NL-OMON47694撤回3 期
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%) - BAX 855 PUP
适应症
试验速览
- 阶段
- 3 期
- 状态
- 撤回
- 入组人数
- 5
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional
入排标准
- 年龄范围
- 2 至 11（—）
入选标准
- •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
排除标准
- •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
- •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
- •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.
研究者
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