NCT03347253终止不适用
A Prospective Study in Subjects With Late Onset Pompe Disease Who Are Currently Being Treated With Enzyme Replacement Therapy
适应症
试验速览
- 阶段
- 不适用
- 状态
- 终止
- 入组人数
- 12
- 试验地点
- 21
- 主要终点
- Evaluate degree of change in muscle function and respiratory endpoints over time
研究概览
简要总结
The purpose of the study is to evaluate changes in key clinical outcome measures (eg, motor, respiratory, fatigue) in adult subjects with late-onset Pompe disease (LOPD) subjects receiving standard-of-care enzyme replacement therapy (ERT). Additionally, information gained may be used in the design and conduct of future studies in LOPD subjects.
详细描述
The objective of this study is to evaluate the baseline characteristics and degree of change over time in clinical outcome measures commonly used to evaluate patients with LOPD.
研究设计
- 研究类型
- Observational
- 观察模型
- Case Only
- 时间视角
- Prospective
入排标准
- 年龄范围
- 18 Years 至 75 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Subject has a diagnosis of Pompe disease based on documented deficiency of GAA activity and a documented GAA mutation.
- •Male and female subjects between 18 years and 75 years, inclusive and ≥ 50 kg.
- •Subject must be currently receiving standard-of-care ERT (alglucosidase alfa) at a dose of 20 mg/kg dose every other week.
- •Subject must have been on ERT for the preceding 2 years or more.
- •Subject must have an upright forced vital capacity (FVC) within 35 to 90% of predicted normal (NHANES III reference values), based on the higher of the screening or baseline value, if their 6 minute walk distance (6MWD) is > 200 m. Subject must have an upright FVC within 40 to 90% of predicted normal (NHANES III reference values), based on the higher of the screening or baseline value, if their 6MWD is ≤ 200 m. If FVC is between 80 and 90% of predicted normal, the subject may enter the study if the percent predicted FVC value drops by 10% predicted or more in supine position
- •Subject is able to walk at least 100 m in the 6MWT and the assessment is noted as valid.
排除标准
- •Subject has received any investigational therapy or pharmacological treatment for Pompe disease, other than alglucosidase alfa within 30 days or 5 half lives, whichever is shorter, prior to the Baseline Visit or is anticipated to do so during the course of the study
- •Subject is on any of the following prohibited medications within 30 days of baseline:
- •miglitol (eg, Glyset)
- •miglustat (eg, Zavesca)
- •acarbose (eg, Precose, Glucobay)
- •voglibose (eg, Volix, Vocarb, Volibo)
- •Subject requires use of invasive or non-invasive ventilatory support for > 6 hours a day while awake.
- •Subject has a medical or any other extenuating condition or circumstance that may, in the opinion of the investigator, pose an undue safety risk to the subject or compromise his/her ability to comply with protocol requirements. This includes clinical depression (as diagnosed by a psychiatrist or other mental health professional) with uncontrolled or poorly controlled symptoms.
- •Subject is breastfeeding, or is pregnant or planning to become pregnant within the next 2 years.
- •Other exclusion criteria according to the Lumizyme/Myozyme instructions for use.
结局指标
主要结局
Evaluate degree of change in muscle function and respiratory endpoints over time
时间窗: 6-15 month
To evaluate the degree of change in muscle function and respiratory endpoints over time in patients with Late Onset Pompe disease
次要结局
未报告次要终点
研究者
研究点 (21)
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