BAY79-4980 Compared to rFVIII-FS in Previously Treated Patients With Severe Hemophilia A

Study Overview
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Brief Summary
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A study to assess treatment with a new formulation of recombinant factor VIII reconstituted with liposomes (BAY79-4980) to evaluate whether a once-a-week treatment is safe and can prevent bleeds in subjects with severe haemophilia A.

Official Title
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Randomized, Active-controlled, Double-blind, Parallel Design Study to Evaluate the Efficacy and Safety of a Once-a-week Prophylaxis Treatment With BAY79-4980 Compared to Three Times-per-week Prophylaxis With rFVIII-FS in Previously Treated Patients With Severe Hemophilia A

Conditions
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Hemophilia A

Intervention / Treatment
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* Biological: rFVIII-FS/pegylated liposomes (BAY79-4980)
* Biological: rFVIII-FS/WFI (BAY14-2222)


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Males aged 12 to 70 years Subjects with severe hemophilia A (< 1% factor VIII [FVIII]:C) Subjects with equal or greater than 150 exposure days (EDs) with any FVIII in total Subjects who have been on-demand treatment with a minimum of 1 relevant bleed per month or have been on secondary prophylaxis treatment with not more than a 3x/week schedule Subjects with bleeding events and/or treatments during the last 6 months prior to study entry which are documented in the subjects medical records Subjects with no measurable inhibitor activity Subjects with no history of FVIII inhibitor antibody formation Written informed consent by subject and parent / legal representative, if < 18 years Exclusion Criteria: Subjects who are receiving primary prophylaxis Subjects on prophylaxis with documented requirements of > 75 IU/kg/week Subjects with any other bleeding disease beside hemophilia A (i.e., von Willebrand disease) Subjects with abnormal renal function Subjects with elevated hepatic transaminases Subjects on treatment with immunomodulatory agents within the last 3 months prior to study entry or during the study Subjects with known hypersensitivity to the active substance, mouse or hamster protein, liposomes or polyethyleneglycol (PEG) Subjects who require any pre-medication for FVIII injections

Ages Eligible for Study
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Minimum Age: 12 Years
Maximum Age: 70 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: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: rFVIII-FS/pegylated liposomes (BAY79-4980)<br>35 IU/kg body weight of BAY79-4980 1x/week plus 2 dummy injections/week (dummy = rFVIII (recombinant factor VIII)-FS (formulated with sucrose) excipient reconstituted in WFI (sterile water for injection)) | Biological: rFVIII-FS/pegylated liposomes (BAY79-4980)<br>* 35 IU/kg body weight intravenous 1x/week for 52 weeks This arm will be stopped by 30.04.10 the subjects will be offered to change to the active comparator arm<br>|
| Active Comparator: rFVIII-FS/WFI (BAY14-2222)<br>25 IU/kg body weight of rFVIII-FS 3x/week (employing 1 percent POPC (1-palmitoyl-2-oleoyl-sn-glycero-3-phosphocholine)-alone liposome (rFVIII-FS-POPC) as blinding agent used for first weekly injection and rFVIII-FS in WFI for 2nd and 3rd injection) | Biological: rFVIII-FS/WFI (BAY14-2222)<br>* 25 IU/kg body weight intravenous 3x/week for 52 weeks<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Percentage of Participants With Less Than 9 Total Bleeds Per Year | Bleeds occurring on the same day were counted as one bleeding event. Bleeds occurring within 72 hours into the same location were also counted as one bleeding event. | up to one year | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Percentage of Participants With Less Than 5 Joint Bleeds Per Year | Bleeds occurring on the same day were counted as one bleeding event. Bleeds occurring within 72 hours into the same location were also counted as one bleeding event. | up to one year | 
| Number of Joint Bleeds Per Participant Per Year in Responders | Bleeds occurring on the same day were counted as one bleeding event. Bleeds occurring within 72 hours into the same location were also counted as one bleeding event. Responders were the subjects with less than 9 total bleeds per year | up to one year | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Hemophilia A, FVIII disease, rFVIII