DENUFOSOL TETRASODIUM INHALATION SOLUTION IN PATIENTS WITH CF LUNG DISEASE

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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 characterized by chronic airways blockage and infection. In CF, the transport of water, sodium ions, and chloride ions is abnormal. This results in thickened airway secretions that obstruct airways and make patients more likely to get chronic bacterial infections. Preliminary studies suggest that therapy targeted to this abnormality in ion transport may be beneficial. INS37217 is a compound that may improve transport of chloride and water out of airway cells, therefore hydrating secretions and making them easier to cough out. In addition, INS37217 makes the cilia beat more frequently, which may improve the body's ability to clear secretions out of the lung. This study is a multi-center, double-blind, randomized, placebo-controlled, parallel-group trial comparing the safety and efficacy of two dose levels of INS37217 Inhalation Solution to placebo administered over 28 days via the PARI LC STAR¿ nebulizer in subjects with mild to moderate cystic fibrosis lung disease. Seventy-two subjects will be enrolled with approximately 24 subjects in each of three treatment groups Subjects will be randomly assigned in a 1:1:1 ratio to one of three treatment arms delivered in 4 mL of 5 mg/mL (20 mg), 15 mg/mL (60 mg) INS37217 or 4 mL of placebo (0.9% w/v solution of sterile sodium chloride) for the duration of the treatment period. Randomization will be stratified on the basis of screening lung function (FEV1 75% of predicted) and chronic TOBI¿ use (yes vs. no).