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BAX 1Z14   |   14-375
Phase 3b Continuation Study of the Safety and Efficacy of Prophylactic BAX 855 in PTPs with Severe Hemophilia A

Disease(s)
Hemophilia A
Hospital(s)
Main Campus
Phase(s)
Phase 3
Stage(s)
Type(s)
Therapeutic
Drug(s)
PEGylated rFVIII (BAX 855)

Contact Information
Cancer Answer Line

866.223.8100

8:00 am - 4:30 pm, Monday - Friday


Description

Primary Objectives

  • 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 annualized bleed rate (ABR) of spontaneous bleeding episodes

Secondary Objectives

  • Efficacy
    • To determine the total ABR (spontaneous and traumatic bleeding episodes)
    • To determine the overall hemostatic efficacy rating of BAX 855 for treatment of breakthrough bleeding episodes
    • To determine the length of intervals between bleeding episodes
    • To characterize the hemostatic efficacy of BAX 855 for treatment of bleeding episodes by the number of BAX 855 infusions for treatment
    • To determine total weight-adjusted consumption of BAX 855 for prophylaxis and for treatment of bleeding episodes
    • To assess Patient Reported Outcomes (PROs) over time for subjects receiving BAX 855
  • Safety
    • 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

Exploratory Objectives

  • To assess patient satisfaction, patient activity levels, and health resource use over time for subjects receiving BAX 855
  • To determine the potential correlation between TGA parameters, FVIII trough levels and ABR

Inclusion Criteria
Subjects transitioning from other BAX 855 studies who meet ALL of the following criteria are eligible for this study:
  1. Subject has completed a previous BAX 855 study and is willing to immediately transition into this continuation study.
  2. Subject is ≤75 years of age at screening of the previous BAX 855 study.
  3. Subject continues to have a Karnofsky (for subjects aged ≥ 16 years) or Lansky (for subjects aged < 16 years) performance score of ≥ 60 (see Section 20.5).
  4. 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.
  5. 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.
  6. 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.
  7. Subject and/or legally authorized representative is willing and able to comply with the requirements of the protocol.
BAX 855 naive subjects who meet ALL of the following criteria are eligible for this study:
  1. Subject is ≤75 years of age at screening.
  2. Subject is naive to BAX 855.
  3. Subject has severe hemophilia A (FVIII clotting activity < 1%) as confirmed by central laboratory at screening after at least a 72-hour washout period.
  4. Subject aged ≥ 6 years has documented previous treatment with plasma-derived FVIII or rFVIII for ≥ 150 EDs.
  5. Subject aged < 6 years has documented previous treatment with plasma-derived FVIII concentrates or rFVIII for ≥ 50 EDs.
  6. Subject is currently receiving prophylaxis or on-demand therapy with FVIII.
  7. Subject has a Karnofsky (for subjects aged ≥ 16 years) or Lansky (for subjects aged < 16 years) performance score of ≥ 60 (see Section 20.5).
  8. Subject is HIV-; or HIV+ with stable disease and CD4+ count ≥ 200 cells/mm3, as confirmed by central laboratory at screening.
  9. Subject 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, subject presents with a negative urine pregnancy test and agrees to employ adequate birth control measures for the duration of the study.
  11. Subject and/or legally authorized representative is willing and able to comply with the requirements of the protocol.

Exclusion Criteria
Subjects transitioning from other BAX 855 studies who meet ANY of the following criteria are not eligible for this study:
  1. Subject had detectable FVIII inhibitory antibodies (≥ 0.6 BU using the Nijmegen modification of the Bethesda assay) as confirmed by central laboratory at screening.
  2. Subject 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. Subject 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. Subject has severe chronic hepatic dysfunction (eg, ≥ 5 times upper limit of normal alanine aminotransferase [ALT], as confirmed by central laboratory at screening).
  5. Subject has severe renal impairment (serum creatinine > 2.0 mg/dL), as confirmed by central laboratory at screening.
  6. Subject experienced a life-threatening or gastrointestinal bleeding episode within 3 months prior to study entry.
  7. Subject is scheduled to use other PEGylated drugs during study participation.
  8. Subject 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. 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.
  10. Subject is a family member or employee of the investigator.
BAX 855 naive subjects who meet ANY of the following criteria are not eligible for this study:
  1. Subject has detectable FVIII inhibitory antibodies (≥ 0.6 BU using the Nijmegen modification of the Bethesda assay) as confirmed by central laboratory at screening.
  2. Subject 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. Subject has been diagnosed with an inherited or acquired hemostatic defect other than hemophilia A (eg, qualitative platelet defect or von Willebrand's disease).
  4. Subject has known hypersensitivity towards mouse or hamster proteins, PEG, or Tween 80.
  5. Subject has severe chronic hepatic dysfunction eg, ≥ 5 times upper limit of normal ALT, as confirmed by central laboratory at screening).
  6. Subject has severe renal impairment (serum creatinine > 2.0 mg/dL), as confirmed by central laboratory at screening.
  7. Subject experienced a life-threatening or gastrointestinal bleeding episode within 3 months prior to study entry.
  8. 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.
  9. Subject 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 IP or investigational device during the course of this study.
  10. 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.
  11. Subject is a family member or employee of the investigator.

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