View clinical trials related to Cystic Fibrosis.
Filter by:The purpose of this study is to evaluate the efficacy and safety of ivacaftor in subjects with cystic fibrosis (CF) who have a non-G551D cystic fibrosis transmembrane regulator (CFTR) gating mutation (any one of the following CFTR mutations: G178R, G551S, S549N, S549R, G970R, G1244E, S1251N, S1255P, or G1349D).
The purpose of this study is to evaluate the efficacy and safety of ivacaftor in subjects with cystic fibrosis (CF) who have the R117H-CFTR mutation.
The study will include 10 adult patients with cystic fibrosis. The aim of the study is to evaluate whether Tobramycin 300 mg aerosol once-a-day for 28 days can reduce the bacterial load in the airways. Secondary end-point is the evaluation of variation in bacterial susceptibility during and at the end of study period.
Cystic fibrosis (CF) is characterized by airway inflammation and infection leading to progressive destruction of lungs. One of the most important abnormalities in CF is an abnormal processing of the mutated CFTR protein through the endoplasmic reticulum that causes abnormal location or even absence of the protein at the apical plasma membrane of airway epithelial cells. This abnormality results in a marked dehydration of the airway surface fluid, decreased mucus transport and airway obstruction. Nevertheless, the events that occur very early during the progression of the disease at the airway level in infants are not known. At cellular level, it has also been reported that the CFTR expression and localization could be related to the differentiation state of the airway epithelium. Furthermore, it has been reported that gap junctions could be involved in dysregulate inflammation process. In CF infants, many answers are still lacking. For a better understanding of the early stages of cystic fibrosis, it is of major interest to study respiratory epithelial cells obtained as early as possible. In 15 CF infants and 15 control infants, a nasal brushing will be performed by means of a soft sterile cytology brush. Samples will be used for cytological and functional studies: ciliary beating frequency, cAMP-dependent chloride efflux, potassium efflux, tight and gap junctions functionalities. These studies will be done in basal conditions and will be repeated after activation of the nasal epithelial cells by the bacteria, such as Staphylococcus aureus, which can be found very early in the course of CF disease.
There is ample evidence that children treated for a brain tumour (BT) often develop deficits in social and emotional functioning. The investigators wish to examine the cause of these deficits, i.e. the underlying neuropsychological deficit(s). The aim is to study impairment and developmental delay in social cognition (and related cognitive functions) caused by brain damage in patients treated for a BT in childhood as compared to a reference group of chronically ill children. If we can identify the specific deficits these patients experience, neuropsychological treatment and guidance can be developed to give patients the most optimal chances to live as normal as possible, to improve their quality of life (QoL) and to prevent them from developing depression and anxiety. Eventually, an intervention programme could be developed based on our results, to improve social, vocational and emotional QoL.
The prevalence of methicillin resistant Staphylococcus aureus (MRSA) respiratory infection in Cystic Fibrosis (CF) has increased dramatically over the last decade. Evidence suggests that persistent infection with MRSA may result in an increased rate of decline in Forced Expiratory Volume (FEV)1 and shortened survival. Currently there are no conclusive studies demonstrating an effective aggressive treatment protocol for persistent MRSA respiratory infection in CF. Data demonstrating an effective and safe method of clearing persistent MRSA infection are needed. The purpose of this study is to evaluate the safety and efficacy of a 28-day course of vancomycin for inhalation, 250 mg twice a day, (in combination with oral antibiotics) in eliminating MRSA from the respiratory tract of individuals with CF and persistent MRSA infection. Subjects will be assigned in a 1:1 ratio to either vancomycin for inhalation (250 mg twice a day) or taste matched placebo and will be followed for 3 additional months. In addition, both groups will receive oral rifampin, a second oral antibiotic (TMP-SMX or doxycycline, protocol determined), mupirocin intranasal cream and chlorhexidine body washes. Forty patients with persistent respiratory tract MRSA infection will be enrolled in this trial.
This is an open, multicenter, prospective, randomised and cross over study, comparing in patients with cystic fibrosis aged > 6 years, 2 periods of 6 weeks of oxygen therapy or room air, separated by a wash out period of 2 to 6 weeks.
Aspergillus infection is an infectious complication which frequently occurs in cystic fibrosis. The efficacy of azole therapy in patients with cystic fibrosis with persistent positive sputums for Aspergillus is still unknown. Furthermore, the efficacy of itraconazole and voriconazole in this indication has never been evaluated in a large prospective controlled clinical trial, even though many teams already use it. The ATCF study aims to assess in patients with cystic fibrosis with persistent Aspergillus positive cultures the efficacy of itraconazole and voriconazole on the negativisation of the sputum cultures for Aspergillus.
Title: Duplication in Chitotriosidase (CHIT1) Gene and the Risk for Aspergillus Lung Disease in CF Patients. Aim: To evaluate the link between CHIT1 duplication in CF patients and the predisposition to ABPA or persistent Aspergillus infection. Patients: 40 CF patients. Design: Observational, single visit. Methods: All patients will be assessed for pulmonary function tests (PFT), sputum cultures, and blood tests for: CHIT1 duplication, immunoglobulin E (IgE) and Eosinophils levels. Part of the patients will be assessed for RAST, skin prick test. Primary outcome measure is the difference in CHIT1 genotyping between the groups.
Chronic renal failure is a serious complication of lung transplantation especially in patients with cystic fibrosis. Their medical history prior to the Lung Transplantation has already exposed to kidney damage. Post-lung transplantation, these patients are subjected to renal toxicity anticalcineurins they receive large doses. The measurement of renal function of patients by formula to estimate GFR in routine use is unreliable and other markers seem indispensable. The purpose of this study is to evaluate two markers, PIIINP (Procollagen III aminoterminal peptide N), whose urinary levels was correlated to the intensity of fibrosis in different types of kidney disease.