The purpose of this study is to investigate safety, immunogenicity and hemostatic efficacy of PEGylated recombinant FVIII (BAX 855) in previously untreated patients (PUPs) < 6 years of age with severe hemophilia A (baseline FVIII level < 1%) and < 3 EDs to ADVATE, BAX 855 or plasma transfusion.



Eligible Ages
Under 5 Years
Eligible Genders
Accepts Healthy Volunteers

Inclusion Criteria

  1. Participant is < 6 years old at the time of screening
  2. Participant is previously untreated with < 3 exposure days (EDs) to ADVATE, BAX 855 or plasma transfusion at any time prior to screening
  3. Participant has severe hemophilia A (Factor VIII (FVIII) < 1%) as determined by the central laboratory, or a historical FVIII level < 1% as determined at any local laboratory, optionally supported by an additional FVIII gene mutation consistent with severe hemophilia A
  4. Participant is immune competent with a CD4+ count > 200 cells/mm^3, as confirmed by the central laboratory at screening
  5. Parent or legally authorized representative is willing and able to comply with the requirements of the protocol

Additional inclusion criteria for Part B (immune tolerance induction (ITI))

1. Parent or legal representative has/have voluntarily provided signed informed consent for ITI portion

2. Participant has a confirmed positive high titer inhibitor (> 5.00 Bethesda unit (BU)) or has a positive confirmed low titer inhibitor (≥ 0.6 BU) as determined by the central laboratory based on a second repeat blood sample with

1. poorly controlled bleeding despite increased BAX 855 doses, or

2. requires bypassing agents to treat bleeding

Exclusion Criteria

  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 a 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 been previously treated with any type of FVIII concentrate other than ADVATE or BAX 855, or was administered ADVATE, BAX 855 or plasma transfusion for ≥ 3 EDs at any time prior to screening
  5. Participant receives >2 EDs of ADVATE in total during the periods prior to enrollment and during the screening period, until the baseline infusion.
  6. The participant's weight is anticipated to be < 5 kg at the baseline visit
  7. Participant's platelet count is < 100,000/mL
  8. Participant has known hypersensitivity towards mouse or hamster proteins, polyethylene glycol (PEG) or Tween 80
  9. Participant has severe chronic hepatic dysfunction [eg, > 5 times upper limit of normal alanine aminotransferase (ALT), aspartate aminotransferase (AST), or a documented international normalized ratio (INR) > 1.5] in his medical history or at the time of screening
  10. Participant has severe renal impairment (serum creatinine > 1.5 times the upper limit of normal)
  11. 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
  12. Participant is scheduled to receive during the course of the study a systemic immunomodulating drug (e.g. corticosteroid agents at a dose equivalent to hydrocortisone greater than 10 mg/day or α-interferon) other than anti-retroviral chemotherapy
  13. 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
  14. Parent or legally authorized representative 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
  15. Parent, legally authorized representative or participant are 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.

Additional exclusion criteria for Part B (ITI)

1. Spontaneous disappearance of the inhibitor prior to ITI

2. FVIII inhibitor titer ≥ 0.6 BU is not confirmed by a second new blood sample and determined at the central laboratory

3. Inability or unwillingness to comply with the protocol

Study Design

Phase 3
Study Type
Intervention Model
Single Group Assignment
Primary Purpose
None (Open Label)

Arm Groups

ArmDescriptionAssigned Intervention
Previously untreated patients (PUPs)
<6 years of age with severe hemophilia A (baseline Factor VIII (FVIII) level < 1%)
  • Biological: PEGylated Recombinant Factor VIII
    Polyethylene glycol (PEG)-ylated full-length recombinant FVIII (rFVIII)
    Other names:
    • BAX855
    • BAX 855

Recruiting Locations

More Details

Baxalta now part of Shire

Study Contact

Shire Contact
+1 866 842 5335


Study information shown on this site is derived from ClinicalTrials.gov (a public registry operated by the National Institutes of Health). The listing of studies provided is not certain to be all studies for which you might be eligible. Furthermore, study eligibility requirements can be difficult to understand and may change over time, so it is wise to speak with your medical care provider and individual research study teams when making decisions related to participation.