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Prophylaxis Versus on Demand Treatment for Children With Hemophilia A

Primary Purpose

Hemophilia A

Status

Completed

Phase

Phase 4

Locations

China

Study Type

Interventional

Intervention

Recombinant Factor VIII (Kogenate FS, BAY14-2222)

About this trial

This is an interventional treatment trial for Hemophilia A

Eligibility Criteria

2 Years - 16 Years (Child)MaleDoes not accept healthy volunteers

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

Sites / Locations

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Recombinant Factor VIII

Arm Description

Outcomes

Primary Outcome Measures

Difference of Annualized Number of All Bleeds Between On-demand and Prophylaxis Period

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

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

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.

Full Information

NCT ID

NCT01810666

First Posted

March 12, 2013

Last Updated

April 30, 2015

Sponsor

Bayer

1. Study Identification

Unique Protocol Identification Number

NCT01810666

Brief Title

Prophylaxis Versus on Demand Treatment for Children With Hemophilia A

Official Title

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

Study Type

Interventional

2. Study Status

Record Verification Date

April 2015

Overall Recruitment Status

Completed

Study Start Date

March 2013 (undefined)

Primary Completion Date

January 2014 (Actual)

Study Completion Date

January 2014 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor

Name of the Sponsor

Bayer

4. Oversight

Data Monitoring Committee

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Hemophilia A

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 4

Interventional Study Model

Single Group Assignment

Masking

None (Open Label)

Allocation

N/A

Enrollment

30 (Actual)

8. Arms, Groups, and Interventions

Arm Title

Recombinant Factor VIII

Arm Type

Experimental

Intervention Type

Biological

Intervention Name(s)

Recombinant Factor VIII (Kogenate FS, BAY14-2222)

Intervention Description

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 Outcome Measure Information:

Title

Difference of Annualized Number of All Bleeds Between On-demand and Prophylaxis Period

Description

Annualized bleedings period 1 minus period 2 ITT analysis set.

Time Frame

Week 1-12 (on-demand treatment) and 13-24 (prophylactic treatment)

Secondary Outcome Measure Information:

Title

Difference of Annualized Number of Joint Bleeds Between On-demand and Prophylaxis Period

Description

Annualized joint bleedings period 1 minus period 2 ITT analysis set.

Time Frame

Week 1-12 (on-demand treatment) and 13-24 (prophylactic treatment)

Title

Difference of Intra-individual Change of Joint Function During Each Period Assessed by the Hemophilia Joint Health Score Between On-demand and Prophylaxis Period

Description

Time Frame

From baseline to Week 12 (on-demand treatment) and Week 24 (prophylactic treatment)

10. Eligibility

Sex

Male

Minimum Age & Unit of Time

2 Years

Maximum Age & Unit of Time

16 Years

Accepts Healthy Volunteers

Eligibility Criteria

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

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

Bayer Study Director

Organizational Affiliation

Bayer

Official's Role

Study Director

Facility Information:

City

Guangzhou

State/Province

Guangdong

ZIP/Postal Code

510515

Country

China

City

Wuhan

State/Province

Hubei

ZIP/Postal Code

430022

Country

China

City

Beijing

ZIP/Postal Code

100730

Country

China

City

Beijing

Country

China

City

Tianjin

Country

China

12. IPD Sharing Statement

Links:

URL

http://www.clinicaltrialsregister.eu/

Description

Click here to find information about studies related to Bayer Healthcare products conducted in Europe

Learn more about this trial

Prophylaxis Versus on Demand Treatment for Children With Hemophilia A

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