China ADVATE PTP Study

Study to Evaluate Efficacy and Safety of ADVATE in the Treatment of Previously Treated Patients With Hemophilia A

The purpose of this study is to assess efficacy, safety and pharmacokinetics of ADVATE in the treatment and prevention of bleeding episodes (BEs)

Main Inclusion Criteria:

• Ethnic Chinese
• is of any age
• has a documented diagnosis of severe or moderately severe hemophilia A (congenital FVIII deficiency: baseline Factor VIII (FVIII) ≤ 2%)
• has documented and verified >50 exposure days (EDs) to FVIII (recombinant or plasma derived)
• is receiving on-demand treatment with FVIII at the time of enrolment in this study
• has negative history of inhibitor development
• is HIV negative or HIV positive with stable disease and CD4+ count ≥ 200 cells per mm^3
• is negative for Hepatitis C virus (HCV); Or participant is HCV positive with chronic stable hepatitis as assessed by investigator

Main Exclusion Criteria:

• has prior history of hypersensitivity or anaphylaxis associated with receipt of FVIII
• is diagnosed with other bleeding disorder(s) other than hemophilia A, including but not limited to thrombocytopenia (platelet count < 100000 /mL)
• has been exposed to an investigational product (IP) within 30 days prior to the screening visit or is scheduled to participate in another clinical study involving an IP or investigational device during participation in the study
• is planned, or likely to have surgery during the study period
• has end-stage renal failure or evidence of a severe or uncontrolled systemic disease as judged by the investigator
• has active hepatic disease (alanine aminotransferase (ALT) and aspartate aminotransferase (AST) levels > 5 times the upper limit of normal)
• has clinical or laboratory evidence of severe liver impairment including (but not limited to) a recent & persistent international normalized ratio (INR) >1.4, and/or the presence of splenomegaly and/or significant spider angioma on physical exam, and/or a history of esophageal hemorrhage or documented esophageal varices
• is a family member of the investigator or site staff