Prospective Surveillance of Lung Development During Childhood, Adolescence and Adulthood in Healthy and Patients With Cystic Fibrosis
试验速览
- 阶段
- 不适用
- 状态
- 进行中（未招募）
- 入组人数
- 250
- 试验地点
- 1
- 主要终点
- Multiple Breath Washout
研究概览
简要总结
Cystic fibrosis (CF) is the most common lethal inherited disease in Caucasian populations. To improve survival, it is essential to understand the development, progression and treatment of CF lung disease throughout early childhood.
Therefore the overall objective is to prospectively assess the clinical utility of novel and non-invasive measuring methods, namely Multiple Breath Washout and functional lung MRI in the longitudinal clinical surveillance of patients with CF and compare the results to those of healthy controls.
详细描述
Background and project rationale:
Cystic fibrosis (CF), the most common lethal inherited disease in Caucasian populations, affects approximately 1:2500 live births. It is a multisystem disorder with respiratory morbidity and mortality being the leading cause of death. Despite improved survival in successive birth cohorts, the current median survival age of patients with CF is about 40 years. To improve survival, it is essential to understand the development, progression and treatment of CF lung disease throughout early childhood. Therefore tracking of lung function throughout childhood may provide important insights into the development and progression of CF lung disease. Spirometry, the standard lung function test for decades, is sensitive only for airflow limitation arising in large airways and insensitive for assessment of small or peripheral airway involvement, whereby the CF lung disease emerges in the small airways. Two promising techniques to assess small airway function in young children include the multiple breath washout (MBW) lung function test and functional Matrix-Pencil magnetic resonance imaging (MP-MRI).
Project Objectives and Design:
The overall objective of this project is to prospectively assess the clinical utility of MBW and MP-MRI in the longitudinal clinical surveillance of patients with CF. Therefore this study: i) Examines differences in MBW and MP-MRI outcomes between patients with CF and healthy controls. ii) Assesses the short term (over 1h) repeatability of MBW and MP-MRI outcomes in patients with CF and healthy controls. iii) Assesses whether MBW and MP-MRI outcomes are associated with clinical lung disease in patients with CF. iv) Determines whether changes in MBW and MP-MRI outcomes are associated with progression of lung disease in patients with CF. v) Compares the breath-by-breath regional and temporal changes in functional MRI signal with breath-by-breath changes in MBW phase outcomes in patients with CF and healthy controls.
Methods:
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
- 3 Years 至 18 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 是
入选标准
- •Individuals with CF:
- •Diagnosis of CF
- •Signed written informed consent
- •≥3 - 18 years of age, depending on the cooperation and if lung function measurements are possible
- •Healthy volunteers:
- •Signed written informed consent
- •Informed consent of participant and if under 18 years, legal representative respectively
- •Children and adults with no history of chronic lung disease or acute respiratory infection in the four weeks prior to the study visit
- •≥3 - 18 years of age, depending on the cooperation and if lung function measurements are possible
排除标准
- •The presence of any one of the following exclusion criteria will lead to exclusion of the participant, for example:
- •Women who are pregnant or breast feeding.
- •Intention to become pregnant during the course of the study
- •Lack of safe contraception, defined as: Female participants of childbearing potential, not using and not willing to continue using a medically reliable method of contraception for the entire study duration, such as oral, injectable, or implantable contraceptives, or intrauterine contraceptive devices, or who are not using any other method considered sufficiently reliable by the investigator in individual cases.
- •Please note that female participants who are surgically sterilised/hysterectomised or post-menopausal for longer than 2 years are not considered as being of child bearing potential.
- •Other clinically significant concomitant disease states (e.g. renal failure, hepatic dysfunction, cardiovascular disease, etc.)
- •Known or suspected non-compliance, drug or alcohol abuse
- •Continuous glucose monitor
- •Inability to follow the procedures of the study, e.g. due to language problems, psychological disorders, etc. of the participant
- •Metal in body, e.g. pacemaker
- •Participation in another study with investigational drug within the 30 days preceding and during the present study
- •Subjects which are respiratory insufficient to attend on the lung function measurements (oxygen demand)
- •Subjects who are unable to perform the MRI without sedation
- •Participants which were born preterm (<
- •week of pregnancy)
- •Current smokers
- •In addition for individuals with CF:
- •Known diseases other than related to CF
- •In addition for healthy individuals:
- •Current upper respiratory infection (cough, cold, fever) will lead to postponement of the visit to 4 weeks after the end of symptoms
结局指标
主要结局
Multiple Breath Washout
时间窗: Every third month up to 50 years. Healthy controls only during 1 year.
Longitudinal assessment of lung volume and ventilation inhomogeneity
Functional MP-MRI
时间窗: Every twelfth month up to 50 years. Healthy controls only during 1 year (2 time points).
Longitudinal assessment of percentage of the lung volume with impaired fractional ventilation and relative perfusion
次要结局
- Spirometry: FEF(Every third month up to 50 years. Healthy controls only during 1 year.)
- Morphological MRI(Every twelfth month up to 50 years. Healthy controls only during 1 year (2 time points))
- Body plethysmography: sRAW(Every third month up to 50 years. Healthy controls only during 1 year.)
- Spirometry: FEV1(Every third month up to 50 years. Healthy controls only during 1 year.)
- Spirometry: FVC(Every third month up to 50 years. Healthy controls only during 1 year.)
- Respiratory symptoms(Every third month up to 50 years. Only CF patients.)
- CF-related quality of life(Every third month up to 50 years. Only CF patients.)
- Body plethysmography: FRC(Every third month up to 50 years. Healthy controls only during 1 year.)
- Exacerbations(Every third month up to 50 years. Only CF patients.)
- Microbiology: presence of respiratory pathogens(Every third month up to 50 years. Only CF patients.)
- Body plethysmography: TLC(Every third month up to 50 years. Healthy controls only during 1 year.)
- Microbiology: abundance of respiratory pathogens(Every third month up to 50 years. Only CF patients.)
研究者
Kathryn Ramsey
Principal Investigator
Insel Gruppe AG, University Hospital Bern