Active Comparator: Study Design

The active comparator trial studied the efficacy and safety of Cayston compared to Tobramycin Nebulizer Solution (TNS)

Active Comparator Trial: Study design8

Study Description

  • Open-label, randomized, parallel-group, active comparator study of 268 patients ≥6 years of age with FEV1 ≤75% predicted, with CF and pulmonary Pa infection
  • The study was conducted in 91 CF centers in Europe and the US from 2008 to 2010


  • Patients were randomized (1:1) to three 28-day on/off treatment courses of CAYSTON 75 mg, 3 times daily or tobramycin nebulizer solution (TNS) 300 mg, twice daily
  • The 26-week active-comparator period was followed by an optional, 24-week, open-label, single-arm, extension period of 3 CAYSTON treatment cycles for eligible patients at European study sites
  • Additional TNS use was prohibited during the Active Comparator period
  • Additional antipseudomonal antibiotics could be administered for symptoms consistent with the diagnosis of acute pulmonary exacerbation
  • Patients receiving additional antipseudomonal antibiotics at any point after randomization could continue study treatment

Co-primary Endpoints

  • Superiority of CAYSTON for actual change in FEV1% predicted across 3 treatment cycles
  • Non-inferiority of CAYSTON relative change in FEV1% predicted at Day 28

Treatment Experience

  • TNS use ≥84 days in the previous year was reported for 85.1% of patients
  • Patients with a history of intolerance of TNS were excluded from study enrollment

Active Comparator Trial: Baseline demographics and clinical characteristics8

CAYSTON Active Comparator: Baseline demographics and clinical characteristics CAYSTON Active Comparator: Baseline demographics and clinical characteristics
*n=131, 131, and 262 for 3 columns.
Approved indication is for patients with FEV1 ≥25% or ≤75% predicted.
Enrollment included ≤40 patients with <84 days inhaled tobramycin use in the previous year. One patient was mistakenly stratified to ≥84 days, and 2 patients were mistakenly stratified to <84 days.
Learn About Active Comparator Trial Efficacy Results