Volatilomic Approaches for the Study of CFTR Modulators
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 入组人数
- 50
- 试验地点
- 2
- 主要终点
- volatile organic compounds (VOC) profile
研究概览
简要总结
This study relies on the hypotheses that (1) exhaled breath is intimately correlated to the patient's lung condition and that (2)the composition of exhaled breath , i.e. the VOCs profile, will be significantly modified from the first days of treatment by CFTR modulators in a or pauci/symptomatic patients such as young children under 12 years old. The non-invasive and longitudinal collection and analysis of exhaled breath may reveal modifications in signaling pathways impacted by these treatments on the very short term. This study is a single-center pilot study.
详细描述
This is a single-center prospective cohort study that plans to include 20 children with cystic fibrosis aged 6 to 12 years old who will initiate Kaftrio® in early 2023.
The children will be monitored for one month; three visits are planned as part of routine care (before initiation of treatment, in the course of the first week and after one month of treatment) during which exhaled breath collection and analysis will also be performed. Access to clinical data collected throughout routine follow-up of these children (analysis of induced sputum, urine and blood, sweat test, respiratory function tests) will be granted upon patient/parent authorization.
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
- 2 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Patients with cystic fibrosis initiating Kaftrio® treatment.
- •Patients and holders of parental authority not opposing participation in this research.
- •Patients affiliated to a Health Insurance system or beneficiaries.
排除标准
- •Patients deprived of liberty or under guardianship.
- •Pregnant or breastfeeding patients.
- •Lung transplanted patients.
结局指标
主要结局
volatile organic compounds (VOC) profile
时间窗: At 0 day, 7 days, and 1 month of treatment
identification of VOCs in exhaled breath with a significant variation between 0 day, 7 days, and/or 1 month of treatment
次要结局
- Weight(At 0 day, 7 days, and 1 month of treatment)
- Induced sputum - immunology(At 0 day, 7 days, and 1 month of treatment)
- Sweat test(At 0 day, 7 days, and 1 month of treatment)
- Induced sputum - microbiology(At 0 day, 7 days, and 1 month of treatment)
- Urine(At 0 day and 1 month of treatment)
- Blood(At 0 day and 1 month of treatment)
- Spirometry(At 0 day, 7 days, and 1 month of treatment)