BAX 855 Continuation
Condition: Hemophilia A
MeSH terms: Hemophilia A
Intervention: BAX855
Intervention description: Antihemophilic Factor (Recombinant), PEGylated
Intervention MeSH terms: Factor VIII; BAX 855
Phase: Phase 3
Study type: Interventional
Primary purpose: Prevention
Allocation: Non-Randomized
Intervention model: Parallel Assignment
Masking: None (Open Label)
Enrollment: 218.0
Sex: All
Maximum age: 75 Years
Healthy volunteers: No

Summary:

      To continue the evaluation of the safety and efficacy of BAX 855 for prophylaxis and
      treatment of bleeding episodes in adult and pediatric previously treated patients (PTPs) aged
      ≤ 75 years of age with severe hemophilia A.
    

Eligibility criteria:

        INCLUSION CRITERIA

        Participants Transitioning from Other BAX 855 Studies:

        Participants transitioning from other BAX 855 studies can be provided with the continuation
        study informed consent form (ICF) prior to the end of study visit to review and consider
        participation in this continuation study. These participants will complete any additional
        screening assessments within 2 weeks of the previous study's end of study visit and will
        return to the study site within 6 (± 1) weeks of the previous study end of study visit to
        confirm eligibility for this continuation study.

          -  Participants transitioning from other BAX 855 studies who meet ALL of the following
             criteria are eligible for this study:

               1. Participant has completed a previous BAX 855 study and is willing to immediately
                  transition into this continuation study.

               2. Participant is ≤75 years of age at screening of the previous BAX 855 study.

               3. Participant continues to have a Karnofsky (for participants aged ≥ 16 years) or
                  Lansky (for participants aged < 16 years) performance score of ≥ 60.

               4. Participant is human immunodeficiency virus negative (HIV-); or HIV+ with stable
                  disease and CD4+ count ≥ 200 cells/mm^3, as confirmed by central laboratory at
                  screening.

               5. Participant is hepatitis C virus negative (HCV-) by antibody or polymerase chain
                  reaction (PCR) testing (if positive, antibody titer will be confirmed by PCR), as
                  confirmed by central laboratory at screening; or HCV+ with chronic stable
                  hepatitis.

               6. If female of childbearing potential, participant presents with a negative urine
                  pregnancy test and agrees to employ adequate birth control measures for the
                  duration of the study.

               7. Participant and/or legally authorized representative is willing and able to
                  comply with the requirements of the protocol.

          -  BAX 855 Naïve Participants:

        BAX 855 naïve participants who are ≥ 12 years of age can only be enrolled in this
        continuation study after enrollment in the phase 2/3 pivotal study is closed. BAX 855 naïve
        participants who are < 12 years of age can only be enrolled in this continuation study
        after enrollment in the pediatric previously treated patient (PTP) study is closed.

        - Enrolment of BAX 855 naïve participants will only start once the sponsor has notified the
        study sites accordingly.

        BAX 855 naïve participants who meet ALL of the following criteria are eligible for this
        study:

          1. Participant is ≤75 years of age at screening.

          2. Participant is naïve to BAX 855.

          3. Participant has severe hemophilia A (FVIII clotting activity < 1%) as confirmed by
             central laboratory at screening after at least a 72-hour washout period.

          4. Participant aged ≥ 6 years has documented previous treatment with plasma-derived FVIII
             or rFVIII for ≥ 150 exposure days (EDs).

          5. Participant aged < 6 years has documented previous treatment with plasma-derived FVIII
             concentrates or rFVIII for ≥ 50 EDs.

          6. Participant is currently receiving prophylaxis or on-demand therapy with FVIII.

          7. Participant has a Karnofsky (for participants aged ≥ 16 years) or Lansky (for
             participants aged < 16 years) performance score of ≥ 60.

          8. Participant is HIV-; or HIV+ with stable disease and CD4+ count ≥ 200 cells/mm^3, as
             confirmed by central laboratory at screening.

          9. Participant is 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.

         10. If female of childbearing potential, participant presents with a negative urine
             pregnancy test and agrees to employ adequate birth control measures for the duration
             of the study.

         11. Participant and/or legally authorized representative is willing and able to comply
             with the requirements of the protocol.

        EXCLUSION CRITERA

        - Participants Transitioning from Other BAX 855 Studies:

        Participants transitioning from other BAX 855 studies who meet ANY of the following
        criteria are not eligible for this study:

          1. Participant had detectable factor VIII (FVIII) inhibitory antibodies (≥ 0.6 Bethesda
             unit (BU) using the Nijmegen modification of the Bethesda assay) as confirmed by
             central laboratory at screening.

          2. Participant has developed FVIII inhibitory antibodies (≥ 0.6 BU using the Nijmegen
             modification of the Bethesda assay as determined at central laboratory in a previous
             BAX 855 study).

          3. Participant has acquired a hemostatic defect other than hemophilia A (eg, qualitative
             platelet defect or von Willebrand's disease) in a previous BAX 855 study.

          4. Participant has severe chronic hepatic dysfunction (eg, ≥ 5 times upper limit of
             normal alanine aminotransferase [ALT], as confirmed by central laboratory at
             screening).

          5. Participant has severe renal impairment (serum creatinine > 2.0 mg/dL), as confirmed
             by central laboratory at screening.

          6. Participant experienced a life-threatening or gastrointestinal bleeding episode within
             3 months prior to study entry.

          7. Participant is scheduled to use other PEGylated drugs during study participation.

          8. Participant is planning to take part in any other clinical study during the course of
             the continuation study, with the exception of any other parallel BAX 855 study.

          9. Participant has medical, psychiatric, or cognitive illness or recreational
             drug/alcohol use that, in the opinion of the investigator, would affect participant
             safety or compliance.

         10. Participant is a family member or employee of the investigator.

               -  BAX 855 Naïve Participants:

        BAX 855 naïve participants who meet ANY of the following criteria are not eligible for this
        study:

          1. Participant has detectable FVIII inhibitory antibodies (≥ 0.6 BU using the Nijmegen
             modification of the Bethesda assay) as confirmed by central laboratory at screening.

          2. Participant has history of FVIII inhibitory antibodies (≥ 0.6 BU using the Nijmegen
             modification of the Bethesda assay or the Bethesda assay) at any time prior to
             screening.

          3. Participant has been diagnosed with an inherited or acquired hemostatic defect other
             than hemophilia A (eg, qualitative platelet defect or von Willebrand's disease).

          4. Participant has known hypersensitivity towards mouse or hamster proteins, polyethylene
             glycol (PEG), or Tween 80.

          5. Participant has severe chronic hepatic dysfunction eg, ≥ 5 times upper limit of normal
             ALT, as confirmed by central laboratory at screening).

          6. Participant has severe renal impairment (serum creatinine > 2.0 mg/dL), as confirmed
             by central laboratory at screening.

          7. Participant experienced a life-threatening or gastrointestinal bleeding episode within
             3 months prior to study entry.

          8. Participant has current or recent (< 30 days) use of other PEGylated drugs prior to
             study participation or scheduled use of such drugs during study participation.

          9. Participant has participated in another clinical study involving an IP other than BAX
             855 or device within 30 days prior to enrollment or is scheduled to participate in
             another clinical study involving an investigational product (IP) or investigational
             device during the course of this study.

         10. Participant has medical, psychiatric, or cognitive illness or recreational
             drug/alcohol use that, in the opinion of the investigator, would affect participant
             safety or compliance.

         11. Participant is a family member or employee of the investigator.