Research

Inhaled Denufosol for Treatment of Cystic Fibrosis

Study of Denufosol Tetrasodium Inhalation Solution in Patients with Cystic Fibrosis Lung Disease

Protocol Description

This aim of this Phase III, multi-center, international study is to confirm the effectiveness and test the safety of the inhaled study drug, denufosol tetrasodium. This study drug uses a new approach to treating cystic fibrosis that bypasses the basic defect in the lung’s cells. It is possible that denufosol, used early in treatment of the disease, may help patients clear mucus more easily from their lungs, which may in turn help preserve lung function and decrease the cycle of repeated lung infections that leads to decreased lung function.

Eligibility Criteria

Males and females, ages 5 and up, who have cystic fibrosis and meet other eligibility criteria, may be eligible to participate.
Males: Age 5 and up
Females: Age 5 and up

Requirements
Following a screening procedure, participants will be randomly assigned to take either denufosol or a placebo, taken as a 15-minute aerosol treatment three times a day for up to 6 months. Additionally, participants will visit the Pediatric Clinical and Translational Research Center for periodic checkups and pulmonary function tests, and patients will be required to keep a daily symptoms diary.
Visits: Up to 7
Duration: 6 months

Status: Open for Enrollment

Source of Support
Inspire Pharmaceuticals, Inc.

Additional Resources
Study description at National Institutes of Health