Study Results

• What We Learned:

• Primary Findings:

  Effectiveness:

  One hundred thirty-five CF subjects (age >/=18 years) with pulmonary PA were enrolled in this Phase 2 study to evaluate safety and efficacy of Fosfomycin/Tobramycin for inhalation (FTI). Subjects received open-label Cayston® (75 mg TID) for 28-days before being randomized to treatment with either FTI 80/20mg or FTI 160/40mg or placebo BID for 28 days. The trial met its primary efficacy endpoint: after 28 days of treatment, the FTI 160/40 mg dosing group maintained the improvement in FEV1 % predicted achieved by the 28-day AZLI run-in period, in contrast to the placebo treatment group, where lung function declined toward pre-AZLI levels. The treatment effect was 6.2% favoring FTI (p=0.002).

   

  FEV1 % predicted was also maintained in the FTI 80/20 mg dosing group. The treatment effect on mean PA sputum density was statistically significant for the FTI 80/20 mg group versus placebo. The proportion of patients hospitalized and the number of hospitalization days did not differ significantly between treatment groups over the course of the study. Improvement in respiratory symptoms achieved by the AZLI run-in, as measured by the CFQ-R and RSSQ, was better maintained in the FTI 80/20 mg treatment group than in the FTI 160/40 mg or placebo groups.

  Safety:

  FTI was well tolerated. Adverse events, primarily cough, were consistent with CF disease. The FTI 80/20 mg group was more likely to complete treatment and reported fewer respiratory AEs than those in the FTI 160/40 group.

• Citation:

  Am J Respir Crit Care Med 2012;DOI 10.1164/rccm.201105-0924OC;185(2):171-180

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