Trials on Recombinant Factor VIII (Kogenate FS, BAY14-2222)

Search Similar Trials

Sponsors and Collaborators

Medical Conditions

Drug Interventions

CROs by country

CROs in Peru

Conditions

Rare Diseases

Drug Interventions

Dietary Supplements

Sponsor/Collaborators

Locations

3
Subscribe