Active Comparator: Efficacy Study

Important information about the Active Comparator Study

  • This study was designed and funded by Gilead Sciences, Inc. (GSI) with input from all authors on this publication. Some of the authors had consultancy with or were employees and/or shareholders of GSI
  • The results and conclusions of this single study should be interpreted in light of its limitations, including, but not limited to, the open-label design and inclusion of previously treated patients with inhaled tobramycin (TNS). A comparable study has not been conducted to verify these results
  • There are conflicting views regarding the validation of the Cystic Fibrosis Questionnaire-Revised (CFQ-R). The FDA determined that the CFQ-R was not validated in accordance with its final Guidance for Industry: Patient-Reported Outcome Measures: Use in Medical Product Development to Support Labeling Claims4
  • All results from the extension phase of the Active Comparator Trial are exploratory and therefore caution should be taken when interpreting the results

Active Comparator Trial: Co-primary efficacy endpoints8

Superiority analysis: Adjusted mean actual change (SE) in FEV1% across 3 treatment cycles

CAYSTON: 2.05% (0.69); TNS: -0.66% (0.72)
Treatment Difference (CAYSTON-TNS): 2.70% (95% CI: 0.98%, 4.43%; p=0.002)

CAYSTON Superiority analysis: Adjusted mean actual change (SE) in FEV% across 3 treatment cycles

In this open-label study, standard error bars are for graphical representation of variability, and inferences regarding statistical significance should not be made.

Non-inferiority analysis: Mean relative change in FEV1% at Day 28

CAYSTON: 8.35%; TNS: 0.55%
Treatment Difference (CAYSTON-TNS): at Day 28: 7.80% (95% CI: 3.86%, 11.73%; p<0.001)

CAYSTON Non-inferiority analysis: Mean relative change in FEV% at Day 28

In this open-label study, standard error bars are for graphical representation of variability, and inferences regarding statistical significance should not be made.


Active Comparator Trial: Adjusted Mean Change in CFQ-R8,*

Adjusted mean change in CFQ-R

Average of 3 Courses
Δ=4.1
p=0.019

CAYSTON Non-inferiority analysis: Mean relative change in FEV% at Day 28

*CFQ-R RSS: CF questionnaire-revised respiratory symptoms score.

In this open-label study, standard error bars are for graphical representation of variability, and inferences regarding statistical significance should not be made.

Learn About Active Comparator Trial Safety Results
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