Evaluation of the Response to CFTR Modulators in Patients With Cystic Fibrosis Less Than 18 Years of Age
试验速览
- 阶段
- 不适用
- 状态
- 招募中
- 发起方
- 入组人数
- 600
- 试验地点
- 1
- 主要终点
- Lung Imaging
研究概览
简要总结
CFTR modulators should improve the prognosis of Cystic Fibrosis. Identifying patients under the age of 18 responding to CFTR modulators as well as detecting possible toxicity is an important medical objective given the potential side effects and the high cost of these molecules.
This observational follow-up cohort study is carried out as part of routine care.
The main objective is to assess the evolution of pulmonary structural impairment by low-dose CF scan at the end of the first year of CFTR modulator therapy.
The secondary objectives are to evaluate structural impairment at low dose scan at 3 years and 5 years of CFTR modulator treatment, the evolution of respiratory functional parameters, growth, puberty, lung infection, sweat test, quality of life and pancreatic function, as well as tolerance of modulators including liver toxicity.
详细描述
Cystic fibrosis (CF) is a deadly disease. This is due to overinfected chronic obstructive pulmonary disease that progresses to end-stage respiratory failure. CFTR modulators should improve the prognosis of CF, as they may slow the progression of patients' lung disease. Assessing their impact in the paediatric population is becoming a major issue. Children and adolescents under the age of 18 are a target cohort because they have a lung disease that is still poorly developed. Early prescription of CFTR modulators is therefore a priority but requires evidence of absence of toxicity. Identifying patients under the age of 18 responding to CFTR modulators as well as detecting possible toxicity, is an important medical objective given the potential side effects and the high cost of these molecules.
The outcomes previously used in Phase III studies (FEV1, frequency of exacerbations, nutritional status) are insufficiently sensitive in this population.
Other criteria need to be analyzed to identify the response to CFTR modulators in the short and medium term. The investigators hypothesize that the assessment of pulmonary structural impairment by low-dose lung CT-scan as part of routine care could be a much more sensitive criterion for the development of lung disease under CFTR modulators.
This observational follow-up cohort study is carried out as part of routine care. It does not involve a specific collection for research. Excess bronchial secretions and blood will be kept instead of being discarded in the event of a possible requalification for research.
The main objective is to assess the evolution of pulmonary structural impairment by low-dose CF scan at the end of the first year of CFTR modulator therapy The secondary objectives are to assess following criteria
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
- 否
入选标准
- •Children with cystic fibrosis under the age of 18 under CFTR modulator therapy
排除标准
- •Patients with cystic fibrosis without indication for CFTR modulator therapy
- •Patients over the age of 18
- •Pregnant or lactating women
研究组 & 干预措施
CF children treated with CFTR modul
Cystic fibrosis patients under 18 years treated with CFTR modulators according to french health recommendations observational cohort study
干预措施: CFTR Modulators (Drug)
结局指标
主要结局
Lung Imaging
时间窗: at 5 years, as part of national guidelines
Lung structural injury assessed by Low Dose CT, as part of routine care
次要结局
- Lung Clearance Index - Lung Clearance Index(longitudinal monitoring of assessments carried out as part of routine care during 5 yrs)
- colonization of bronchial secretions(longitudinal monitoring of assessments carried out as part of routine care during 5 yrs)
- weight in kilogrammes(longitudinal monitoring of assessments carried out as part of routine care during 5 yrs)
- bronchial infectious exacerbations(longitudinal monitoring of assessments carried out as part of routine care during 5 yrs)
- quality of life questionnaire(longitudinal monitoring of assessments carried out as part of routine care during 5 yrs)
- height in meters(longitudinal monitoring of assessments carried out as part of routine care during 5 yrs)
- pubertal evolution(longitudinal monitoring of assessments carried out as part of routine care during 5 yrs)
- Forced Vital Capacity (FVC)(longitudinal monitoring of assessments carried out as part of routine care during 5 yrs)
- Force Expiratory Flow 50 (FEV50)(longitudinal monitoring of assessments carried out as part of routine care during 5 yrs)
- Forced Expiratory Flow 25-75 (FEV25-75)(longitudinal monitoring of assessments carried out as part of routine care during 5 yrs)
- sweat test(longitudinal monitoring of assessments carried out as part of routine care during 5 yrs)
- puberty(longitudinal monitoring of assessments carried out as part of routine care during 5 yrs)
- intestine inflammation(longitudinal monitoring of assessments carried out as part of routine care during 5 yrs)
- side effects: declarative collection and monitoring(longitudinal monitoring of assessments carried out as part of routine care during 5 yrs)
- Forced Expiratory Volume in 1 second(FEV1)(longitudinal monitoring of assessments carried out as part of routine care during 5 yrs)
- Residual Volume (RV)(longitudinal monitoring of assessments carried out as part of routine care during 5 yrs)
- Total Pulmonary Capacity(longitudinal monitoring of assessments carried out as part of routine care during 5 yrs)
- ENT quality of life questionnaire(longitudinal monitoring of assessments carried out as part of routine care during 5 yrs)
- Abdominal quality of life questionnaire(longitudinal monitoring of assessments carried out as part of routine care during 5 yrs)
- liver ultrasound(longitudinal monitoring of assessments carried out as part of routine care during 5 yrs)
- serum and fecal pancreatic biological markers(longitudinal monitoring of assessments carried out as part of routine care during 5 yrs)
- bone biological markers(longitudinal monitoring of assessments carried out as part of routine care during 5 yrs)
- bone maturation(longitudinal monitoring of assessments carried out as part of routine care during 5 yrs)
- elastometry (data available in centers with the necessary equipment)(longitudinal monitoring of assessments carried out as part of routine care during 5 yrs)
- glycemic regulation(longitudinal monitoring of assessments carried out as part of routine care during 5 yrs)
研究者
Isabelle SERMET-GAUDELUS
Professor Isabelle Sermet-Gaudelus
Societe Francaise de la Mucoviscidose