Study design (if review, criteria of inclusion for studies)
Multicenter, randomized, parallel group double-blind placebo-controlled trial
Participants
CF patients with mild lung function impairment age 5 years and older. Multicenter, conducted at 102 CF care centers in Australia, Canada and the United States. 685 patients were screened for the study and 466 patients (233 in each group) were randomized to study treatment.
Interventions
The active group (n=233) received 60 mg denufosol via inhalation three times daily
Outcome measures
The primary efficacy endpoint was change in FEV(1) in liters from Day 0 to week 48.
Main results
The adjusted mean change in FEV(1) was 40 mL for denufosol and 32 mL for placebo with a resulting treatment effect of 8 mL (95% CI -0.040, 0.056). The average rate of change in FEV(1) percent of predicted over 0 to 48 weeks was -3.04% for placebo vs. -2.30 for denufosol (a difference of 24% relative to placebo) among all patients. The incidence of pulmonary exacerbation was 26% vs. 21% for the placebo and denufosol groups with no differences in the time to first event. The study treatments were well tolerated and there was no evidence of systemic effects in any safety parameter assessed.
Authors' conclusions
In patients with CF treatment with denufosol for 48 weeks did not improve pulmonary function or reduce the incidence of pulmonary exacerbations.
Keywords: Adolescent; Adult; Child; Other drugs; denufosol; pharmacological_intervention; Inhalation OR nebulised;