Hemophilia A Clinical Trial
Official title:
A Multi-center, Phase III, Non-controlled, Open-label Trial to Evaluate the Pharmacokinetics, Safety, and Efficacy of BAY94-9027 for Prophylaxis and Treatment of Bleeding in Previously Treated Children (Age <12 Years) With Severe Hemophilia A
Hemophilia A is an inherited blood disorder in which one protein, Factor VIII, needed to form
blood clots is missing or not present in sufficient levels. Hemophilia A causes the clotting
process to be slowed and the person experiences bleeds causing serious problems that could
lead to disability. The current standard treatment for severe hemophilia A is infusion of
FVIII to stop bleeding, or regular scheduled treatment to prevent bleeds from occuring. 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 being
evaluated in 50 male subjects, < 12 years of age, with severe Hemophilia A. These subjects
will receive open label treatment with long-acting rFVIII for approximately 6 months (or
longer until 50 exposure days) on a regular schedule at least once every 7-days. Doses and
dose intervals may be adapted to the subject's clinical need. A second group of patients will
receive open label treatment with the same drug for 12 weeks on a regular schedule of
2x/week. Patients will attend the treatment center for routine blood samples and will be
required to keep an electronic diary.
Subjects will be offered participation in an optional extension study to collect observations
for at least an additional 50 exposure days.
n/a
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