Study of VX-809 Alone and in Combination With VX-770 in Cystic Fibrosis (CF) Patients Homozygous...
Cystic FibrosisThe purpose of this study is to evaluate of the safety, efficacy, pharmacokinetics (PK) and pharmacodynamic (PD) effects of lumacaftor (VX-809) alone and when coadministered with ivacaftor (VX-770) in participants with cystic fibrosis, homozygous or heterozygous for the F508del-CFTR mutation.
A Safety and Tolerability Study of Denufosol in 2-4 Year Olds
Cystic FibrosisThe purpose of this study is to evaluate the safety and tolerability of denufosol 60 mg TID in pediatric CF patients 2 to 4 years of age
Phase I Pilot Study of Ad5-CB-CFTR, an Adenovirus Vector Containing the Cystic Fibrosis Transmembrane...
Cystic FibrosisOBJECTIVES: I. Assess the safety and efficacy of gene transfer into the nasal epithelium using Ad5-CB-CFTR, an E1-deleted adenovirus vector containing the cystic fibrosis transmembrane conductance regulator gene, in patients with cystic fibrosis (CF). II. Determine whether ion transport abnormalities in CF airway cells can be corrected.
Study of Pancreatic Enzyme Product in Pediatric Participants With Cystic Fibrosis and Exocrine Pancreatic...
Cystic FibrosisExocrine Pancreatic InsufficiencyThis is an open-label study to evaluate the efficacy and safety of Aptalis' (formerly Eurand) pancreatic enzyme product (PEP) microtabs in pediatric participants under age 7 with cystic fibrosis (CF) and exocrine pancreatic insufficiency (EPI).
Study of VX-770 and Rifampin in Healthy Male Subjects
Cystic FibrosisThe objectives of this study are to evaluate the effects of multiple doses of rifampin on the single-dose pharmacokinetics of VX 770.
A Study of Tobramycin Inhalation Powder From a Modified Manufacturing Process Versus Placebo
Cystic FibrosisThis study is designed to show how well tobramycin inhalation powder works and how safe it is when produced by a modified manufacturing process
Tobramycin Administered by eFlow Rapid Nebulizer: Pharmacokinetic Study
Cystic FibrosisThis study assesses the aerosol delivery characteristics (measured by nebulization time, serum and sputum tobramycin pharmacokinetic parameters) and safety of tobramycin administered for inhalation by PARI eFlow rapid electronic nebulizer (no compressor) vs. PARI LC PLUS Jet Nebulizer (with compressor) in subjects with cystic fibrosis.
Phase II Study of the Safety and Efficacy of Inhaled Alpha-1 Antitrypsin (AAT ) in Cystic Fibrosis...
Cystic FibrosisCystic Fibrosis (CF) is an inherited disorder in which mucus-secreting glands in the lungs produce considerable quantity of thick, sticky secretions that clog the airways, promote bacterial growth and lead to chronic obstruction, inflammation and destruction of the airways. The purpose of this study is to collect data about the resolution of the chronic inflammatory state in addition to assure the safety of the therapy in CF patients.
Safety/Tolerability Study of Arikayce™ in Cystic Fibrosis Patients With Chronic Infection Due to...
Cystic FibrosisThis is a study to determine the safety and tolerability of 28 days of daily dosing of 560 mg of Arikayce™ versus placebo and daily dosing of 70 mg and 140 mg of Arikayce™ versus placebo in patients who have Cystic fibrosis (CF) and chronic infection due to pseudomonas aeruginosa.
Long Term Administration of Inhaled Mannitol in Cystic Fibrosis
Cystic FibrosisThe purpose of this study is to examine the efficacy and safety of 26 weeks treatment with inhaled mannitol in subjects with cystic fibrosis. Previous studies have demonstrated improvements in lung function, mucociliary clearance, changes in physical properties of mucus, 24 hour sputum weight and quality of life. The results of this study are to further investigate and confirm these findings in addition to examine the effect on antibiotic use and chest infections. It is hypothesised that inhaled mannitol will have beneficial effects compared to a control treatment. An open label phase of 26 weeks duration will follow the blinded 26 week phase. During the open label phase all subjects will receive active treatment.