DENUFOSOL TETRASODIUM INHALATION SOLUTION IN PATIENTS WITH MILD CF

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。 德诺福索四钠(INS37217)是一种新型的第二代、化学稳定、选择性的P2Y2受体激动剂，可以恢复或维持CF肺部疾病病程早期患者的粘液纤毛清除，从而保护肺功能，减少不可避免的肺部细菌定植、肺恶化和慢性肺功能下降的复发周期。这项多中心、随机、双盲、安慰剂对照的第三阶段研究将检验在相对健康的CF患者群体中，地诺福索作为一种相对于安慰剂改善肺功能的早期干预疗法。这项研究的目的是评估60毫克的雾化吸入剂量的地诺福索与安慰剂相比，在24周内每天三次(在开放标签期内再给药24周)对轻度慢性肺病患者的有效性、安全性和耐受性，并评估参与试验的轻度肺病患者的健康相关生活质量。

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. Denufosol tetrasodium (INS37217) is a novel second-generation, chemically stable, selective P2Y2 receptor agonist that may have the ability to restore or maintain mucociliary clearance in patients early in the course of CF lung disease, which may therefore preserve lung function and lessen the inevitable recurrent cycles of pulmonary bacterial colonization, pulmonary exacerbations, and chronic lung function decline. This multicenter, randomized, double-blind, placebo-controlled Phase 3 study will examine denufosol as an early intervention therapy to improve lung function relative to placebo in a relatively healthy CF patient population. The objectives of this study are to evaluate the efficacy, safety and tolerability of a 60 mg nebulizer dose of denufosol compared to placebo when administered three times daily over 24 weeks (and for an additional 24 weeks in an open label period) in patients with mild CF lung disease and to evaluate health-related quality of life in patients with mild CF lung disease participating in this trial.