NCT00765414已完成2 期
An Open-Label Extension Study of the Long-Term Safety and Efficacy of Recombinant Human Acid α-Glucosidase (rhGAA) Given as Enyzme Replacement Therapy to a Single Patient With Pompe Disease (Glycogen Storage Disease Type II) Who Were Previously Enrolled in Genzyme-Sponsored Enzyme Replacement Therapy Studies
Genzyme, a Sanofi Company1 个研究点 分布在 1 个国家目标入组 1 人开始时间: 2003年4月1日最近更新:
适应症
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 发起方
- 入组人数
- 1
- 试验地点
- 1
- 主要终点
- The objective of this extension study was to monitor the long-term safety and efficacy of a single patient
研究概览
简要总结
This extension study was to monitor the long-term safety and efficacy of rhGAA treatment in a single patient with infantile-onset Pompe disease who were previously treated with rhGAA in a Genzyme study.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 16 Years 至 —（Child, Adult, Older Adult）
- 性别
- Female
- 接受健康志愿者
- 否
入选标准
- •Provide written informed consent prior to participating in any study related procedures;
- •Currently enrolled in Protocol AGLU01402
- •Have the ability to comply with the clinical protocol, which required extensive clinical evaluations for an extended period of time.
排除标准
- •Was pregnant or unwilling to use approved birth control during the course of the study;
- •Had experienced any unmanageable AEs under Protocol AGLU01402 (as determined and agreed upon by the Principal Investigator and sponsor) due to rhGAA that would preclude continuing ERT;
- •Was participating in any other investigational study.
结局指标
主要结局
The objective of this extension study was to monitor the long-term safety and efficacy of a single patient
时间窗: 3 years
次要结局
未报告次要终点
研究者
研究点 (1)
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