Exploring Adherence to Preventative Inhaled Therapy and Outcomes for People With Cystic Fibrosis Who Take Ivacaftor/Tezacaftor/Elexacaftor.
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 入组人数
- 1,385
- 试验地点
- 1
- 主要终点
- Change in Forced Expiratory Volume (FEV)
研究概览
简要总结
Cystic fibrosis (CF) is a life-limiting and life-long genetic condition which requires intensive preventative treatment to manage the symptoms and progression of disease. While preventative treatments target the effects of cystic fibrosis, precision medicines target the underlying dysfunction of the cystic fibrosis transmembrane regulator (CFTR) protein at a cell level.
The first of these expensive precision medicines also known as modulator therapies, Ivacaftor, was shown to be highly effective in clinical trials with an increase of over 10% in lung function. Real-world studies showed an increase of only 6% and a return to baseline lung function by year five of treatment. Preventative therapies were continued during the Ivacaftor clinical trials whereas there is real world evidence of declining inhaled preventative therapy use following Ivacaftor initiation. This is a potential explanation for the efficacy-effectiveness gap. The first study in the National Efficacy Effectiveness Modulator Optimisation (NEEMO) programme is exploring this (REC ref: 21/HRA/4940, IRAS 301975).
Ivacaftor/Tezacaftor/Elexacaftor is the most recent modulator available, commissioned in the UK (United Kingdom) in 2020, and suitable for around 90% of people with cystic fibrosis. It is not yet known if the efficacy effectiveness gap seen with Ivacaftor also exists for Ivacaftor/Tezacaftor/Elexacaftor. There is also uncertainty about the continued need for preventative inhaled therapy alongside the prescription of Ivacaftor/Tezacaftor/Elexacaftor. This second study in the NEEMO programme is a cohort, observational study and will explore adherence to inhaled preventative therapies in adults with cystic fibrosis before and after commencing Ivacaftor/Tezacaftor/Elexacaftor, and in those not prescribed Ivacaftor/Tezacaftor/Elexacaftor. It will also look at the relationship between adherence to preventative inhaled therapy and outcome for adults with CF taking Ivacaftor/Tezacaftor/Elexacaftor. The analysis will use routinely collected pseudo anonymised data from the CFHealthHub learning health system (CFHealthHub), alongside anonymised data from the CF registry and routinely collected clinical data.
详细描述
Aim of the study:
To understand the impact of adherence to preventative inhaled therapies on outcomes in people with CF (pwCF) treated with Ivacaftor/Tezacaftor/Elexacaftor.
Background:
Cystic Fibrosis (CF) is a multi-system, life-long genetic condition affecting around 10,500 people in the UK with a median predicted survival of 49 years. The gene mutations responsible for CF result in dysfunctional cystic fibrosis transmembrane conductance regulator (CFTR), which acts as a chloride channel. Impaired chloride secretion in CF leads to sticky and thick secretions for a range of epithelial tissues such as the airways and pancreatic duct. This results in malabsorption, suboptimal nutritional status, recurrent respiratory infections, progressive lung damage and respiratory failure.
Standard treatment for CF, until recently, has focused on managing the symptoms and consequences of CF. Inhaled therapies, specifically inhaled antibiotics, mucolytics and osmotics, are particularly important to reduce exacerbations and maintain lung health in people with CF.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Other
入排标准
- 年龄范围
- 16 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Diagnosis of cystic fibrosis
- •Attend one of the seventeen specialist CF centres who are part of, or have previously been part of, the CFHealthHub learning health system
- •Have given consent for routinely collected clinical data to be used as per the CFHealthHub trials within cohorts consent and/or the CF registry consent
- •Aged 16 or over (attend an adult CF centre)
排除标准
- •Has not given consent for routinely collected clinical data to be used as per the CFHealthHub trials within cohorts consent and/or the CF registry consent. This will be determined at a site level.
结局指标
主要结局
Change in Forced Expiratory Volume (FEV)
时间窗: 5 years
Change from Baseline in Forced Expiratory Volume at 5 years
次要结局
- Change in unadjusted adherence to preventative inhaled therapy(5 years)
- Change in Body Mass Index (BMI)(5 years)
- Change in effective adherence to preventative inhaled therapy(5 years)
- Change in adherence to Ivacaftor/Tezacaftor/Elexacaftor.(5 years)