View clinical trials related to Cystic Fibrosis.
Filter by:The purpose of this study is to find out whether the High Frequency Digit Triplet test can be used to screen patients with cystic fibrosis for hearing loss in conditions of health and pulmonary exacerbation. It is also designed to find out the youngest age at which a child can perform the test, the prevalence of hearing loss in a CF population and the prevalence of genetic mutations known to be associated with hearing loss in the same population.
The purpose of the study was to evaluate lung clearance index (LCI) by a standardized procedure in a well characterized study setting and to assess feasibility of LCI as a more sensitive method than forced expiratory volume at 1 second (FEV1) to measure effectiveness of antibiotic therapy in patients with CF aged 6 years and older with mild to moderate lung disease.
Cystic fibrosis related diabetes (CFRD) is a common co-morbidity in patients with CF. The underlying pathophysiology of cystic fibrosis related diabetes (CFRD) is still a matter of investigation. In addition to localized tissue damage developing similar to that of the exocrine pancreas, other mechanisms may be involved. We have shown that a potential contributing factor to the patho-physiology of CFRD may be an abnormal gut derived hormonal profile, specifically of lower incretin hormone responses, prior to development of CFRD. We propose that an altered incretin response, probably due to impaired interaction of nutrients with the gut mucosa due to thickened secretions, may play a role in the development of the disease. Specifically, low GIP and GLP-1, may explain the poor β-cell function observed in these patients prior to CFRD appearance. These incretins have known trophic effects on β-cells, and thus their lower levels may contribute to the development of quantitative as well as qualitative defects in β-cell function and thus may lead to the development of CFRD. Thus, increasing levels of these incretins using a DPP-IV inhibitor may improve glucose metabolism and delay/prevent the development of CFRD. We hypothesize that Saxagliptin will increase the oDI compared to placebo and will thus provide relative protection from diabetes development and in addition we expect that Saxagliptin will lead to overall increased insulin concentrations and thus shift the metabolic milieu to a more anabolic state. This will manifest as weight gain and reduction in inflammation.
Cystic Fibrosis (CF) is a life limiting illness. Median predicted UK survival is 41.4 years (UK CF Registry 2011). The commonest cause of death is respiratory failure. Non invasive ventilation (NIV) is a system which delivers a preset pressure to supplement the size and depth of each breath. It is introduced in CF to manage established respiratory failure. A nose or a mask which covers both the nose and mouth allows flexible ventilation, is used just at night, or for part of the day in addition or for 24 hours as clinical status indicates. It is introduced within a normal ward environment and then continued longterm at home.Once respiratory failure is established longterm noninvasive ventilation is introduced throughout 24 hours and multidisciplinary assessment concludes that the timing is appropriate for the individual. This study aims to evaluate a potential development of current practice: the use of non invasive ventilation during hospital admission only to enhance recovery from an acute exacerbation which has caused respiratory failure in those individuals where long term non invasive ventilation is not yet indicated. A mixed methods design will allow description of the experience of noninvasive ventilation during a semistructured interview to add to understanding of the results from an experiment designed to measure the differences between noninvasive ventilation and standard care. Aim: To compare the clinical efficacy with the patient experience of NIV on recovery from an acute respiratory exacerbation complicated by respiratory failure in adult Cystic Fibrosis.
The purpose of this study is the psychometric validation of a self-administered dyspnea questionnaire, usable in clinical practice in order to assess dyspnea and its impact on patients with chronic respiratory diseases.
This Phase 1 study in F508del-CFTR homozygous CF patients is being conducted to assess the pharmacokinetics and absorption dynamics of N91115 compared with healthy subjects in order to identify an initial starting dose for Phase 2 trials.
With better medical care, patients with cystic fibrosis (CF), a life-threatening disease, are enjoying longer lives. As the CF life expectancy increases, conditions such as CF-related diabetes (CFRD) become more prevalent. Nutrition plays a major role in maintaining optimal health in cystic fibrosis (CF). This project is designed to investigate nutrition-related factors, such as diet and body composition, on outcomes in patients with CF. The data generated from this study will be used to inform future nutrition intervention studies in adults with CF and CFRD.
The objective of this study is to determine if tobramycin inhalation powder (TIP) can reduce the amount of Burkholderia Cepacia Complex (BCC) species - type of bacteria, in the sputum of cystic fibrosis patient.
The morbidity and mortality associated with Cystic Fibrosis (CF) are the result of chronic suppurative lung disease. The aggressive use of antibiotics is one of the mainstays of treatment in CF, however, the problems of multiple drug resistance and adverse reactions are major clinical issues. Cysteamine is a licensed drug used in the treatment of cystinosis. In vitro work suggests that cysteamine has properties of potential benefit in CF. Cysteamine is a potent mucolytic, it disrupts biofilms, it is antimicrobial, and synergises with other antibiotic agents. CF is characterised by malabsorption and it is not known whether cysteamine is absorbed in CF, furthermore it is not known if cysteamine enters the bronchial secretions. It is not possible to assume that the pharmacokinetics of cysteamine in patients with CF are the same as those reported for cystinosis. Objectives: to characterise the pharmacokinetic profile of cysteamine in people with CF, to ascertain whether cysteamine enters the bronchial secretions and the tolerability of cysteamine by patients with CF. Method: a single centre, single group open label investigation of the tolerability and pharmacokinetics of oral cysteamine (Cystagon) when administered to patients with Cystic Fibrosis at the dose licensed for use in cystinosis. Setting: adult CF clinic, Aberdeen Royal Infirmary. Target population: 12 patients aged ≥18years with CF associated lung disease who are clinically stable. Intervention: Oral cysteamine (Cystagon) will be increased from 450mg od to 450mg qds over three weeks, they will remain on 450mg qds for two weeks. Assessment: face to face health outcome assessments will be carried out for all participants at recruitment/baseline, 1, 2, 3, and 5 and 6 weeks. Serial blood cysteamine levels will be measured in the first 24 hours after the first dose. Sputum cysteamine will be quantified after two weeks of full dose cysteamine 450mg qds. Disease specific health status (CFQ-R) will be assessed at baseline and after two weeks of full dose. At each assessment, lung function (FEV1, FVC), adverse reactions and serious adverse events will be ascertained. Blood samples will be taken for measurement of haematological and biochemical parameters. Sputum samples at each assessment will be analysed for microbial load and spinnbarkeit.
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.