Study Results

• What We Learned:

  This study was closed early due to poor enrollment. No significant conclusions could be drawn.

• Primary Findings:

  Effectiveness:

  This study was conducted between December 2012 and January 2015. Of the 88 participants who received study drug, 42 received CAT and 46 received the intermittent treatment. A total of 72 participants completed the treatment phase (CAT: N=36 and Intermittent: N=36). The study was ultimately closed to enrollment early because a feasibility assessment during the study indicated that the enrollment goal would not be reached. Feasibility issues included competing CF trials, increasing use of CAT as standard of care, and the approval of a dry powder formulation of inhaled tobramycin after this study started. The primary efficacy endpoint was the rate of pulmonary exacerbations during the study. Although the rate of pulmonary exacerbations was lower in the CAT treatment group than the intermittent treatment group, leading to a 25.7% reduction in exacerbation rate for the CAT treatment group, the difference was not statistically significant (p=0.25).

   

  Additional evaluations included average change in lung function (absolute change from baseline FEV1% predicted), percent of participants treated for a pulmonary exacerbation, time to first exacerbation, hospitalization rates and average change in the CFQ-R Respiratory Symptom Scale. No significant differences were seen between the CAT and intermittent groups for these evaluations.

  Safety:

  CAT was well tolerated and the adverse event profile was similar for both groups.

• Citation:

  J Cyst Fibros ;DOI 10.1016/j.jcf.2016.05.001

For current information about the overall development status of this drug, please check the Drug Development Pipeline.