Active Comparator: Extension Phase
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 Extension Phase: Study Design8
The open-label Active Comparator period of the study was followed by an optional 24-week, open-label, single-arm extension period of 3 CAYSTON treatment cycles.
All patients who participated in the extension phase of the Active Comparator Study were treated with CAYSTON*
*Eligible patients for the extension phase were from European study sites only (n=133; patients continuing CAYSTON from active comparator phase: 68; patients starting CAYSTON after TNS in active comparator phase: 65).
Active Comparator Extension Phase: Efficacy Results8
Study Extension Phase: Percent Change in FEV1% Predicted from Week 0
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 Extension Phase: Adverse Reactions8
Adverse events occurring during the extension period8
- Severe adverse events were experienced by 15/133 (11.3%) patients, most commonly cough (n=7/133), productive cough (n=5/133), and pyrexia (n=5/133)
- One life-threatening event was reported (lung infiltration unrelated to treatment)
- Two patients died due to complications of CF disease; both deaths were considered unrelated to treatment
- Adverse events considered by investigators as treatment-related were reported for 6.8% of patients (n=9/133), most commonly respiratory incidents (n=6/133)