A PHASE 3, PLACEBO-CONTROLLED TRIAL WITH AZTREONAM LYSINATE FOR INHALATION IN CF

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This subproject is one of many research subprojects utilizing the resources provided by a Center grant funded by NIH/NCRR. The subproject and investigator (PI) may have received primary funding from another NIH source, and thus could be represented in other CRISP entries. The institution listed is for the Center, which is not necessarily the institution for the investigator. Patients with cystic fibrosis (CF) often have chronic lung infections. Long-term antibiotic treatment, coupled with gains in the life span of CF patients, make the development of bacterial resistance to antibiotics a significant concern for CF doctors. The development of new antibiotics or formulations is very important for the treatment of patients with CF. Aztreonam is an approved antibiotic with antibacterial activity similar to that of aminoglycoside antibiotics. When given intravenously, it has been shown to be safe and efficacious for treatment of lung infections in patients with CF. A new formulation of aztreonam, Aztreonam for Inhalation (AI), has been developed as an option for treatment of pulmonary infections. Aztreonam kills bacteria through a different mechanism than aminoglycosides, thus, aztreonam should not contribute to the emergence of aminoglycoside-resistant bacteria. In a phase I study and phase II study of AI in stable patients with CF (n=100), AI appeared to be safe and well-tolerated, resulted in a significant killing of Pseudomonas bacteria and improved lung function after 7 and 14 days of treatment with the 75 mg dose two times a day for 14 days. Results also suggested that the use of bronchodilators may improve AI deposition into the lungs. This phase III double-blind, multicenter, randomized, placebo-controlled clinical trial (n=300) will evaluate the safety and efficacy of two doses of AI over 28 days in 300 patients with CF.