View clinical trials related to Cystic Fibrosis.
Filter by:The objectives of the study are to demonstrate that Tobrineb®/Actitob®/Bramitob® is non-inferior to TOBI® in the primary efficacy variable, forced expiratory volume in one second (FEV1) percent of predicted normal, and to compare the safety in participants with cystic fibrosis and chronic infection of the lungs with Pseudomonas aeruginosa.
Multicenter, explorative, phase IIIb, open-label study to assess the efficacy and safety of Ultrase® MT12, in the control of steatorrhea and clinical signs and symptoms of malabsorption in CF children with pancreatic insufficiency (PI). This study is sponsored by Aptalis Pharma (formerly Axcan).
The primary objective of the study was to evaluate the safety and tolerability of VX-809 in participants with cystic fibrosis (CF) who are homozygous for the F508del mutation on the CF transmembrane conductance regulator (CFTR) gene.
Elevated fecal calprotectin is a biomarker for intestinal inflammation in cystic fibrosis
This study will be performed at the University of Washington. Forty subjects with Cystic Fibrosis will be enrolled, with 20 randomized into the usual care arm and 20 randomized into the intervention arm. All subjects will be enrolled for 6 months and have 3 study visits. One at baseline, midpoint, and final. The intervention arm subjects may have more study visits depending on their respiratory symptoms. The intervention arm subjects will perform home spirometry twice a week with a PiKo-6 hand held spirometer. They will also have a home-based telemonitoring system connected to their phone line. They will be prompted twice a week to answer questions regarding their health via the telemonitor. Subjects who are not able to utilize the home-based telemonitoring system will answer the questions regarding their health via the internet from their home computer. If the subject's spirometry falls by greater than 10% from baseline or the CF respiratory symptoms change in 3 or more of the 8 respiratory symptoms captured by the telemonitor questionnaire, the subject will be called by the research staff and clinically evaluated by the study PIs within three days. The usual care subjects will continue with their routine care at the University of Washington CF Clinic.
The purpose of this research is to determine how a person's lungs will uptake [18F]fluorodeoxyglucose (FDG), as measured with positron emission tomography (PET) scanning in young cystic fibrosis (CF) patients.
Serine proteases belonging to the elastase family are mainly responsible for lung tissue destruction as observed during cystic fibrosis. But anti-inflammatory therapies based on systemic or aerosolized protease-inhibitors administration, have not given the expected results until now. One reason would be the impaired access of therapeutic inhibitors to their molecular targets. It was recently shown that neutrophils actively secrete neutrophil extracellular traps (NETs) made of DNA that binds cationic proteases among other molecules. NETs together with DNA passively released from dead neutrophils contribute to the viscosity of CF expectorations which explains that rhDNase treatment fluidifies expectorations and improves the patient status. Preliminary experiments in our laboratory have shown that DNA degradation was associated with a significant increase of proteolytic activity in the sputum soluble fraction. However the efficacy of exogenous inhibitors is also improved in these conditions. Using the specific substrates and methodologies that we developed previously to measure cell-surface associated proteolytic activities, we will study the effects of DNase on the activity of individual proteases, their biodistribution in sputum and their regulation by potential therapeutic inhibitors. Enzymatic, immunochemical and microscopic (confocal and scanning) techniques will first be used for ex vivo studies on sputa freshly collected at the adult and paediatric CRCM in Tours, then on sputa from patients before and after administration of aerosolized rhDNase. We hypothesize that a better understanding of the biodistribution of neutrophil serine proteases and especially their binding to DNA will help designing new therapeutic strategies that facilitate inhibitor access to their protease targets.
Patients with cystic fibrosis (CF) suffer from chronic infections of the lower respiratory tract that can be caused by one or multiple bacteria, including Pseudomonas aeruginosa, which has been particularly problematic to eradicate and been implicated as the major cause of morbidity and mortality in CF patients. Aerosol delivery of antibiotics directly to the lung increases the local concentrations of antibiotic at the site of infection resulting in improved antimicrobial effects compared to systemic administration. Bacterial resistance to current aerosol antibiotic treatments indicate a need for improved therapies to treat CF patients with pulmonary infections caused by multi-drug resistant Pseudomonas aeruginosa and other bacteria. High concentrations of MP-376 delivered directly to the lung are projected to have antimicrobial effects on even the most resistant organisms. MP-376 is a novel formulation of the fluoroquinolone levofloxacin that has been optimized for aerosol delivery using the PARI electronic eFlow® nebulizer. Preclinical and early clinical studies in adults show that aerosol doses of MP-376 appear to be safe and well tolerated, and exert an antimicrobial effect when administered once or twice daily. High concentrations of levofloxacin in the lung delivered using MP-376 are expected to be active against CF pathogens such as P. aeruginosa and S. aureus, including those resistant to aminoglycosides (such as TOBI®) and other inhaled antimicrobial agents. Inhaled MP-376 can be delivered rapidly and efficiently using the PARI eFlow® nebulizer system. This Phase 1 study is being performed to obtain safety, tolerability and PK data in children ages 6-16 in order to determine if MP-376 is safe, prior to enrolling children of these ages in the planned pivotal Phase 3 studies.
Repeated measurements of lung water by single transpulmonary thermodilution during the first postoperative days following lung transplantation
The purpose of this study is to compare conventional breast imaging and diagnostic work-up (2 dimensional imaging) to digital breast tomosynthesis (3 dimensional imaging) in the appearance of non-calcified breast masses. It is thought that non-calcified breast masses will be better visualized with the new 3D technology.