CLINICAL TRIAL: DENUFOSOL TETRASODIUM INHALATION SOLUTION IN PATIENTS WITH MILD

点击翻译按钮获取中文摘要

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.