Phase 3, prospective, multi-center, open label study to investigate safety, immunogenicity, and hemostatic efficacy of PEGylated Factor VIII (BAX 855) in previously untreated patients (PUPs) < 6 years with severe hemophilia A (FVIII < 1%)
- Conditions
- 10035534bleeding disorderhemofilia
- Registration Number
- NL-OMON47694
- Lead Sponsor
- Baxalta Innovations GmbH
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Withdrawn
- Sex
- Not specified
- Target Recruitment
- 5
1. Subject is < 6 years old at the time of screening
2. Subject is previously untreated with <3 EDs to ADVATE, BAX 855 or
plasma transfusion at any time prior to screening
3. Subject has severe hemophilia A (FVIII < 1%) as determined by the
central laboratory, or a historical FVIII level < 1% as determined at any
local laboratory and an optional additional FVIII gene mutation
consistent with severe hemophilia A
4. Subject is immune competent with a CD4+ count > 200 cells/mm3, as
confirmed by central laboratory at screening
5. Parent or legally authorized representative is willing and able to
comply with the requirements of the protocol
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 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. 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 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. Subject receives > 2 EDs of ADVATE in total during the periods prior to
enrollment and during the screening period, up until the baseline visit.
6. The subject's weight is anticipated to be < 5 kg at the baseline visit.
7. Subject's platelet count is < 100,000/mL
8. Subject has known hypersensitivity towards mouse or hamster proteins, PEG or
Tween 80
9. Subject has severe chronic hepatic dysfunction [eg, > 5 times upper limit of
normal alanine aminotransferase (ALT), aspartate aminotransferase (AST), or a
documented INR > 1.5] in his medical history or at the time of screening
10. Subject has severe renal impairment (serum creatinine > 1.5 times the upper
limit of normal)
11. 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
12. Subject 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
antiretroviral chemotherapy
13. 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
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 subject safety or compliance
15. Parent, legally authorized representative or subject 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.
Study & Design
- Study Type
- Interventional
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method <p>Incidence of FVIII inhibitor development.</p><br>
- Secondary Outcome Measures
Name Time Method