NCT03045042Unknown不适用
Search for Serum/Plasma Biomarkers in Pompe's Disease
Fundació Institut de Recerca de l'Hospital de la Santa Creu i Sant Pau1 个研究点 分布在 1 个国家目标入组 35 人开始时间: 2017年4月最近更新:
适应症
干预措施
相关药物
试验速览
- 阶段
- 不适用
- 发起方
- 入组人数
- 35
- 试验地点
- 1
- 主要终点
- MicroRNA in serum samples
研究概览
简要总结
This study aims to analyze serum and plasma samples from patients with late onset Pompe disease treated and not treated with enzyme replacement therapy (ERT) to identify microRNA that could be specific of the disease. The investigators will correlate the concentration of these microRNA with several muscle function tests and quantitative muscle MRI to know whether they are good biomarkers of progression.
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
- 2 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 是
入选标准
- •Genetic confirmation of Pompe disease
排除标准
- •Pregnancy
研究组 & 干预措施
Treated patients
Patients with late onset Pompe disease treated with the enzyme replacement therapy
干预措施: Enzyme Replacement Agent (Drug)
结局指标
主要结局
MicroRNA in serum samples
时间窗: Baseline
Investigators are going to study microRNA following a microarray analysis of serum samples from patients treated and non treated
次要结局
- Muscle function test(Baseline and one year)
- Whole body muscle MRI(Baseline a and one year)
- Changes in microRNA concentration(One year period)
研究者
研究点 (1)
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