Details

Details

Title A Phase 2/3, Multi-Center, Open Label Study of Efficacy, Safety, and Pharmacokinetics of PEGylated Recombinant Factor VIII (BAX 855) Administered for Prophylaxis and Treatment of Bleeding in Previously Treated Patients with Severe Hemophilia A

IRB BAX 1Z13

CC 13-836

Hospital Main Campus

Phase Phase 2, Phase 3

Disease Hemophilia A

Drug BAX-855, tergenpumapucel-L

Description

Description

Primary
  1. The primary objective is to compare the annualized rates of bleeding episodes (ABR) between subjects receiving a prophylactic regimen of BAX 855 with an on-demand treatment regimen.
Secondary
  1. The key secondary objective is to estimate the rate of success of BAX 855 for treatment of bleeding episodes.
Inclusion Criteria

Inclusion Criteria

  1. Subject and/or legal representative has/have voluntarily provided signed informed consent
  2. Subject is 12 to 65 years old at the time of screening
  3. Subject is male with severe hemophilia A (FVIII clotting activity < 1%) as confirmed by central laboratory at screening after the appropriate washout period or a documented FVIII clotting activity < 1%
  4. Subject has been previously treated with plasma-derived FVIII concentrates or recombinant FVIII for ≥ 150 documented exposure days (EDs)
  5. Subject is currently receiving prophylaxis or on-demand therapy with FVIII
  6. Subject has a Karnofsky performance score of ≥ 60 at screening (Table 7)
  7. 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
  8. 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
  9. Subject is willing and able to comply with the requirements of the protocol
Exclusion Criteria

Exclusion Criteria

  1. Subject has detectable FVIII inhibitory antibodies (≥ 0.4 BU using the Nijmegen modification of the Bethesda assay) as confirmed by central laboratory at screening
  2. Subject has history of FVIII inhibitory antibodies (≥ 0.4 BU using the Nijmegen modification of the Bethesda assay or ≥ 0.6 BU using 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 Willebrands 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 alanine aminotransferase (ALT), as confirmed by central laboratory at screening, or a documented INR > 1.5]
  6. Subject has severe renal impairment (serum creatinine > 2.0 mg/dL), as confirmed by central laboratory at screeningM
  7. Subject 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
  8. Subject has participated in another clinical study involving an 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
  9. Subject has a 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