AZTREONAM LYSINATE FOR INHALATION FOR CYSTIC FIBROSIS

这个子项目是许多研究子项目中利用 资源由NIH/NCRR资助的中心拨款提供。子项目和 调查员(PI)可能从NIH的另一个来源获得了主要资金， 并因此可以在其他清晰的条目中表示。列出的机构是 该中心不一定是调查人员的机构。 这项研究是一项双盲、多中心、随机、安慰剂对照试验，使用氨曲南来辛酸盐雾化吸入(AI)治疗囊性纤维化(CF)合并需要经常使用抗生素的肺部铜绿假单胞菌(PA)患者。多达250名CF患者将从全美60个CF中心随机抽取。CF患者必须有(PA)，并在过去12个月内使用超过3个疗程的妥布霉素溶液吸入(TSI)，FEV1值=预期的25%至=75%，BE=6岁。这项研究将评估两个剂量方案(AI)28天治疗的安全性和有效性，并与安慰剂进行比较，以及AI在28天疗程(TSI)后维持或改善临床状况的能力。研究时间表将包括筛查期、治疗期、治疗中期访问和五次后续访问。总学习时间为126天。主要的疗效结果将是需要一个疗程的吸入或静脉注射抗生素，并记录医生对原因的评估。安全性将通过监测不良事件、呼吸道反应性、生命体征、临床症状以及血清化学和血液学进行评估。微生物学将通过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)的变化来评估。其他结果指标包括FEV1、FVC、FEF25-75的变化和经修订的CFQ-R(CFQ-R)评估的临床症状的变化、医生对全球变化的评估、患者对全球变化的评估、住院、体重变化、旷课/工作天数以及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.