NCT00113035已完成不适用
Screening Protocol to Evaluate Acid Alpha Glucosidase (GAA) Activity and GAA Gene Mutations in Patients With Late Onset Pompe Disease for Potential Inclusion in Future Clinical Studies With Myozyme (Alglucosidase Alfa)
Genzyme, a Sanofi Company5 个研究点 分布在 1 个国家目标入组 60 人开始时间: 2005年5月最近更新:
适应症
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 发起方
- 入组人数
- 60
- 试验地点
- 5
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 primary objective of this study is to identify potential candidates for future clinical studies in Pompe disease.
研究设计
- 研究类型
- Observational
- 时间视角
- Prospective
入排标准
- 年龄范围
- 8 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Must provide written informed consent prior to any study-related procedures being performed
- •Clinical presentation consistent with late-onset Pompe disease, or a current clinical diagnosis of late-onset Pompe disease
- •Must be greater than or equal to 8 years of age
- •Must be able to ambulate (use of assistive devices, such as a walker, cane or crutches, is permitted)
排除标准
- •Requires the use of invasive ventilatory support
- •Requires the use of noninvasive ventilatory support while awake and in an upright position
- •Use of any investigational product within 30 days prior to study enrollment
- •Unwillingness to comply with protocol requirements
- •Has clinically significant organic disease, unstable medical condition, serious or intercurrent illness
- •Is pregnant or lactating
- •Has participated in the Prospective Observational Study in Patients with Late-Onset Pompe Disease (AGLU02303, "LOPOS")
研究者
研究点 (5)
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