Active clinical trials for "Cystic Fibrosis"

Cystic fibrosis is so far a disease whose treatment remains essentially symptomatic care teams perceive the boundaries of a support mainly focused on drug treatments. The patient had no other choice but to learn to live with their disease, the goal is to make every effort so that tames the disease while maintaining high self-esteem that gives meaning to his life, allowing, among other things, a good grip of treatment. Through this research we hope to encourage the integration of art therapy in the course of care of cystic fibrosis patients, from children to adults before and after transplantation, in connection with the patient education and counseling

The academic investigator - initiated trial will evaluate in a postapproval setting whether, and if yes, to what extent and variability, the treatment with lumacaftor in combination with ivacaftor reverses the p.Phe508del CFTR - mediated basic defect in p.Phe508del homozygous subjects with cystic fibrosis under real life conditions.

The investigators know that adherence to medications in children with cystic fibrosis (CF) is poor. Forgetfulness has often been reported as a barrier to adherence by both CF patients and their parents. Many of the investigators patients also report being motivated by the results of their lung function studies (PFTs) to stay adherent to their medications. In this study, the investigators would like to see if providing medication reminders and allowing patients to measure their lung function at home will lead to better adherence. This will be a pilot study to determine the feasibility of providing such as a device to children with CF.

Current guidelines on the diagnoses and management of cystic fibrosis (CF) related diabetes recommend treatment for diabetes based on diagnostic criteria derived from adults with type 2 diabetes. Increasing evidence supports treating early glucose abnormalities in cystic fibrosis patients to target CF specific outcomes, including lung function and nutrition (BMI-Body Mass Index). However, the criteria and timing of when to start insulin therapy in the 'prediabetic' state are unclear. A more accurate characterization of blood sugar variability in youth with and without CF will help the investigators better interpret continuous glucose monitor (CGM) findings in patients with CF prediabetes and diabetes and more accurately identify those individuals at greatest risk for disease progression.

Colistin is a polymixin antibiotic used in the treatment of multidrug-resistant gram-negative infections. Given the limited use of colistin and previous challenges with laboratory assays to determine plasma concentrations, there is a lack of knowledge of the pharmacokinetic profile of colistin. The purpose of the investigators observational prospective pharmacokinetic cohort study is to examine the steady-state pharmacokinetic and pharmacodynamic properties of intravenous colistimethate sodium in cystic fibrosis and critically ill patients.

Cystic fibrosis (CF) is a multisystem disease characterized by viscid secretions in multiple organ systems. Lung infection and damage account for most of the disease burden. Acute changes in respiratory signs and symptoms termed Pulmonary Exacerbations require treatment with intravenous antibiotics and hospital admission. These episodes cause substantial disruption to people's lives and impact on lung function, quality of life and lifespan. Current treatment regimes require improvement but further study is needed to identify who might benefit from a different approach. This observational study aims to assess if multi-dimensional measurements taken during treatment correspond with later treatment response. This may allow us to personalise treatment more effectively in the future and to better understand how individuals respond to treatment.

The aim of this study is to assess whether the 1 minute Sit to Stand (STS) test can be used as a measure of submaximal endurance during a pulmonary exacerbation of Cystic Fibrosis (CF) for patients in the acute care setting. We hypothesize that if the STS test is a valid measure of submaximal cardiovascular endurance, it will moderately correlate with 6 minute walk distance (6MWD).

This study seeks to define the role of CD4+ and CD8+ T cell memory responses in the immunologic failure of patients with cystic fibrosis (CF) to clear infections. In a normal host, the immune system clears pathogens upon re-infection more swiftly and efficiently than during an initial infection, in great part due to the recall and effector functions of memory T cells. In CF, far less is understood regarding the response of T cell memory when hosts reencounter antigens, otherwise known as pulmonary exacerbations. Pulmonary exacerbations are pivotal events that lead to a decline in health status among CF patients, with many never recovering to baseline health. CF patients will be recruited from patients followed by the Adult CF Program at National Jewish Health. Following enrollment at the time of antibiotic initiation, blood will be collected at two different time points. The first samples will be collected within 24 hours of starting IV antibiotic therapy. The second blood specimen will be collected at the end of hospitalization, after a minimum of 5 days. At the time of each blood draw, complete blood counts, a sputum sample, and simple spirometry will be measured as part of the standard care of a CF exacerbation. Isolated PBMCs will be stained with antibodies to designate cell surface phenotype. They will then be sorted to identify the T cell population. These cells will be tested on their ability to clear pathogens. The relationship between cellular immune responses and clinical indicators of pulmonary status will be examined by fitting linear mixed models.

The long-term goal of this project is to examine the effects of cystic fibrosis (CF) and cystic fibrosis related diabetes (CFRD) on brain structure and function

To determine personal exposure to air pollution in children with cystic fibrosis; To determine airway macrophage uptake of inhaled particulate matter in cystic fibrosis children; To establish whether prostaglandin E2 affects particulate matter removal in airway.