NCT02191917终止不适用
A Study of Respiratory Muscle Strength, Including Effort-Independent Measures, in Subjects With Late-Onset Pompe Disease
适应症
试验速览
- 阶段
- 不适用
- 状态
- 终止
- 入组人数
- 8
- 试验地点
- 2
- 主要终点
- Maximal Inspiratory Pressure (MIP)
研究概览
简要总结
Study 701-201 is a study in patients with late-onset Pompe disease (LOPD). The study will test respiratory muscle strength initially and again after 24 weeks in subjects treated or not treated with BMN 701 .
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 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 procedures
- •Documented diagnosis with late-onset Pompe disease - At least 18 years of age at study entry
- •Willing and able to comply with all study procedures
排除标准
- •Requires 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 patient well-being, study completion, or data collection
- •Allergy to tools or procedures used for respiratory muscle testing
结局指标
主要结局
Maximal Inspiratory Pressure (MIP)
时间窗: Baseline, Week 28
Pulmonary measure of change in maximal inspiratory pressure (MIP)
次要结局
- Maximal Expiratory Pressure (MEP)(Baseline, Week 28)
研究者
研究点 (2)
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