NCT00125879已完成2 期
A Long-Term Continuation Study of Patients With Infantile-Onset Pompe Disease Who Were Previously Enrolled in Protocol AGLU01602
Genzyme, a Sanofi Company28 个研究点 分布在 7 个国家目标入组 16 人开始时间: 2005年6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
- 16
- 试验地点
- 28
- 主要终点
- Long-term Safety and Efficacy
研究概览
简要总结
Pompe disease (also known as glycogen storage disease type II) is caused by a deficiency of a critical enzyme in the body called acid alpha-glucosidase (GAA). Normally, GAA is used by the body's cells to break down glycogen (a stored form of sugar) within specialized structures called lysosomes. In patients with Pompe disease, an excessive amount of glycogen accumulates and is stored in various tissues, especially heart and skeletal muscle, which prevents their normal function. The overall objective of this study is to evaluate the long-term safety and efficacy of Myozyme treatment in patients with infantile-onset Pompe disease.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •The patient's legal guardian(s) must provide written informed consent prior to any study-related procedures being performed
- •The patient and his/her legal guardian(s) must have the ability to comply with the clinical protocol
- •The patient must have completed Protocol AGLU01602.
排除标准
- •Patient has experienced any unmanageable adverse event (AE) in Protocol AGLU01602 due to Myozyme that would preclude continuing treatment with Myozyme
结局指标
主要结局
Long-term Safety and Efficacy
时间窗: 52 weeks
次要结局
未报告次要终点
研究者
研究点 (28)
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