Conditions: Cystic Fibrosis Burkholderia Infections
Keywords: Cystic Fibrosis Aztreonam Lysine lung infection Burkholderia CFQ-R inhaled antibiotic
Allocation: RANDOMIZED | Intervention model: CROSSOVER
Enrollment: 102
Healthy volunteers: no

Brief summary:
The purpose of this research study was to determine if an experimental drug called Aztreonam for Inhalation Solution (AZLI) was safe and effective to treat Burkholderia lung infections in patients with cystic fibrosis (CF).

Spirometry was used to assess pulmonary function, and the revised Cystic Fibrosis Questionnaire (CFQ-R) was used to assess quality of life. The CFQ-R is a validated, patient-reported outcome tool used to measure health-related quality of life for children and adults with CF.

The study consisted of a 24-week randomized phase, and a 24-week open-label phase. Primary and secondary efficacy analyses were conducted for the 24-week randomized phase only. Safety data were collected for both the randomized and open-label phases.

Detailed description:

Arms:
arm 1: Participants were randomized to receive AZLI for up to 24 weeks and may have continued to receive AZLI during the open-label phase for up to an additional 24 weeks. arm 2: Participants were randomized to receive placebo to match AZLI for up to 24 weeks and may have switched to AZLI during the open-label phase for up to 24 weeks.

Interventions: intervention 1: AZLI intervention 2: Placebo
Intervention descriptions: intervention 1: Aztreonam for inhalation solution (AZLI; 75 mg aztreonam/52.5 mg lysine monohydrate) was administered three times a day, with at least 4 hours between doses, using the investigational nebulizer. intervention 2: Placebo to match AZLI (lactose and sodium chloride) was administered three times a day, with at least 4 hours between doses, using the investigational nebulizer.

Protocol document: