NCT00763932已完成2 期
A Multicenter, Open-Label Extension Study of the Long-Term Safety and Efficacy of Recombinant Human Acid α-Glucosidase (rhGAA) in Patients With Pompe Disease (Glycogen Storage Disease Type II) Who Were Previously Enrolled in Genzyme-Sponsored Enzyme Replacement Therapy Studies
Genzyme, a Sanofi Company9 个研究点 分布在 3 个国家目标入组 7 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
- 7
- 试验地点
- 9
- 主要终点
- The objective of this extension study was to monitor the long-term safety and efficacy
研究概览
简要总结
This extension study was to monitor the long-term safety and efficacy of rhGAA treatment in patients with infantile-onset Pompe disease who were previously treated with rhGAA derived from the Synpac cell line
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
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •The patient was enrolled in Protocol AGLU-008-01, AGLU-009-02, or AGLU01502
- •The patient's legal guardian(s) provided written informed consent prior to any study related procedures being performed
- •The patient and his/her guardian(s) were able to comply with the clinical protocol, which required extensive clinical evaluations for an extended period of time.
排除标准
- •Patients were excluded from this study if they did not meet the specific inclusion criteria, or if the patient experienced any unmanageable AE in Protocol AGLU-008-01, AGLU-009-02, or AGLU01502 (as determined and agreed upon by the Principal Investigator and Genzyme Corporation), due to Synpac rhGAA therapy, that would preclude continuing therapy
结局指标
主要结局
The objective of this extension study was to monitor the long-term safety and efficacy
时间窗: 3 years
次要结局
未报告次要终点
研究者
研究点 (9)
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