View clinical trials related to Cystic Fibrosis.
Filter by: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.
Azithromycin is an antibiotic currently prescribed continuously in cystic fibrosis patients. It was shown that this treatment taken every day or every week for 12 months, can improve the respiratory state of patients. From the second year of treatment, it would appear there is more profit to continue such treatment. The main objective is to study the association between continuous use of azithromycin and lung function measured by Forced expiratory volume in one second (FEV1), after two years of treatment.
Children with Cystic fibrosis (CF) have to undergo several needle-related procedures during outpatient visits or hospital stay. It is common for children with CF to display distress and behavioural problems during invasive procedures. Children with chronic diseases have a lower threshold of pain compared to non-chronic patients. Effective management of needle distress in children with CF is critical. Although pharmacological and non pharmacological methods are used during needle -related procedures to lower painful stimuli nearly all patients with CF experience anxiety. Distraction has been shown to be an effective technique that directs children's attention away from noxious stimuli. The application of Virtual Reality (VR) in the medical field has been shown to be successful already 15 years ago. VR has found its use during the medication of burns and in patients undergoing cancer treatments whereas so far few studies have been published to assess its efficacy in reducing needle related pain and distress in children and none in children with CF. Objectives To assess the efficacy of VR in reducing pain and distress during venipuncture in children with CF compared to routine care. Project description Randomized controlled parallel trial with a 1 to 1 allocation ratio. The use of VR compared to standard care during venipuncture in children with CF (age 6-18 years) attending the outpatient clinic of the CF Centre of Florence will be compared over the period of 1 year. Patients randomly assigned to arm A will use VR during the procedure, those patients assigned to arm B will receive routine care. Anticipated output Determination of the efficacy of VR in lowering pain and distress during venipuncture.
In patients with cystic fibrosis (CF) the commonest cause of death is respiratory failure. Respiratory failure can have many causes. However, in patients with CF a major contributor is the impairment of the muscles required for breathing (respiratory muscles). Respiratory muscle impairment can result from poor nutrition. Lung function declines particularly during adolescence whilst body composition also changes at the same time. Thus, the investigators plan to study the relationship of nutrition and body composition to respiratory muscle strength and lung function in children and young people with CF aged between 12-18 years. The body mass index (BMI) is currently used in the clinical setting to measure nutritional status in CF. At King's College Hospital (KCH) there are portable devices to assess both respiratory muscle function and lung function. The research team will use a Bioelectrical Impedance Analysis (BIA) device to assess body composition, including BMI and lean body mass (LBM). The aim of the study is primarily to assess whether measurements of LBM impairment may better relate to poor lung function compared to BMI and secondly to examine whether lung and respiratory muscle function correlates with exercise tolerance.
This is a Phase 3, 2-part (Part A and Part B), open-label, multicenter study evaluating the pharmacokinetics (PK), safety, tolerability, and pharmacodynamics (PD) of multiple doses of lumacaftor/ivacaftor (LUM/IVA) in subjects 2 through 5 years of age (inclusive) with cystic fibrosis (CF), homozygous for F508del. Subjects who participate in Part A may participate in Part B, if they meet the eligibility criteria.
The main objective of register-SAFETIM is to assess the impact of the transition from pediatric to adult cystic fibrosis care center on changes in lung function and nutritional status of patients. This is a multicenter, observational, longitudinal, with analysis of the French national registry data of patients with cystic fibrosis. Our study will assess the clinical features of adolescent patients with cystic fibrosis during the transition from pediatric care to adult care. It will explore factors associated with the impact of the transition on the clinical course of adolescent patients (respiratory and nutritional functions).
Cystic fibrosis (CF), a life-shortening genetic disease, is marked by acute episodes during which symptoms of lung infection increase and lung function decreases. These pulmonary exacerbations are treated with varying antibiotics for varying time periods based on needs determined by individual patients, their families, and the health care providers. Cystic fibrosis pulmonary guidelines for the treatment of pulmonary exacerbation published by the Cystic Fibrosis Foundation (CFF) in 2009 provided recommendations for treatment and also identified key questions for which additional studies were needed. A strong desire among clinicians to reduce treatment durations (and reduce cost, inconvenience, and potential toxicities) is in conflict with belief that patients not responding robustly to treatment might benefit from extending treatment. This randomized, controlled, open-label study is designed to evaluate the efficacy and safety of differing durations of IV treatment, given in the hospital or at home for a pulmonary exacerbation in adult patients with CF.
Investigators will examine temporal and regional dynamic changes in the microbiome of Cystic Fibrosis patients to explore microbiome features that are associated with an inflammatory phenotype. Investigators hypothesize that temporal and spatial differences in lung microbiome are associated with host inflammatory responses. While chronic and polymicrobial airway colonization are commonly recognized in cystic fibrosis (CF), it is unclear what factors of the microbial environment lead to infection with pathogenic microorganism. This is a multi center, longitudinal cohort of adult Cystic Fibrosis subjects recruit4ed from NYU and Columbia to understand how changes in the airway microbiome may affect the host inflammatory responses in Cystic Fibrosis (CF). There will be three approaches to understanding inflammatory responses; 1) a longitudinal assessment of temporal changes in the microbiome over a 6-month period of clinical stability; 2) comparison of the regional differences in airway microbiome between lung segments with more versus less disease; 3) evaluation of functional aspects of the lung microbiome.
This study will investigate the link between glucose abnormalities and elements critical to muscle function including mass, composition and energy metabolism. the primary goal of the study is to determine whether Cystic Fibrosis (CF) disease is associated with muscle dysfunction, especially in the presence of glucose intolerance. This is a longitudinal cohort study of 3 main groups: CF subjects without Cystic Fibrosis-related diabetes (CFRD), healthy matched controls and CF subjects with newly diagnosed CFRD started on insulin therapy.
Mucus in the airways of patients with CF represents an area for bacteria proliferation, microbial infection and inflammation. Similar to the lung, the esophagus provides an environment for bacterial to grow. The overall goal of this proposal is to characterize the esophageal microbiota of children with CF that are treated or untreated with acid blockade medication and to measure its possible impact on respiratory disease to develop novel treatment strategies.