NCT01411228已完成3 期
A Multicenter Extension Study 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
- 3 期
- 状态
- 已完成
- 发起方
- Pfizer
- 入组人数
- 15
- 试验地点
- 3
- 主要终点
- Hemoglobin
研究概览
简要总结
A protocol to extend the assessment of the safety and efficacy of taliglucerase alfa in pediatric subjects (2 to <18 years old) with symptoms and clinical manifestations of Gaucher disease who completed treatment in Protocols PB-06-002 (switchover study from imiglucerase) or PB-06-005 (naïve treatment with taliglucerase alfa).
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- Triple (Participant, Care Provider, Investigator)
入排标准
- 年龄范围
- 2 Years 至 18 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Successful completion of Protocol PB-06-002 or PB-06-005
- •The subject, parent(s) or legal guardian(s) signs an informed consent and/or assent
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 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
60 Units/kg
Experimental
干预措施: Taliglucerase alfa (Drug)
30 Units/kg
Experimental
干预措施: Taliglucerase alfa (Drug)
结局指标
主要结局
Hemoglobin
时间窗: Baseline, months 9, 12 and 24
Median and interquartile range. Baseline is the value obtained from the parent study: PB-06-005 for 30 and 60 Units/kg arms and PB-06-002 from Switchover arm.
次要结局
- Chitotriosidase(Baseline, months 9, 12 and 24)
- Spleen Volume(Baseline, months 12 and 24)
- Platelet Count(Baseline, months 9, 12, 24 and 33-36)
- Liver Volume(Baseline, months 12 and 24)
研究者
研究点 (3)
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