Active clinical trials for "Cystic Fibrosis"

Repeated measurements of lung water by single transpulmonary thermodilution during the first postoperative days following lung transplantation

Pulmonary infections are the major cause of mortality and morbidity in cystic fibrosis (CF); patients frequently have to take antibiotics which often cannot be given orally or by aerosol but have to be administered intravenously. In order to reduce the number of venepunctures, totally implanted venous access devices (TIVAD) or Ports have been used to administer antibiotics and other infusions. The use of Port systems has been increasing in recent years, especially for those patients requiring frequent intravenous treatments. Having a TIVAD in place makes venous access quicker and also reduces trauma, suffering and pain. However, there are important complications associated with TIVADs which can be early (pneumothorax, arterial puncture, severe bruising) or late (infections, thromboembolic complications and occlusion). Although the use of TIVADs in CF is increasing, there is little CF-specific literature available on the epidemiology and risk of TIVAD complications. Also, literature is scarce about clinical criteria for deciding to insert a TIVAD. Therefore, so far clinical decisions were based mainly on experiences of TIVAD use in other diseases, such as cancer. With this prospective observational study we will survey a large population of Italian CF people with TIVAD in order to: collect data about current clinical conditions of CF people with TIVAD; investigate about clinical criteria that led to the decision of positioning a TIVAD; observe the possible onset of late complications.

In patients with Cystic Fibrosis (CF) the clinical course of lung disease is crucial for individual prognosis and life expectancy. Imaging modalities are important in the assessment of follow up of structural lung changes and monitoring of pulmonary complications in CF. Although high resolution computed tomography (HRCT) is the accepted gold standard for evaluation of morphological lung changes in CF, chest-X-ray is widely used as standard imaging procedure for assessment and follow up in these young patients due to less radiation exposure. Magnetic resonance imaging (MRI) has not been used for lung imaging in CF so far. Studies from the 80's and early 90's were not able to show any impact for the use of MRI in CF. Due to recent technical developments MRI of the lung became possible. Our study group was able to show that MRI is a competitive imaging modality for evaluating changes of the CF-lung in comparison to the gold standard (HRCT). So far only patients from the age of 6-7 years were examined. According to recent studies CF is a disease which starts in utero. Therefore it can lead to extensive pulmonary changes even in infants and young children. In this age group lung function testing is difficult and not broadly available. An early optimized therapy is crucial for the long term course and outcome of the pulmonary disease. The aim of this study is to evaluate morphological and functional MRI for early diagnosis of lung changes in children (0-6 years) with CF.

Background By means of measurements of series of non-invasive inflammatory markers in exhaled breath (condensate), a reflection of inflammatory processes and oxidative stress, can be obtained. Thereby, these techniques could be important in monitoring asthma and CF lung disease in children. Fractional exhaled nitric oxide (FeNO) and inflammatory markers in exhaled breath condensate (EBC) reflect ongoing inflammation and oxidative stress in the airways. These markers have a promising capacity for monitoring diagnoses of CF and asthma lung disease. Aim To study the course of inflammatory markers in time in children with asthma and CF, in stable periods and during pulmonary exacerbations. In addition, we study the ability of inflammatory markers to predict safe tapering of medical treatment in both populations. To study the course of inflammatory markers in EBC during an exacerbation. To study which IM are already elevated before a clinical exacerbation is evident and can predict exacerbations in time. To study which inflammatory markers can predict safe discontinuation of antibiotics in children with CF, or tapering of inhaled corticosteroids in children with asthma. To study the relationship between inflammatory markers in EBC, the severity and control of CF and asthma, the symptoms and lung function within patients will be analysed. Methods Children with CF (n=30) and children with asthma (n=40) were recruited included from our outpatient clinic. During this longitudinal study patients visit the outpatient clinic were followed-up for 12 months; every two months during one year. patients visited our outpatient clinic. In addition to these standard visits, During exacerbations patients four extra visits were planned during an exacerbation. were asked to visit the University Hospital Maastricht four times. These additional visits were planned with a maximum of two times during the study. By means of a home monitor, children were asked to assess measurements of Besides measurements in the University Hospital, children measured forced expiratory volume in one second (FEV1) at home using a home monitor, to record medication use, and, to record presence and severity of pulmonary symptoms. Outcome parameters were: 1) FeNO assessment in exhaled air, 2) inflammatory markers in EBC, 3) lung function parameters, 4) specific questionnaires to assess asthma and CF control and severity, 5) data originating from the home monitor.

This is a pilot study of inhaled antibiotic regimens is a pilot study examining clinical and laboratory endpoints of patients on inhaled antibiotic treatments. We hypothesize that alternation therapy utilizing Cayston and Tobi will inhibit antibiotic resistance and that alternation therapy will result in a decreased incidence of antibiotic resistance to Cystic Fibrosis (CF) microbial isolates. The long term strategic goal is to develop a model biometric system for selecting a patient's optimal inhaled antibiotic regimen by utilizing clinical and microbiological parameters.

Cystic fibrosis (CF) affects men and women equally, but after the onset of puberty, women with CF have a lower life expectancy than men with CF. Despite these known differences, the link between CF symptom trends and the menstrual cycle remains critically understudied. To address this gap, this study will investigate changes in CF-specific symptoms among women with CF to evaluate whether and how they correlate with their menstrual cycle. Specifically, the investigators hope to examine whether CF-related symptoms change throughout the menstrual cycle, what the impact of those symptoms is on quality of life, and how feasible it is to use a period tracking app to track CF-related symptoms throughout the menstrual cycle. Investigators are asking women ages 18-45 with CF, who have regular menstrual cycles, to participate. Study procedures, including online surveys, period tracking, and interview, will take approximately 3 months.

Background : Diabetes is correlated to an increase in patient morbidity and mortality. The pathophysiology of diabetes is still poorly understood. Significant variations in blood sugar have been shown over time in patients with cystic fibrosis with or without diabetes. No study has evaluated the association between the nutritional profile and the glucose tolerance in adult with cystic fibrosis, by integrating data on energy consumption (detailed nutritional profile) and energy expenditure (rest energy expenditure, body composition, and physical activity). Given the epidemiological changes in cystic fibrosis correlated to the increased prevalence of diabetes and the clinical impact of nutritional status, the association between these factors remains to be studied. Objectives of the study : - Main objective : To describe adult patients with cystic fibrosis in terms of : Nutritional profile Food behaviour Physical activity Rest energy expenditure Body composition - Secondary objectives : To research the association between the nutritional profile and the glucose tolerance.

The general aims of this project are (i) to identify predictive epigenetic biomarkers of lung disease severity in Cystic Fibrosis, (ii) to characterize a non-invasive cellular model, spontaneous sputum, for the analysis of these epigenetic biomarkers, (iii) to analyze the variations in DNA methylation for a same patient over time.

This is a prospective, multicenter, observational study designed to collect gastrointestinal related data in patients with Cystic Fibrosis (CF).

Specific aims: To elucidate the biological mechanism that leads to pulmonary and nutritional improvement in CF patients following treatment with ivacaftor using advanced techniques to assess changes of the pulmonary and nutritional status To examine the relation between the individual response to ivacaftor and the presence of modifier genes associated with CF disease severity, To assess altered CFTR function using new available in vivo tests, To validate newly developed in vivo sweat tests with well established functional tests, To establish correlation between the CFTR response to Vx-770 measured in a new ex vivo method (organoids) and the actual clinical and/or functional response in individual patients, To examine response in other CF-specific features such as aqua wrinkling. To examine if sleep/activity level changes. To establish a biorepository to enable further investigations.