AZTREONAM LYSINATE FOR INHALATION FOR CYSTIC FIBROSIS

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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. This study is a double-blind, muticenter, randomized, placebo-controlled trial with Aztreonam Lysinate for Inhalation (AI) in cystic fibrosis (CF) patients with pulmonary P. aeruginosa (PA) requiring frequent antibiotics. Up to 250 CF patients will be randomized from up to 60 CF centers throughout the U.S. CF patients must have (PA) present and have been using greater than 3 courses of Tobramycin Solution for Inhalation (TSI) within the last 12 months, have FEV1values = 25% to = 75% of predicted and be = 6 years of age. This study will assess the safety and efficacy of a 28-day treatment with (AI) at two dosage regiments compared to placebo and the ability of AI to maintain or improve clinical status following a 28 day course of (TSI). The study schedule will include a screening period, a treatment period, mid-treatment visit, and five follow-up visits. The total study period will be 126 days. The primary efficacy outcome will be the time to need for a course of inhaled or IV antibiotics with documented physician assessment of reason. Safety will be evaluated by monitoring adverse events, airway reactivity, vital signs, clinical symptoms, and serum chemistry and hematology. Microbiology will be evaluated by change in PA (CFU), disappearance or appearance of other bacterial pathogens, and change in minimum inhibitory concentration (MIC) of Aztreonam for PA. Other outcome measures include changes in FEV1, FVC, FEF 25-75 and changes in clinical symptoms as assessed by the CF Questionnaire-Revised (CFQ-R), physician's assessment of global change, patients' assessment of global change, hospitalizations, change in weight, missed school/work days, and change in CF symptoms and severity.