An Open-Label, Single-Arm, Safety and Efficacy Study of Recombinant Human Factor IX (rFIX; BeneFIX ) in Children Less Than 6 Years of Age With Severe Hemophilia B

Wyeth Research0 个研究点目标入组 25 人开始时间: 2015年4月6日最近更新: 2015年7月10日

适应症

试验速览

暂无简介。

•1.Severe hemophilia B (plasma FIX activity level [FIX:C] less than or equal to [=<]1 percent [%]).
•2.Less than 5 years of age (in order to complete study treatment before age 6).
•3.In the investigator’s judgment, the subject and/or caregiver will be compliant to study procedures.
•4.The parent or legal guardian has voluntarily signed and dated written informed consent before any screening procedures.
•Are the trial subjects under 18? yes
•Number of subjects for this age range: 25
•F.1.2 Adults (18-64 years) no
•F.1.2.1 Number of subjects for this age range
•F.1.3 Elderly (>=65 years) no
•F.1.3.1 Number of subjects for this age range

•1.The subject has a currently detectable FIX inhibitor (or history of an inhibitor) defined as greater than or equal to [>=] 0.6 Bethesda Units (BU). A family history of inhibitors will not exclude the subjects.
•2.Impaired hepatic function defined as greater than (>) 2.5 * Upper limit of normal (ULN) of Alanine aminotransferase (ALT), Aspartate aminotransferase (AST) and/or total bilirubin, except
•in documented hyperbilirubinemia of the newborn.
•3.Impaired renal function (serum creatinine >1.25 *ULN).
•4.Albumin less than (<) Lower limit of normal (LLN).
•5.Platelet count <100,000 per millimetre cube (mm^3).
•6.Abnormal prothrombin time (>1.25 * ULN).
•7.Known hypersensitivity to protein products or agents related to the test article, eg, hamster proteins.
•8.Documented HIV infection; or active infection with hepatitis.
•9.Any genetic coagulation disorder other than hemophilia B (with the exception of vitamin K-dependent coagulation deficiency of the newborn).
•10.Received any investigational drug within 30 days before entering the study.
•11.Any condition (medical or social) that, in the investigator's judgment, makes participation in the study not advisable.

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