View clinical trials related to Cystic Fibrosis.
Filter by:To determine if transient elastography (TE), when combined with ultrasound (US) pattern characterization can improve the prediction of progression to a nodular pattern on US. To confirm the feasibility of obtaining TE measurements in children with Cystic Fibrosis (CF) To prospectively assess whether TE data are associated with conventional laboratory markers of hepatic fibrosis To determine the variability of TE measurements taken at different sites in the same patient
This study investigates the use of cysteamine in the treatment of adults with Cystic Fibrosis who are experiencing an exacerbation of CF-associated lung disease. There are six different potential dosing regimens, including one that is placebo.
Cystic fibrosis (CF) is the most common fatal inherited condition in Caucasians, causing recurrent chest infections and premature death due to lung failure. When patients develop chest infections their symptoms usually slowly worsen over the course of several days to weeks. Due to this gradual onset, patients often seek medical attention several days or weeks after symptoms start to worsen. The Investigators believe that if they were able to monitor patients more closely they could diagnose and treat chest infections earlier and consequently improve health outcomes. The HOMECF study aims to investigate whether home monitoring is beneficial for adults with CF. 100 subjects will be randomly allocated, 50 to receive home monitoring and 50 to receive routine clinical care for 12 months. Subjects receiving home monitoring will measure their lung function and symptoms twice weekly and this data will be transmitted to the medical team by means of a modified mobile phone. the Investigators hypothesize that home monitoring will allow them to diagnose chest infections at an earlier stage and reduce hospital inpatient days. They will also assess the subjects' experience of receiving home monitoring, the impact on body weight and lung function and and conduct a full health economic analysis to assess value for money. They will also ask subjects to collect a urine sample once weekly to allow us to measure urinary levels of inflammatory markers. Subjects will be recruited at the West Midlands Adult CF Centre in Birmingham. The research team are well placed to carry out the study because it is a large regional adult CF centre with an excellent record of conducting clinical research.
The objective of study is to prospectively determine if CPET with blood gas analysis should have a prognostic value in CF. The study plans to include 300 cystic fibrosis patients. Inclusion criteria will be: age >15 years, cystic fibrosis confirmed by chloride sweat test or genetic analysis, clinical and functional stability in the 2 month before CPET. Patients will perform a maximal exercise test on a cycloergometer during the inclusion visit, with pulmonary function testing and a six-minute walk test. The study will also include a visit every 6 months with: body mass index calculation, pulmonary function testing with DLCO (diffusing lung capacity for carbon monoxide), a six minute walk test, and antibacteriological study of sputum. The results of this study could help identify earlier the patients for referral to a lung transplantation centre, by using the usual criteria and the CPET abnormalities.
The aim of our study is to assess miRNAs expression profiles in the circuling blood of patients with cystic fibrosis and highlight "signatures" that could reflect the pulmonary status of patients
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
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.
The objective of this study is to investigate the safety, tolerability, and pharmacokinetics of BI 443651 in male and female healthy volunteers and subjects with Cystic Fibrosis (CF).
This study will evaluate the efficacy and safety of CTP-656 in patients with cystic fibrosis (CF) who have a cystic fibrosis transmembrane conductance regulator (CFTR) gating mutation.
The purpose of this study is to determine which biological marker, or association of biological markers, best predict clinical response of cystic fibrosis patients to CFTR modulators.