Active clinical trials for "Cystic Fibrosis"

Specific Aim 1. Determine if valid results of non-sedated MRI based assessments of liver stiffness and lipid content can be obtained in more than 90% of children and young adults with cystic fibrosis. Specific Aim 2. Determine hepatic lipid content using the HepaFat sequence and liver stiffness using MRE. Investigators will compare the results obtained by MRI with PUSH study grayscale ultrasounds in CF patients with normal, heterogeneous, homogeneous or nodular (cirrhotic) pattern on ultrasound. Specific Aim 3. Creation of an imaging core lab to centralize evaluation of MR imaging data, allow for remote image upload, electronic data storage, and remote image viewing/interpretation. This infrastructure will be utilized to standardize image post processing. Specific Aim 4. Using the longitudinal PUSH study, determine if MRI based imaging improves discrimination of subjects at risk for progression to advanced CF liver disease (development of cirrhosis) compared to using US imaging alone

This is a pilot cross-sectional study of measured transcutaneous CO-oximetry in children with inflammatory and non-inflammatory conditions.

Evolution of medical care for patients with cystic fibrosis appeared in recent years: prolongation of life expectancy with consequently increased comorbidities and the use of lung transplantation, systematic neonatal screening of new born since 2002, prescription of expensive new molecules or new presentation. This evolution justifies to realize an update of statistics of costs of care and to assess adherence to treatment

In order to further enable physiopathology studies, the aim of this project is to validate an in vitro model of cystic fibrosis created using induced pluripotent stemcell (iPS) differentiated bronchial epithelium from cystic fibrosis (CF) patients homozygous for the p.Phe508del mutation of the cystic fibrosis transmembrane conductance regulator (CFTR) gene.

Our primary purpose is to compare the prevalence of sleep disorders in children aged 6 to 17 with cystic fibrosis versus controls with a Sleep disorder screening score, the SDSC. Our hypothesis is that patients aged 6 to 17 with cystic fibrosis have a higher prevalence of sleep disturbances than the general population of the same age group. Our secondary hypothesis is that these sleep disorders are mixed and that there are non-respiratory causes, sometimes modifiable by simple non-medical treatment and that's why our secondary purpose is to identify the responsible factors, in particular non-respiratory factors in the 2 groups and to compare them.

Cystic fibrosis (CF) is the most common inherited disease in the western world. On a yearly basis, 56% of CF patients, or nearly 17,000 individuals in the US, suffer from acute pulmonary exacerbations (APE). The purpose of this study is to test a candidate assay for its ability to diagnose APE, the most important disease event in CF. While previous studies have been able to identify biomarkers of CF prognosis and risk stratification, three markers have demonstrated characteristics ideal for APE diagnosis: CD64, TLR2, and GILT. CD64 is a cellular receptor, expressed on numerous cells of the immune system, whose role is to bind antibodies which are attached to infected cells or pathogens. TLR2 plays a major role in early host-microbial interactions. GILT has been shown to be more precise in targeting immune responses against antigens and influences T lymphocyte response. This study looks to identify the differences in the expression of neutrophil CD64 and CD4+ T cell TLR2 and GILT between acute illness and baseline health as a sensitive marker of acute pulmonary exacerbation so that it may facilitate rapid hematologic diagnosis of the condition. The study also looks to compare sensitivity and specificity of the assays above to standard measures, such as health related quality of life scores (CFQ-R), loss of lung function, white blood cell counts and CRP, for diagnosing acute exacerbations.

Staphylococcus aureus is not only one of the first pathogens infecting the airways of cystic fibrosis (CF) patients, but also a highly prevalent microorganism (>60% of all CF patients; European and American CF registries; (4,25), which often persists for several years in the respiratory tract of CF patients. The purpose of this study is to dissect infection by S. aureus from colonization. Therefore, the following non-interventional prospective, longitudinal multicenter study will be conducted to develop the following hypothesis: CF patients with high bacterial loads are more likely to be infected by S. aureus than patients with low bacterial loads. Primary endpoint: bacterial load of sputum cultures Secondary endpoints: nasal carriage molecular analysis of S. aureus (Monoclonal/polyclonal) serum: S. aureus-specific antibodies, S100A12, IL-8, TNF-alpha sputum: S100A12, IL-8, myeloperoxidase S. aureus therapy regimens lung function tests: FEV1, deltaFVC , deltaMEF25 BMI development Inclusion criteria: S. aureus cultures for more than 6 months within the last year, children (>6 years) and patients, who are able to perform lung function tests Exclusion criteria: P. aeruginosa and/or B. cepacia cultures from the specimens for more than 6 months within the last year before recruitment or during the study period In addition to microbiological investigations and clinical laboratory tests, the actual clinical situation will be evaluated and reported during the study period. The results of this observational study will be used to carefully plan a clinical interventional study. Furthermore, with the results it might be possible to characterize a subpopulation of patients, which is at greater risk for S. aureus infections.

This is a research study where researchers are collecting blood to evaluate the genetic characteristics of individuals with chronic lung diseases, including asthma, COPD (chronic obstructive pulmonary disease), interstitial lung disease, cystic fibrosis, and lung cancer. The investigators hope to be able to identify an association between a genetic make-up in the blood samples and the risks of developing a particular lung disease, or severity of a lung disease. The findings of this study might be important to develop future preventative methods and potential treatments for the management of lung disease.

OBJECTIVES: I. Determine the amount of hepatic glucose production derived from gluconeogenesis and glycogenolysis in the post-absorptive state in patients with cystic fibrosis. II. Determine de novo lipogenesis in relationship to resting energy expenditure in this patient population.

OBJECTIVES: I. Determine the prevalence of nontuberculous mycobacteria in sputum cultures from patients with cystic fibrosis. II. Compare the clinical course of patients with negative versus positive cultures.