Following the recent success of a phase 3b clinical trial for Aztreonam in cystic fibrosis (CF) patients with Burkholderia infections, Gilead Sciences, Inc., a research-based biopharmaceutical company engaged in CF drug discovery and development, completed another phase 3 clinical trial to evaluate the safety of inhalable Aztreonam (AZLI) in children up to 12 years of age with CF and chronic Pseudomonas aeruginosa (PA) infection in the lower airways. A total of 61 participants from 25 study sites in the United States and Europe were given treatment.
The primary purpose of this open-label interventional study was the treatment of the opportunistic infection, consisting of three 28-day courses of AZLI three times daily, each followed by 28 days off the drug for a total treatment of 6 months.
Safety data was collected throughout the course of the treatment, and outcome measures included spirometry testing to evaluate pulmonary function and the revised Cystic Fibrosis Questionnaire (CFQ-R) to evaluate the quality of life of the patients, both increasing above baseline in treated subjects. Change in Pseudomonas Aeruginosa sputum density (colony-forming units per gram) was also evaluated, showing a decrease during the course of the treatment. Overall, researchers reported no safety issues, which means that AZLI could become a viable treatment for infection in CF patients in the near future.
Pseudomonas aeruginosa is the most important pathogen in the CF airway, and can cause acute and chronic infections and in extreme cases can lead to death of the patient.
P.aeruginosa is able to undergo a series of adaptive changes such as loss of flagellar and pilin-dependent motility, lipopolysaccharide changes and antibiotic resistence, which ultimately have an adverse impact in the clinical course of CF disease.