Real-life Follow-up of Cystic Fibrosis Patients Treated With Ivacaftor+Lumacaftor (Orkambi*)
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 入组人数
- 852
- 试验地点
- 1
- 主要终点
- Causes of treatment discontinuation
研究概览
简要总结
The purpose of the study is to examine the real-life safety and effectiveness of the novel combination ivacaftor+lumacaftor in eligible patients with cystic fibrosis (CF). All patients with CF were eligible if they were 12 years and older, started ivacaftor+lumacaftor outside of a clinical trial between December 15th 2017 and December 15th 2018 in an accredited CF center in France. Patient followed-up is based on standardized recommendation of the French Cystic Fibrosis Society. Each patient is followed 1 year.
详细描述
Each patient is followed one year with visits at months 1, 3, 6 and 12.
At each visit, the following data are recorded:
- Treatment discontinuation or not. If the treatment was discontinued, reasons for discontinuation
- Adverse effects
- Lung function (spirometry)
- Body mass index
- Pulmonary exacerbations (intravenous antibiotics)
- Sputum microbiology
- Liver enzymes are measured at each visit
At the initial and 12 visits, a yearly CF examination is proposed to the patients:
- Blood tests
- Chest CT scans
- Body plethysmography
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Retrospective
入排标准
- 年龄范围
- 12 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Patient aged 12 years or older.
- •Patient with Cystic Fibrosis with presence of two mutations DF508 in the CFTR gene
- •Patient treated with ivacaftor+lumacaftor (Orkambi)
排除标准
- •Refusal to participate in the study
- •Start of Orkambi as part of a clinical trial
研究组 & 干预措施
Orkambi treated patients
All patients with CF who started ivacaftor+lumacaftor outside of a clinical trial between January 22nd 2016 and January 22nd 2017.
干预措施: Ivacaftor+lumacaftor (Drug)
结局指标
主要结局
Causes of treatment discontinuation
时间窗: 1 year
Rates of treatment discontinuation
时间窗: 1 year
Timing of treatment discontinuation
时间窗: 1 year
次要结局
- Chloride concentration(1 year)
- Forced vital capacity (FVC)(1 year)
- Forced expiratory volume in 1 sec (FEV1)(1 year)
- Pulmonary exacerbations(1 year)
- Body mass index(1 year)