Enterobacteria Strain-level Characterization in Children With Inborn Errors of Metabolism (IEM)
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 发起方
- 入组人数
- 22
- 试验地点
- 1
- 主要终点
- Contribution of micriobiota to disease severity
研究概览
简要总结
Study around children with inborn errors of metabolism (IEM) and their healthy siblings. Collection of stool and urine to assess contribution of microbiota to disease severity.
详细描述
This study aims to collect biological samples (stool and urine) from children with inborn errors of metabolism, IEM (like UCD (urea cycle disorder), PA (propionic aciduria) and MMA (methylmalonic aciduria) and their healthy siblings.
The main focus of the study is to assess the contribution of the intestinal microbiota to disease severity in children that suffer from different forms of IEM and potentially find microbiota targets that could be used in the design of therapeutic/prophylactic agents.
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
- — 至 18 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 是
入选标准
- •Children or siblings of children that have been diagnosed with one of the following diseases: any type of UCD, PA or MMA
排除标准
- •Children or siblings of children that have not been diagnosed with one of the following diseases: any type of UCD, PA or MMA
- •use of an investigational drug or device less than 30 days prior to the study, or current enrollment in another investigational drug or device study less than 30 days prior to the study
- •considered to be poor attendees or unlikely for any reason to be able to comply with the study procedures, in the opinion of the investigator
结局指标
主要结局
Contribution of micriobiota to disease severity
时间窗: 6 months
Assess contribution of intestinal microbiota to disease severity in children that suffer from different forms of IEM via changes in frequencies, absolute colonization levels or strain identity of microbiota species
次要结局
- Disease aggravating microbiota (microbiota target)(6 months)
研究者
Emma Marie Caroline Slack
Prof. Dr.
ETH Zurich