A phase 3 prospective, uncontrolled, multicenter study evaluating pharmacokinetics, efficacy, safety, and immunogenicity of BAX 855 (PEGylated full-length Recombinant FVIII) in previously treated pediatric patients with severe hemophilia A
- Conditions
- Severe hemophilia A10005330
- Registration Number
- NL-OMON41794
- Lead Sponsor
- Baxter
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Completed
- Sex
- Not specified
- Target Recruitment
- 2
1. The 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/or a FVIII gene mutation consistent with severe hemophila A.
2. Subject is <12 years old at the time of screening.
3. Subjects aged >=6 to <12 years of age have been previously treated with plasma-derived and/or rFVIII concentrate(s) for a minimum of 150 EDs (based on the subjects* medical records).
4. Subjects <6 years of age have been previously treated with plasma-derived and/or rFVIII concentrate(s) for at least 50 EDs (based on the subjects* medical records).
5. Subject is human immunodeficiency virus (HIV) negative; or HIV positive with stable disease and CD4+ count of >=200 cells/mm3, as confirmed by central laboratory.
6. The subject and/or legal representative accepts prophylactic treatment over a period of 6 months.
7. The subject and/or the legal 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 confirmed history of FVIII inhibitory antibodies (>=0.6 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 known hypersensitivity towards mouse or hamster proteins, PEG, or Tween 80.
4. Subject has been diagnosed with an inherited or acquired hemostatic defect other than hemophilia A (eg, qualitative platelet defect or von Willebrand*s disease).
5. Subject*s platelet count is <100,000/µL.
6. Subject has severe chronic hepatic dysfunction (eg, >=5 times upper limit of normal [ULN] alanine aminotransferase as confirmed by central laboratory at screening, or a documented international normalized ratio >1.5).
7. Subject has severe renal impairment (serum creatinine >1.5 times ULN).
8. Subject is scheduled to receive during the course of the study, an immunomodulating drug (eg, corticosteroid agents at a dose equivalent to hydrocortisone >10 mg/day, or a-interferon) other than anti-retroviral chemotherapy.
9. 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.
10. 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.
11. 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.
12. Subject*s legal representative is 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>The incidence of FVIII inhibitory antibodies (>=0.6 BU using the Nijmegen<br /><br>modification of the Bethesda assay).</p><br>
- Secondary Outcome Measures
Name Time Method