Denufosol Inhalation Solution for People with CF and Mild Lung Disease (TIGER 1)
Restore Airway Surface Liquid
Number of Participants Being Recruited:
Single / Multi-Center:
STUDY BACKGROUND INFORMATION:
The purpose of this trial was to evaluate the safety and effectiveness of inhaled Denufosol designed to enhance the hydration and clearance of mucus in the lungs of CF patients. In people with CF, mucus builds up and clogs the lungs making breathing very difficult. The thick mucus also causes bacteria to get stuck in the airways, causing inflammation and infections that lead to lung damage. The trial compared one dose strength of the drug, denufosol, to a placebo in patients with mild CF lung disease. The drug was designed to enhance the lung's natural mucosal hydration and mucociliary clearance.
5 Years - 999 Years
>= 75 Percent Predicted
P. aeruginosa status:
B. cepacia status:
Other Primary Eligibility Requirements:
Note: Detailed eligibility criteria information may be available on clinicaltrials.gov. If a specific
trial listing for this trial is available, a link to the specific clinicaltrials.gov listing will be present in the "More Information" section below.
Three hundred fifty-two CF patients (Age >= 5 years) with normal to mildly impaired lung function were enrolled in this Phase 3 study that evaluated the efficacy and safety of denufosol. Patients received either 60 mg denufosol or placebo nebulized three times daily for 24 weeks followed by 24 week treatment with open-label denufosol. The primary endpoint was met for the study with mean changes in FEV1 from baseline to Week 24 significantly greater for denufosol (0.048 L) than for placebo (0.003L) (treatment effect, 0.045; P = 0.047).
No significant differences between groups were observed for secondary endpoints including exacerbation rate and other measures of lung function.
Denufosol was well tolerated and adverse event and growth profiles were similar to placebo.