A double-blind, randomised efficacy and safety study of taliglucerase alfa enzyme replacement therapy in children and adolescents with Gaucher disease (non-neuronopathic and chronic neuronopathic)

Protalix Biotherapeutics0 个研究点目标入组 10 人开始时间: 2012年4月10日最近更新: 2012年4月18日

适应症

试验速览

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•Males and females 2 to <18 years old.
•Diagnosis of Gaucher disease with leukocyte acid ß-glucosidase activity =30% of the mean of the reference range for healthy subjects.
•Subjects who have not received enzyme replacement therapy (ERT) in the past or who have not received ERT in the past 12 months and have a negative anti-glucocerebrosidase antibody assay.
•Subjects who have not received substrate reduction therapy (SRT) in the past 12 months.
•Subjects whose clinical condition, in the opinion of the investigator, requires treatment with enzyme replacement therapy (ERT).
•Are the trial subjects under 18? yes
•Number of subjects for this age range: 10
•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

•Currently taking another investigational drug for any condition.
•Presence of neurological signs and symptoms characteristic of Gaucher disease with complex neuronopathic features other than longstanding oculomotor gaze palsy.
•Presence of unresolved anemia due to iron, folic acid, or vitamin B12 deficiency
•Previous hypersensitivity reaction to Cerezyme® (imiglucerase) or Ceredase® (alglucerase).
•History of allergy to carrots.
•Presence of HIV, HBsAg or hepatitis C infections.
•Subject's parent(s) or legal guardian(s) are unable to understand the nature, scope and possible consequences of the study.
•Presence of any medical, emotional, behavioral or psychological condition that in the judgment of the Investigator would interfere with the subject's compliance with the requirements of the study.

发起方