View clinical trials related to Cystic Fibrosis.
Filter by:Non-invasive methods of the quantification of fibrosis may help to assess the development of fibrosis at a specific moment of the evolution of the disease, in the order to decide the initiation of treatment with ursodeoxycholic acid which may slow the progression to hepatic cirrhosis: - FibroTest, - The ultrasound impulse elastography, Fibroscan (FS) - Ultrasound Elastography by ShearWave (SWE) - Magnetic resonance elastography (MRE). The purpose of this study is to assess the contribution of these methods in the diagnosis of hepatic fibrosis during the evolution of the cystic fibrosis.
The purpose of this study is to determine the efficacy of a non invasive method of detecting the pulmonary disease in order to initiate treatment against cystic fibrosis as soon as possible. Moreover this screening procedure permits to note the improvement following the treatment and to choose the optimal treatment in term of efficacy.
The purpose of this study is to demonstrate that Miglustat restores the function of the cystic fibrosis transmembrane conductance regulator (CFTR) in adult patients with cystic fibrosis homozygous for the F508del mutation.
Clinical studies of lumacaftor + ivacaftor (combo therapy) produced better FEV1 (forced expiratory volume in 1 second) improvements than ivacaftor alone, without further improvement in sweat chloride results. To help understand why sweat chloride was unresponsive, the investigators will use a newly developed sweat secretion test that provides accurate, in vivo readout of CFTR (cystic fibrosis transmembrane conductance regulator) function in the sweat gland secretory coil. The investigators devised a protocol to determine if short courses of ivacaftor (3.5 days) will produce significant increases in WT (Wild-Type, i.e. normal) CFTR open probability by measuring CFTR-dependent sweating (C-sweat) in subjects with WT CFTR.
To use the sputum colour chart as a predictor for cystic fibrosis exaerbations
YKL-40 is proposed as a biomarker of various inflammatory disease diabetes and lung disease including cystic fibrosis. In those cross-sectional studies, a unique value of YKL-40 is used to correlate with clinical, physiological, or biological determinants of disease severity (like FEV1 for example in lung disease). There is only one longitudinal study that showed a correlation between circulating levels of YKL-40 and the decline of lung function in smokers sampled from the general population. In order to better understand the potential role of YKL-40 in CF pathophysiology, and to determine its potential role as a biomarker of disease evolution, it is essential to proceed with further clinical evaluation. The investigators propose to perform an observational prospective cohort study to determine if variation of YKL-40 concentration over 24 months correlates with the clinical evolution of the patients.
This trial aims to analyze whether the inhalation of hypertonic saline combined with a positive expiratory pressure (PEP) device increases the amount of sputum expectorated during the chest physiotherapy session ( inhalation + bronchial drainage).
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.
The lungs of most patients with cystic fibrosis (CF) become chronically infected with bacteria called Pseudomonas aeruginosa during childhood. This infection is now known to consist of free-living bacteria (known as "planktonic bacteria") and bacteria in colonies on body surfaces known as "biofilms". The bacteria in biofilms are more resistant and tolerant to antibiotics. Current CF treatment of exacerbations aims to eradicate or control pseudomonal infection using aggressive antibiotic regimes. Despite this treatment many patients develop chronic infection which is never cleared. Chronic infection causes damage to the lungs. Patients colonised with Pseudomonas are more unwell and die at a younger age. Our laboratory has established that low dose nitric oxide (NO) can disrupt pseudomonal biofilms in the laboratory. This pilot study will discover whether non-toxic levels of NO administered to participants during an episode of acute infection (exacerbation) will disrupt bacteria from biofilms and increase the effectiveness of antibiotic therapy. This protocol describes a participant-blind randomised controlled pilot study of treatment with nitric oxide gas during an acute infective exacerbation (also known simply as an "acute exacerbation"). Patients with CF aged 12 or above will be asked to take part. They will be randomised to receive 7 days either of inhaled nitric oxide gas or placebo alongside standard therapy during an exacerbation. Sputum samples will be obtained before, during and after the treatment period for microbiological analysis. The primary endpoint will be the microbiological effect on bacterial biofilms before and after NO adjunctive therapy. Secondary microbiological endpoints will include the between group differences in pseudomonal colony forming units (CFU"s), biofilm NO levels and detailed characterisation of biofilms before and after treatment. Secondary clinical endpoints will include lung function and well-established indicators quality of life. The aim of this randomised pilot study is as proof of concept and to guide the design of a large multi-centre trial to definitively evaluate the effectiveness of NO or NO donors as adjunctive therapy in CF.
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.