Study of Denufosol Tetrasodium in Patients With Cystic Fibrosis Lung Disease
Cystic FibrosisThe objectives of this study are to evaluate the safety, tolerability and preliminary efficacy of two dose levels of INS37217 (denufosol tetrasodium) Inhalation Solution in patients with cystic fibrosis (CF) lung disease.
A Study in Pediatric Patients With Cystic Fibrosis Lung Disease
Cystic FibrosisThe purpose of this study is to investigate the safety and effectiveness of two dose strengths of study drug compared to placebo in pediatric patients with cystic fibrosis (CF).
Comparison of Inhaled Mannitol and rhDNase in Children With Cystic Fibrosis
Cystic FibrosisThe purpose of this study is to determine the medium term efficacy and safety profile of inhaled mannitol, on its own and also as an additional therapy to rhDNase (pulmozyme). In particular, we will assess the impact on: lung function; airway inflammation; sputum microbiology; exacerbations; quality of life; adverse events; exercise tolerance; total costs of hospital and community care; and cost-effectiveness.
Single Dose of pGM169/GL67A in CF Patients
Cystic FibrosisThe study objectives are to assess safety, tolerability and gene expression after a single dose of non-viral CFTR gene therapy (pGM169/GL67A) administered to the nose and lungs of patients with cystic fibrosis.
Study Investigating a Delayed-Release Pancrelipase in Patients With Pancreatic Exocrine Insufficiency...
Cystic FibrosisPancreatic Exocrine InsufficiencyThis study will assess the safety and tolerability of pancrelipase delayed release capsules in subjects up to 6 years of age with Pancreatic Exocrine Insufficiency (PEI) due to Cystic Fibrosis.
Tolerability of Hypertonic Saline in Infants With Cystic Fibrosis
Cystic FibrosisThe objective of the study is to determine whether or not inhalation of hypertonic saline will be tolerated by infants with cystic fibrosis and the effect of inhalation on their lung function.
Insulin Glargine Vs Standard Insulin Therapy
Cystic Fibrosis Related DiabetesThis Study is designed to determine whether treatment of CFRD with glargine insulin will improve hemoglobin A1c, weight and muscle mass compared to the traditional regimen of bedtime NPH insulin.
Miglustat in Cystic Fibrosis
Cystic FibrosisSingle Center, Double-Blind, Randomized, Placebo-Controlled, Two-Period/Two-Treatment Crossover Study Investigating the Effect of Miglustat on the Nasal Potential Difference in Patients With Cystic Fibrosis Homozygous for the F508del Mutation
Phase I Study of the Third Generation Adenovirus H5.001CBCFTR in Patients With Cystic Fibrosis
Cystic FibrosisOBJECTIVES: I. Assess the safety and feasibility of gene transfer with the third generation adenovirus H5.001CBCFTR in patients with cystic fibrosis.
Study of Tauroursodeoxycholic Acid for Hepatobiliary Disease in Cystic Fibrosis
Cystic FibrosisOBJECTIVES: I. Determine the optimum dose of tauroursodeoxycholic acid (TUDCA) required to achieve maximal bioavailability for patients with cystic fibrosis-associated liver disease. II. Compare optimized doses of TUDCA with ursodiol (ursodeoxycholic acid; UDCA) for effects on biliary bile acid composition and metabolism, serum biochemistries, fat absorption, and fat-soluble vitamin status in these patients.