DENUFOSOL TETRASODIUM INHALATION SOLUTION IN PATIENTS WITH MILD CF

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。 地诺福索四氢呋喃（INS 37217）是一种新型的第二代、化学稳定的选择性P2 Y2受体激动剂，可能能够在CF肺病病程早期恢复或维持患者的粘膜纤毛清除率，因此可能保护肺功能，并减少肺细菌定植、肺加重和慢性肺功能下降的不可避免的复发周期。这项多中心、随机、双盲、安慰剂对照的3期研究将在相对健康的CF患者人群中检查地努福索作为早期干预治疗，相对于安慰剂改善肺功能。 本研究的目的是在轻度CF肺病患者中评价与安慰剂相比，地努福索60 mg雾化剂每日3次给药持续24周（开放标签期再持续24周）的疗效、安全性和耐受性，并评价参与本试验的轻度CF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.