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Phase 3b Continuation Study of the Safety and Efficacy of Prophylactic BAX 855 in PTPs with Severe Hemophilia A
|PEGylated rFVIII (BAX 855)
- To determine the safety of BAX 855 based on the incidence of FVIII inhibitory antibody development
- To determine the efficacy of BAX 855 based on the ABR of spontaneous bleeding episodes (ie, episodes not associated with trauma)
- To determine the total ABR (spontaneous and traumatic bleeding episodes)
- To determine the rate of success of BAX 855 for the treatment of breakthrough bleeding episodes
- To characterize the success of BAX 855 for treatment of bleeding episodes based on the number of BAX 855 infusions for the treatment and the length of intervals between bleeding episodes
- To determine total weight-adjusted consumption for prophylaxis and for the treatment of bleeding episodes
- To assess Health-Related Quality of Life (HRQoL) over time for subjects receiving BAX 855
- To determine the safety of BAX 855, as assessed by the occurrence of AEs and changes in vital signs and clinical laboratory parameters
- To determine the immunogenicity of BAX 855
- To assess patient satisfaction, patient activity levels, and health resource use over time for subjects receiving BAX 855
- Subject is ≤ 75 years of age at screening.
- Subject is naive to BAX 855.
- Subject has severe hemophilia A (FVIII clotting activity < 1%) as confirmed by central laboratory at screening (after at least a 72-hour washout period) or a documented FVIII clotting activity <1% (confirmation is only required for BAX 855 naive subjects).
- Subject aged ≥ 6 years has documented previous treatment with plasma-derived FVIII concentrates or rFVIII for ≥ 150 EDs.
- Subject aged < 6 years has documented previous treatment with plasma-derived FVIII concentrates or rFVIII for ≥ 50 EDs.
- Subject is currently receiving prophylaxis or on-demand therapy with FVIII.
- Subject has a Karnofsky (see Section 20.3) or Lansky performance score of ≥ 60.
- Subject is human immunodeficiency virus negative (HIV-); or HIV+ with stable disease and CD4+ count ≥ 200 cells/mm3, as confirmed by central laboratory at screening.
- Subject is hepatitis C virus negative (HCV-) by antibody or PCR testing (if positive, antibody titer will be confirmed by PCR), as confirmed by central laboratory at screening; or HCV+ with chronic stable hepatitis.
- If female of childbearing potential, subject presents with a negative urine pregnancy test and agrees to employ adequate birth control measures for the duration of the study.
- Subject and/or legally authorized representative is willing and able to comply with the requirements of the protocol.
- Subject has detectable FVIII inhibitory antibodies (≥ 0.4 BU using the Nijmegen modification of the Bethesda assay) as confirmed by central laboratory at screening.
- Subject has history of FVIII inhibitory antibodies (≥ 0.4 BU using the Nijmegen modification of the Bethesda assay or ≥ 0.6 BU using the Bethesda assay) at any time prior to screening.
- 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).
- Subject has known hypersensitivity towards mouse or hamster proteins, PEG, or Tween 80.
- Subject has severe chronic hepatic dysfunction [e.g., ≥ 5 times upper limit of normal alanine aminotransferase (ALT), as confirmed by central laboratory at screening, or documented at a local laboratory within 6 months prior to screening, or a documented INR > 1.5].
- Subject has severe renal impairment (serum creatinine > 2.0 mg/dL), as confirmed by central laboratory at screening, or documented at a local laboratory within 6 months prior to screening.
- Subject experienced a life-threatening or gastrointestinal bleeding episode within 3 months prior to study entry.
- Subject has current or recent (< 30 days) use of other PEGylated drugs prior to study participation or scheduled use of such drugs during study participation.
- Subject has participated in another clinical study involving an investigational product (IP) other than BAX 855 or device within 30 days prior to enrollment or is scheduled to participate in another clinical study involving an IP or investigational device during the course of this study.
- Subject has medical, psychiatric, or cognitive illness or recreational drug/alcohol use that, in the opinion of the investigator, would affect subject safety or compliance.
- Subject is a family member or employee of the investigator.
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