NCT00250939已完成2 期
Single-center, Open-label Study of Safety, Pharmacokinetics and Efficacy of rhGAA in Patients With Late-Onset Pompe Disease
Genzyme, a Sanofi Company2 个研究点 分布在 1 个国家目标入组 5 人开始时间: 2005年2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
- 5
- 试验地点
- 2
- 主要终点
- safety and PK profile rhGAA
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 is to evaluate the safety, pharmacokinetics (PK) and efficacy of Myozyme treatment.
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 5 Years 至 18 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •patient's legally authorized guardian(s) must provide signed, informed consent prior to performing any study-related procedures; patient's signature required if patient understands informed consent
- •patient must have a diagnosis of Pompe disease based on deficient endogenous GAA activity or GAA gene mutations
- •patient must have demonstrable muscle weakness
- •patient must be greater than or equal to five years of age and younger than eighteen years of age
- •patient must be able to provide 3 reproducible FVC tests in sitting position during screening
- •patient must perform muscle function testing
- •patient must ambulate 10 meters (assistive devices permitted)
- •patient and legal guardian must comply with the clinical protocol
排除标准
- •patient requires the use of invasive ventilatory support
- •patient requires the use of noninvasive ventilatory support while awake and in an upright position
- •patient has received enzyme replacement therapy with GAA from any source
- •patient has used an investigational product within 30 days prior to study enrollment, or is currently enrolled in another clinical or observational study
- •patient has a medical condition, serious intercurrent illness, or other extenuating circumstance that, may significantly interfere with study compliance, including all prescribed evaluations and follow-up activities
- •Female patients pregnant, lactating or unwilling to practice birth control methods during study
- •Male patients unwilling to use barrier contraceptives during study
结局指标
主要结局
safety and PK profile rhGAA
时间窗: 74 weeks
FVC
时间窗: 74 weeks
MMT
时间窗: 74 weeks
Effect of treatment on muscle function
时间窗: 74 weeks
次要结局
未报告次要终点
研究者
研究点 (2)
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