prophylaxis study of recombinant factor viii manufactured protein-free (rahf-pfm) in patients with hemophilia a

Conditions: Hemophilia A

Summary: the primary purpose of this randomized, two-arm parallel clinical study in 66 previously treated patients with severe or moderately severe hemophilia a is to compare the rate of bleeding episodes for standard prophylaxis (20-40 iu/kg every 48 ± 6 hours; actual dose determined by the investigator) with that of alternate prophylaxis (20-80 iu/kg every 72 + 6 hours; actual dose determined by baxter utilizing an algorithm and the patient's pharmacokinetic data). the rates of bleeding episodes for the on-demand regimen and the prophylaxis regimens will also be compared for the cross-over portion of the study. enrolled patients will be treated originally on demand for a period of 6 months and then they will be randomized into one of the prophylaxis arms. prophylactic treatment will last for a period of 12 months +/- 2 weeks.


Eligibility criteria:
inclusion criteria: * the subject has severe or moderately severe hemophilia a as defined by a baseline factor viii level \<= 2% of normal, as tested at screening * the subject has a documented history of at least 150 exposure days to factor viii concentrates (either plasma-derived or recombinant) * the subject is within 7 to 65 years of age * the subject has a karnofsky performance score \> (greater than) 60 * the subject is human immunodeficiency virus negative (hiv-) or is hiv+ with a cd4 count \>= 400 cells/mm³ (cd4 count determined at screening, if necessary) * the subject has been on a documented on-demand treatment regimen for at least 12 months immediately prior to enrollment * the subject has a documented history (e.g. in medical charts or dispensing information, or signed investigator statement) of at least 8 joint hemorrhages in the 12 months immediately prior to enrollment * the subject resides within the coverage area of the mobile compliance device; coverage area will be determined at screening * the subject or the subject's legally authorized representative has provided written informed consent exclusion criteria: * the subject has a known hypersensitivity to factor viii concentrates or mouse or hamster proteins * the subject has a history of factor viii inhibitors with a titer \>= 0.6 bu (by bethesda or nijmegen assay) at any time prior to screening * the subject has a detectable factor viii inhibitor at screening, with a titer \>= 0.4 bu (by nijmegen assay) in the central laboratory * the subject has severe chronic liver disease as evidenced by, but not limited to, any of the following: international normalized ratio (inr) \> 1.4, hypoalbuminemia, portal vein hypertension including presence of otherwise unexplained splenomegaly and history of esophageal varices. * the subject has been diagnosed with an inherited or acquired hemostatic defect other than hemophilia a (e.g., qualitative platelet defect or von willebrand's disease) * the subject has been treated during the last sixty (60) days prior to or is being treated at screening/enrollment with an immunomodulating drug. * the subject has participated in another investigational study within thirty (30) days of enrollment * the subject has previously participated in a clinical study with rahf-pfm * the subject's clinical condition may require a major surgery (defined as moderate to critical risk and perioperative blood loss ≥ 500 ml) during the period of the subject's participation in the study * the subject is female of childbearing potential with a positive pregnancy test