BAY14-2222 Prophylaxis and Joint Function Improvement (Adults)

Phase 4

Completed

• Conditions: Hematologic Disease; Hemophilia A
• Interventions: Drug: Kogenate (BAY14-2222)

• Registration Number: NCT00586521
• Lead Sponsor: Bayer

Brief Summary

The purpose of the study is to evaluate the effect of prophylactic treatment on the number of joint bleeds and quality of life in severe hemophilia A subjects compared to on-demand treatment in a one-group two-treatment schedule design.

In addition, the effect of prophylactic treatment on the joint function, the number of all bleeds, and on the quality of life compared to on-demand treatment and health-economic data will be assessed.

Detailed Description

Not available

Study Timeline

• Study Start: 2006-02
• Study First Submitted: 2007-12-21
• Study First Submitted QC: 2007-12-21
• Study First Posted: 2008-01-04
• Primary Completion: 2008-03
• Study Completion: 2008-03
• Results First Submitted: 2009-04-03
• Results First Submitted QC: 2009-11-09
• Results First Posted: 2009-11-19
• Status Verified: 2014-10
• Last Update Submitted: 2014-10-27
• Last Update Posted: 2014-11-02

Recruitment & Eligibility

• Status: COMPLETED
• Sex: Male
• Target Recruitment: 20

Inclusion Criteria

• Severe hemophilia A (< 1% FVIII)
• 30-45 years of age
• Previously treated subject (> 100 Exposure days to any FVIII)
• On-demand therapy with any FVIII

Exclusion Criteria

• No history of inhibitor
• No planned elective orthopedic surgery during the study duration (13 months)
• No severe concomitant disease
• No history of anaphylactic or other severe reaction to previous FVIII treatment

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
rFVIII-FS (octocog-alfa), (Kogenate FS)	Kogenate (BAY14-2222)	On-demand treatment was to follow the same treatment pattern the subject was using before entering the study. While on prophylactic treatment, all subjects were to be treated at a dose of 20-40 IU/kg, 3 times per week at a stable dose.

Primary Outcome Measures

Name	Time	Method
Number of Joint Bleeds	Months 1-6 (on-demand treatment) and 8-13 (prophylactic treatment)	Number of joint bleeds during Months 8-13 compared to number of joint bleeds during Months 1-6

Secondary Outcome Measures

Name	Time	Method
Quality of Life Compared to On-demand Treatment as Measured by the Haemo-QoL A Questionnaire	Month 13 (end of prophylactic treatment) and Month 6 (end of on-demand treatment)	Total transformed score with a range of 0-100, higher values indicate better outcome. 41 items in 6 domains: physical functioning; role functioning; worry; consequences; positive affect; treatment concern.
Number of All Bleeds	Months 1-6 (on-demand treatment) and 8-13 (prophylactic treatment)	Mean number of all bleeds during Months 8-13 (prophylactic) compared to mean number of all bleeds during Months 1-6 (on-demand)
Physical Assessment Compared to On-demand Treatment as Determined by the Gilbert Score	Month 13 (end of prophylactic treatment) and Month 6 (end of on-demand treatment)	Total score with a range of 0-100, evaluating ankle, knee and elbow, 0 indicates normal function, higher values indicate joint damage