AZTREONAM LYSINATE FOR INHALATION FOR CYSTIC FIBROSIS

该子项目是利用 由NIH/NCRR资助的中心赠款提供的资源。子项目和 研究者（PI）可能从另一个NIH来源获得主要资金， 因此可以在其他CRISP条目中表示。列出的机构是 中心，不一定是研究者的机构。 本研究是一项双盲、多中心、随机、安慰剂对照试验，在需要频繁使用抗生素的肺铜绿假单胞菌（PA）囊性纤维化（CF）患者中使用吸入用赖氨酸氨曲南（AI）。 最多250例CF患者将从美国最多60家CF中心随机分配。CF患者必须存在（PA）并在过去12个月内使用超过3个疗程的妥布霉素吸入溶液（TSI），FEV 1值=预测值的25%至= 75%，年龄= 6岁。本研究将评估与安慰剂相比，两种剂量方案的（AI）治疗28天的安全性和有效性，以及AI在（TSI）治疗28天后维持或改善临床状态的能力。研究时间表将包括筛选期、治疗期、治疗中期访视和5次随访访视。 总研究期为126天。 主要疗效结局将是需要吸入或IV抗生素疗程的时间，并记录医生评估的原因。将通过监测不良事件、气道反应性、生命体征、临床症状以及血清化学和血液学来评价安全性。 将通过PA（CFU）的变化、其他细菌病原体的消失或出现以及氨曲南对PA的最小抑菌浓度（MIC）的变化来评价微生物学。 其他结局指标包括FEV 1、FVC、FEF 25-75的变化和CF量表修订版（CFQ-R）评估的临床症状变化、医生对总体变化的评估、患者对总体变化的评估、住院、体重变化、缺课/工作日以及CF症状和严重程度的变化。

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.