NCT01132690已完成4 期
A Multicenter, Double-blind, Randomized Safety and Efficacy Study of Two Dose Levels of Taliglucerase Alfa in Pediatric Subjects With Gaucher Disease
适应症
干预措施
相关药物
试验速览
- 阶段
- 4 期
- 状态
- 已完成
- 发起方
- Pfizer
- 入组人数
- 11
- 试验地点
- 3
- 主要终点
- Hemoglobin
研究概览
简要总结
This is a multi-center, double-blind trial to assess the safety and efficacy of taliglucerase alfa in untreated subjects (2 to <18 years old) with Gaucher disease randomly assigned to treatment with one of two doses, 30 or 60 units/kg. Subjects will receive an intravenous (IV) infusion of taliglucerase alfa every two weeks. The total duration of treatment will be 12 months. At the end of the 12-month treatment period eligible subjects will be offered enrollment in an open-label extension study if taliglucerase alfa is not commercially available.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
入排标准
- 年龄范围
- 2 Years 至 17 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Males and females 2 to <18 years old.
- •Diagnosis of Gaucher disease with leukocyte acid β-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).
排除标准
- •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.
研究组 & 干预措施
30 units/kg
Experimental
干预措施: Taliglucerase alfa (Drug)
60 units/kg
Experimental
干预措施: Taliglucerase alfa (Drug)
结局指标
主要结局
Hemoglobin
时间窗: Every 3 months for 12 months
median and interquartile range for change from baseline in haemoglobin
次要结局
- Platelet Count(Baseline and 12 months)
- Spleen Volume(Baseline and Month 12)
- Liver Volume(Baseline and Month 12)
- Chitotriosidase(Every 3 months for 12 months)
- Chemokine (C-C Motif) Ligand 18 (CCL18)(Every 3 months for 12 months)
研究者
研究点 (3)
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