A Trial Investigating Safety and Efficacy of Treatment With BAY94-9027 in Severe Hemophilia A



Status:Active, not recruiting
Conditions:Anemia, Hematology
Therapuetic Areas:Hematology
Healthy:No
Age Range:12 - 65
Updated:3/17/2019
Start Date:April 23, 2012
End Date:November 8, 2019

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A Phase II/III, Multicenter, Partially Randomized, Open Label Trial Investigating Safety and Efficacy of On-demand and Prophylactic Treatment With BAY94-9027 in Severe Hemophilia A

Haemophilia A is an inherited disorder in which one of the proteins, Factor VIII, needed to
form blood clots is missing or not present in sufficient levels. In a person with haemophilia
A, the clotting process is slowed and the person experiences bleeds that can result in
serious problems and potential disability.

The current standard treatment for severe haemophilia A is regularly scheduled infusion of
FVIII to keep levels high enough to prevent bleeding. Due to the short half-life of FVIII,
prophylaxis may require treatment as often as every other day.

In this trial safety and efficacy of a long-acting recombinant factor VIII molecule is
evaluated in subjects with severe Hemophilia A.

120-140 patients will receive open label treatment with long-acting rFVIII either on-demand
to treat bleeds or prophylactically for 36 weeks in the main trial plus an optional extension
to continue treatment for at least 100 total exposure days (ED). Patients on prophylactic
treatment will receive study drug at dosing intervals between once and twice a week depending
on their observed bleeding. Patients will attend the treatment centre for routine blood
samples and be required to keep an electronic diary.

Male patients aged 12-65, with severe hemophilia A, previously treated with FVIII for at
least 50 exposure days may be eligible for this study.

Subjects in prophylactic treatment arms will undergo clinical evaluation at 10 weeks. Those
with adequate control of bleeding will undergo randomization to every 5 or 7 day infusion.
Those with continued bleeding will remain in treatment arm and have an increase in dose.

Part B-major surgery - optional sub study included to collect information on efficacy of
BAY94-9027 in major surgical setting. Due to rarity of surgery in this population, enrollment
to this sub-study may be independent of participation in main study.

Inclusion Criteria:

- Male; 12-65 years of age

- Subjects with severe hemophilia A

- Previously treated with factor VIII for a minimum of 150 exposure days

Exclusion Criteria:

- Inhibitors to FVIII (current evidence or history)

- Any other inherited or acquired bleeding disorder in addition to Hemophilia A

- Platelet count < 100,000/mm3

- Creatinine > 2x upper limit of normal or AST/ALT (aspartate aminotransferase/alanine
aminotransferase) > 5x upper limit of normal
We found this trial at
15
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