NCT02221362终止不适用
A Prospective, Noninterventional, Observational Study of Late-Onset Pompe Disease
适应症
试验速览
- 阶段
- 不适用
- 状态
- 终止
- 入组人数
- 30
- 试验地点
- 15
- 主要终点
- To evaluate the degree of change in respiratory and endurance endpoints over time in patients with Pompe disease
研究概览
简要总结
Study 701-901, a multicenter, multinational, longitudinal, non-interventional observational study in subjects, at least 18 years old, diagnosed with late-onset Pompe disease prospectively collects data to understand clinical progression in terms of respiratory function, symptomology, genotype, biochemistry, endurance and selected subject-reported measures for 24 weeks followed by a 240 week additional observation period for up to 100 subjects.
研究设计
- 研究类型
- Observational
- 观察模型
- Case Control
- 时间视角
- Prospective
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
- •Willing and able to provide written informed consent after the nature of the study has been explained and prior to any study-related procedure
- •Diagnosed with late-onset Pompe disease based on current or previous genomic testing and/or endogenous GAA activity
- •At least 18 years of age at study entry
- •Willing and able to comply with all study procedures
排除标准
- •Requires non-invasive ventilatory support while awake and in the upright position
- •Concurrent disease, medical condition, or extenuating circumstance that, in the opinion of the investigator, might compromise study completion or data collection
- •Unable to perform baseline efficacy assessments
结局指标
主要结局
To evaluate the degree of change in respiratory and endurance endpoints over time in patients with Pompe disease
时间窗: 264 Weeks
次要结局
未报告次要终点
研究者
研究点 (15)
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