BAY14-2222 Prophylaxis and Joint Function Improvement (Adults)

Study Overview
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Brief Summary
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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.

Official Title
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A Prospective Controlled Study on the Effect on Bleeding Events and Joint Function in Young Adults With Severe Hemophilia A After a 6 Month Prophylaxis Treatment Compared to on Demand Treatment

Conditions
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Hematologic Disease, Hemophilia A

Intervention / Treatment
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* Drug: Kogenate (BAY14-2222)


Participation Criteria
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Eligibility Criteria
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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

Ages Eligible for Study
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Minimum Age: 30 Years
Maximum Age: 45 Years

Sexes Eligible for Study
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Male

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: rFVIII-FS (octocog-alfa), (Kogenate FS)<br>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. | Drug: Kogenate (BAY14-2222)<br>* One group two treatment schedules, first on-demand then switch to prophylaxis<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Number of Joint Bleeds | Number of joint bleeds during Months 8-13 compared to number of joint bleeds during Months 1-6 | Months 1-6 (on-demand treatment) and 8-13 (prophylactic treatment) | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Number of All Bleeds | Mean number of all bleeds during Months 8-13 (prophylactic) compared to mean number of all bleeds during Months 1-6 (on-demand) | Months 1-6 (on-demand treatment) and 8-13 (prophylactic treatment) | 
| Physical Assessment Compared to On-demand Treatment as Determined by the Gilbert Score | Total score with a range of 0-100, evaluating ankle, knee and elbow, 0 indicates normal function, higher values indicate joint damage | Month 13 (end of prophylactic treatment) and Month 6 (end of on-demand treatment) | 
| Quality of Life Compared to On-demand Treatment as Measured by the Haemo-QoL A Questionnaire | 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. | Month 13 (end of prophylactic treatment) and Month 6 (end of on-demand treatment) | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Coagulation Disorders