Safety Immunogenicity and Hemostatic Efficacy of PEGylated Recombinant FVIII (BAX 855) in Previously Untreated Patients (PUPs) < 6 Years of Age With Severe Hemophilia A

  • STATUS
    Recruiting
  • End date
    Jun 29, 2023
  • participants needed
    120
  • sponsor
    Baxalta now part of Shire
Updated on 2 August 2020
Investigator
Takeda Development Center Americas Contact
Primary Contact
Kaiser Permanente Oakland M.C. (9.6 mi away) Contact
+102 other location
hemostatic
immune tolerance
severe haemophilia a
antihemophilic factor
recombinant factor viii
advate
immune tolerance induction
bax 855

Summary

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.

Details
Treatment PEGylated Recombinant Factor VIII
Clinical Study IdentifierNCT02615691
SponsorBaxalta now part of Shire
Last Modified on2 August 2020

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Eligibility

Yes No Not Sure

Inclusion Criteria

Is your age less than or equal to 5 yrs?
Gender: Male or Female
Do you have any of these conditions: Hemophilia or Hemophilia A?
Participant is < 6 years old at the time of screening
Participant is previously untreated with < 3 exposure days (EDs) to ADVATE, BAX 855 or plasma transfusion at any time prior to screening
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
Participant is immune competent with a CD4+ count > 200 cells/mm^3, as confirmed by the central laboratory at screening
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))
Parent or legal representative has/have voluntarily provided signed informed consent for ITI portion
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
poorly controlled bleeding despite increased BAX 855 doses, or
requires bypassing agents to treat bleeding

Exclusion Criteria

Participant has detectable FVIII inhibitory antibodies ( 0.6 BU using the Nijmegen modification of the Bethesda assay) as confirmed by central laboratory at screening
Participant has a history of FVIII inhibitory antibodies ( 0.6 BU using the Nijmegen modification of the Bethesda assay or the Bethesda assay) at any time prior to screening
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 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
Participant receives >2 EDs of ADVATE in total during the periods prior to enrollment and during the screening period, until the baseline infusion
The participant's weight is anticipated to be < 5 kg at the baseline visit
Participant's platelet count is < 100,000/mL
Participant has known hypersensitivity towards mouse or hamster proteins, polyethylene glycol (PEG) or Tween 80
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
Participant has severe renal impairment (serum creatinine > 1.5 times the upper limit of normal)
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 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 -interferon) other than anti-retroviral chemotherapy
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
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
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)
Spontaneous disappearance of the inhibitor prior to ITI
FVIII inhibitor titer 0.6 BU is not confirmed by a second new blood sample and determined at the central laboratory
Inability or unwillingness to comply with the protocol
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