BAX 855 Pediatric Study
Condition: Hemophilia A
MeSH terms: Hemophilia A
Intervention: Antihemophilic Factor (Recombinant) - Plasma/Albumin Free Method; PEGylated Recombinant Factor VIII; PEGylated Recombinant Factor VIII
Intervention description: Pharmacokinetic (PK) analysis of ADVATE; Pharmacokinetic (PK) analysis of BAX 855; Prophylaxis treatment
Intervention MeSH terms: Factor VIII; BAX 855
Phase: Phase 3
Study type: Interventional
Primary purpose: Treatment
Allocation: Non-Randomized
Intervention model: Parallel Assignment
Masking: None (Open Label)
Enrollment: 75.0
Sex: All
Maximum age: 11 Years
Healthy volunteers: No

Summary:

      The study purpose is:

        -  To assess the incidence of FVIII inhibitory antibodies during 6 months of twice weekly
           prophylactic treatment with BAX 855 or 50 exposure days (EDs), whichever occurs last.

        -  To compare pharmacokinetic (PK) parameters to ADVATE.

        -  To assess hemostatic efficacy in prophylaxis and the treatment of bleeding episodes.

        -  To evaluate safety and immunogenicity.
    

Eligibility criteria:

        Inclusion Criteria:

          -  Severe hemophilia A (Factor VIII (FVIII) <1%) determined by central laboratory.

          -  <12 years old at the time of screening.

          -  Participants aged ≥6 to <12 years of age have been previously treated with
             plasma-derived and/or recombinant Factor VIII (rFVIII) concentrate(s) for a minimum of
             150 exposure days (EDs) (based on the participant's medical records).

          -  Participants <6 years of age have been previously treated with plasma-derived and/or
             rFVIII concentrate(s) for at least 50 EDs (based on the participant's medical
             records).

          -  Participant is human immunodeficiency virus (HIV) negative; or HIV positive with
             stable disease and CD4+ count of ≥200 cells/mm^3, as confirmed by central laboratory.

          -  Participant and/or legal representative accepts prophylactic treatment over a period
             of 6 months.

          -  Participant and/or the legal representative is willing and able to comply with the
             requirements of the protocol.

        Exclusion Criteria:

          -  Participant has detectable FVIII inhibitory antibodies (≥0.4 Bethesda Units (BU) using
             the Nijmegen modification of the Bethesda assay) as confirmed by central laboratory at
             screening.

          -  Participant has a 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.

          -  Participant has known hypersensitivity towards mouse or hamster proteins, polyethylene
             glycol (PEG), or Tween 80.

          -  Participant has been diagnosed with an inherited or acquired hemostatic defect other
             than hemophilia A (eg, qualitative platelet defect or von Willebrand's disease).

          -  Participant's platelet count is <100,000/μL.

          -  Participant has severe chronic hepatic dysfunction (eg, ≥5 times upper limit of normal
             (ULN) alanine aminotransferase (ALT), as confirmed by central laboratory at screening,
             or a documented international normalized ratio (INR) >1.5).

          -  Participant has severe renal impairment (serum creatinine >1.5 times ULN).

          -  Participant is scheduled to receive during the course of the study, an
             immunomodulating drug (eg, corticosteroid agents at a dose equivalent to
             hydrocortisone >10 mg/day, or α-interferon) other than anti-retroviral chemotherapy.

          -  Participant has current or recent (<30 days) use of other PEGylated drugs prior to
             study participation or is scheduled to use such drugs during study participation.

          -  Participant has participated in another clinical study involving an investigational
             product (IP) or investigational 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.

          -  Participant has a medical, psychiatric, or cognitive illness or recreational
             drug/alcohol use that, in the opinion of the Investigator, would affect participant
             safety or compliance.

          -  Participant's legal representative is a member of the team conducting this study or is
             in a dependent relationship with one of the study team members. Dependent
             relationships include close relatives (ie, children, partner/spouse, siblings,
             parents) as well as employees of the investigator or site personnel conducting the
             study.