临床试验/NCT02194881

NCT02194881已完成不适用

Ivacaftor in French Patients With Cystic Fibrosis and a G551D Mutation - Efficacy and Safety Results After the First Year of Treatment in the Real Life Setting.

Assistance Publique - Hôpitaux de Paris1 个研究点分布在 1 个国家目标入组 57 人开始时间: 2014年10月1日最近更新: 2026年3月27日

适应症

干预措施

试验速览

阶段: 不适用
状态: 已完成
发起方: Assistance Publique - Hôpitaux de Paris
入组人数: 57
试验地点: 1
主要终点: FEV1 (in liters and in % predicted)

研究概览研究设计入排标准研究组 & 干预措施结局指标研究者研究点记录与标识符相似试验

研究概览

简要总结

The purpose of this study is to determine whether the treatment with Ivacaftor remains effective and safe in the patients with cystic fibrosis (and at least one G551D CFTR mutation) in the real life setting, after the drug has been approved by the Health authorities.

详细描述

The aims of our study are:

to describe the treated population at initiation of treatment,
to evaluate clinical parameters during the year before Ivacaftor was started, at initiation of treatment and during at least one year of treatment, until June 2014.
to evaluate the tolerance and safety of this treatment.

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: 6 Years 至 —（Child, Adult, Older Adult）
性别: All
接受健康志愿者: 否

入选标准

•French patients with CF aged 6 or older who are homozygous or heterozygous for the G551D mutation
•Treated with Ivacaftor
•First prescription of Ivacaftor before June 1st 2013 (including patients randomized in the VX770 clinical trials)

排除标准

•CF patients younger than 6 years old
•CF patients who have received lung transplantation
•CF patients without a G551D mutation.

研究组 & 干预措施

Ivacaftor 1

patients with CF who are homozygous or heterozygous for the G551D mutation and treated with Ivacaftor

干预措施: CF patients with a G551D mutation and treated with Ivacaftor (Drug)

结局指标

主要结局

FEV1 (in liters and in % predicted)

时间窗: until one years after initiation of treatment

pulmonary function

次要结局

number of antibiotic treatments and number of days of antibiotic treatments(until one year after initiation of treatment)
number of hospitalizations and number of days of hospitalization per year(until one year after initiation of treatment)
pulmonary exacerbations(until one years after initiation of treatment)
Adverse events(until one year after initiation of treatment)
respiratory colonization(until least one year after initiation of treatment)
nutritional status(until one year after initiation of treatment)

研究者

发起方

Assistance Publique - Hôpitaux de Paris

申办方类型

Other

责任方

Sponsor

研究点 (1)

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