View clinical trials related to Cystic Fibrosis.
Filter by:Vitamin D3 therapy was effective in decreasing the frequency of pulmonary exacerbations and preserving lung functions , thereby improving the disease severity even more in non CF than CF bronchiectasis patients
Adult patients with Cystic Fibrosis who are seen at the specialty CF clinic at University of Virginia will be given an option to utilize telemedicine instead of in-person visits for standard clinic visits. Health information from standard of care visits including FEV1, exacerbations, leading to oral or intravenous antibiotics, laboratory results, hospitalization records, and responses to health questionnaires will be recorded for research purposes. Data collected for the research study will be compared to baseline and previous years to determine if there are any deleterious effects for those who transition to telemedicine clinic visits.
Adults with CF regularly receive anti-infective therapy that can result in impaired hearing. Recent studies have identified that up to 50% of adults with CF have hearing loss with resulting significant impact on quality of life. At present, however, there is no national recommendation to screen for hearing loss within adult CF. Formal hearing tests are costly, require specialist staff and equipment and require further outpatient visits resulting in significant impact on health, social and financial resource. We propose to analyse over a 12 month period at two adult cystic fibrosis centres in London whether outpatient tablet based audiometry testing and web-based hearing apps are able to accurately screen for ototoxicity in adults with CF, and whether these methods of screening are acceptable. The results of this study will aim to streamline audiometry screening to improve detection rates, reduce cost of healthcare, enable equity in service delivery, and minimize disruption to the patient's social and work-life using smart technology.
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.
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.
Cystic fibrosis (CF) is the most common lethal inherited disease in Caucasian populations. To improve survival, it is essential to understand the development, progression and treatment of CF lung disease throughout early childhood. Therefore the overall objective is to prospectively assess the clinical utility of novel and non-invasive measuring methods, namely Multiple Breath Washout and functional lung MRI in the longitudinal clinical surveillance of patients with CF and compare the results to those of healthy controls.
Cystic Fibrosis (CF) is a disease that affects salt and water transport in multiple organs. Many CF patients suffer from abdominal pain and this could be due to intestinal inflammation. However, so far we do not know how many of the CF patients actually do have intestinal inflammation when looking at intestinal specimens. There is a proven connection between chronic inflammation and developing colorectal cancer and over the years more CF patients are developing colorectal cancer. Thus, it becomes increasingly important to look for the presence of intestinal inflammation in CF patients since early treatment may improve their symptoms and reduce the risk for colorectal cancer.
In this study, MRI of the lungs of healthy volunteers and participants with cystic fibrosis (stable and participants initiating CFTR modulator treatment) will be performed over a period of 6 months to determine if lung MRI is able detect structural and functional abnormalities/changes in early cystic fibrosis disease. During the 6 month period, 3 study visits will occur. 70 subjects aged 6 and older will participate in this study. Xenon MRI is a non-invasive imaging technique that does not involve x-rays or ionizing radiation. Rather, this imaging method utilizes the same hardware and software principles that are used for conventional proton MRI of patients in a hospital.
Cystic fibrosis related diabetes (CFRD) is a major factor of morbidity and mortality at all disease stages. Insulin deficiency has serious clinical consequences by increasing malnutrition, since protein and lipid catabolism is accelerated in chronic infections. Traditionally, insulin is injected before a meal. Yet, in these patients with highly varied and often staggered nutritional intakes, insulin injection can result in an increased risk of postprandial hypoglycaemia, all the more so as CF patients exhibit decreased glucagon secretion. Recent progress in the development of new insulins mimicking the physiological secretion more closely has led to ultra-fast insulins (fast aspart), allowing for postprandial hyperglycaemia to be better controlled. In Type 1 diabetics treated with basal-bolus, faster-acting aspart insulin injected after a meal enabled metabolic control comparable to injection of aspart insulin prior to the meal. Fast apart insulin is of particular interest with regard to CFRD, wherein postprandial hyperglycaemia occurs early. In CFRD, these insulins are likewise advantageous in that they can be injected after the meal, thus permitting more flexibility in patients with highly varied diets. Moreover, the insulin dose can be adapted depending on dietary intake, thus preventing hypoglycaemia secondary to highly-varied carbohydrate intakes. Due to its flexibility, this insulin therapy is likely to be better accepted by patients with cystic fibrosis.
Cystic fibrosis is a genetic disorder caused by mutations in the Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) gene, leading to pulmonary infections, sinus disease, pancreatic insufficiency, hepatobiliary disease and male infertility, with respiratory failure being the primary cause of death. Cystic Fibrosis Related Diabetes (CFRD) in one of the most common complication of cystic fibrosis (CF) and it's associated with a worse respiratory and nutritional state, with a negative impact on life expectancy. It differs from type 1 diabetes and type 2 diabetes for particular characteristics making this disease a separated clinical entity. To date, there is a lack of evidence on many aspects concerning this disease: - the pathophysiology of the disease: decreased insulin secretion has historically been seen has the major trigger for CFRD, but data about this mechanism are scarce and conflicting. Moreover, the role of insulin-resistance seems to be not consistent, but pulmonary exacerbations are very common and, in this setting, insulin sensitivity can worsen significantly. - the relationship between its development and particular genetic settings: certain CFTR genotypes are known to be most related to the risk of diabetes, and only few susceptibility genes for type 2 diabetes have been evaluated as potential predisposing factors for CFRD. - the relationship between the therapeutic optimization and its impact on metabolic status and lung function: CFRD is known to be associated with worse clinical outcomes, reflected in more frequent clinical exacerbations, greater reduction in lung function, poorer nutritional status and decreased survival. It has also been demonstrated that insulin therapy can improve pulmonary function, increase body weight and reduce lung exacerbations. However, no study on the clinical impact of the optimization of insulin therapy on pulmonary outcomes and life expectancy are available in this population. - finally, no data about potential predisposing pre-transplant risk factors for development of post-transplant DM are available For this reason, the investigators have structured a study with the aim to: - characterize the pathophysiological process leading to CFRD, with assessment of the relative contribution of the insulin resistance and the β-cellular secretion impairment - define the prevalence of CFRD in relation to the mutations of the CFTR gene and to the presence of candidate genes for the development of type 2 diabetes - perform a proteomic analysis to identify potential proteomic biomarkers among CFRD patients - evaluate the body composition, muscle performance and respiratory outcomes in patients on insulin therapy, before and after therapeutic optimization, in a follow-up period of 24 months. - identify eventual predisposing factors for the development of post-transplant diabetes in subjects without pre-transplant CFRD.