DENUFOSOL TETRASODIUM INHALATION SOLUTION IN PATIENTS WITH MILD CF LUNG DISEASE

这个子项目是众多研究子项目之一

This subproject is one of many research subprojects utilizing the resources provided by a Center grant funded by NIH/NCRR. The subproject and investigator (PI) may have received primary funding from another NIH source, and thus could be represented in other CRISP entries. The institution listed is for the Center, which is not necessarily the institution for the investigator. Cystic fibrosis (CF) is a recessive genetic disease, characterized by pulmonary and sinus disease, and gastrointestinal and reproductive tract dysfunction. The disease is caused by mutations in the cystic fibrosis transmembrane regulator (CFTR) gene, which encodes for an apical membrane epithelial protein that functions as a c-AMP-regulated chloride channel and a regulator of other channels. Defective CFTR results in abnormal ion transport and depleted airway surface liquid volume with reduced mucociliary clearance and a propensity for chronic infection of the respiratory tract with resulting inflammation, progressive airway damage and bronchiectasis. CF patients suffer from chronic repeated cycles of pulmonary bacterial colonization, pulmonary exacerbations and chronic lung function decline, which often lead to premature death. Denufosol tetrasodium (INS37217) inhalation solution (denufosol) is a selective P2Y2 receptor agonist under development by InspirePharmaceuticals, Inc. Denufosol may have the ability to restore or maintain mucociliary clearance in patients relatively early in the CF lung disease process. This multicenter, randomized, double-blind, placebo-controlled Phase 3 study will examine denufosol as an early intervention therapy to improve lung function relative to placebo in a relatively healthy CF patient population. Eligible patients will be randomized to receive either denufosol 60 mg or placebo three times daily for a 24-week treatment period.