Hypertonic Saline for Cystic Fibrosis

描述(申请人提供)：囊性纤维化(CF)是一种常染色体致死性疾病，在美国约有30,000人患病。慢性阻塞性肺疾病是导致慢性阻塞性肺疾病死亡的主要原因，其原因是呼吸道分泌物脱水，导致粘液粘连、感染和呼吸道炎症。吸入高渗盐水(HS)是恢复CF呼吸道水化和粘液清除的一种简单方法，高渗盐水会将水吸入到呼吸道表面。呼吸道表面液体润滑层的再水化有助于粘液纤毛清除(MCC)，从而清除吸入的感染物。先前发表的证据表明，短期服用HS可改善CF患者的痰的流变性和转运能力，水化呼吸道表面，改善MCC和肺功能。最近的临床研究支持每日HS治疗CF的疗效，显示出肺功能的改善、肺恶化的减少和生活质量的改善。因此，吸入型HS正在成为CF的标准护理，目前全国约50%的患者使用HS。将HS添加到CF治疗方案的一个后果是CF患者使用HS所需的时间。每天的HS治疗需要大约1小时的时间承诺，这与通常要求这些患者进行其他喷雾剂和物理治疗的~2小时护理时间重叠。因此，迫切需要减少该疗法的治疗负担(时间承诺)，以提高CF患者的生活质量。Parion Science的目标是开发出比目前的治疗方法更有效的治疗方法。Parion正在开发一种专门的过夜服用HS的设备，将减少CF患者的日间治疗需求。Parion已经开发出一种新的鼻腔气雾剂给药装置，旨在通宵服用治疗剂量的HS，舒适、安静，并与睡眠兼容。该设备在临床沉积研究中进行了测试，获得了高于预期的HS气雾剂沉积，证实该设备将能够获得足够高的HS沉积肺剂量，以支持未来的临床试验。在沉积研究成功的基础上，Parion将在第二阶段临床研究中通过评估通过该设备输送HS的耐受性和有效性来继续开发计划。如果成功，我们相信这种方法将提高CF的生活质量 通过减少与日常HS治疗相关的治疗负担，提高这种治疗的依从性。

DESCRIPTION (provided by applicant): Cystic fibrosis (CF) is an autosomal lethal disease characterized that affects ~30,000 individuals in the US. CF lung disease, the major cause of mortality in CF, is caused by dehydration of airway secretions that leads to mucous adhesion, infection and airways inflammation. A simple means to restore hydration and mucous clearance in CF airways is the inhalation of hypertonic saline (HS), which osmotically draws water onto the airway surface. Rehydration of the lubricant layer of the airway surface liquid facilitates mucociliary clearance (MCC) and therefore the removal of inhaled infectious agents. Previously published evidence has shown that short-term administration of HS improves the rheologic properties and transportability of sputum, hydrates the airway surface, and improves MCC and lung function in patients with CF. Recent clinical studies support the efficacy of daily HS treatments for CF demonstrating improvements in lung function, a reduction in pulmonary exacerbations, and improvements in quality of life. As a result, inhaled HS is becoming a standard of care for CF and is now used by ~50% of patients nationwide. One consequence of the addition of HS to CF treatment regimens is the time required by CF patients for its administration. Daily HS treatments require ~1 hour of time commitment, which is superimposed on the ~2 hours of care typically asked of these patients for other aerosols and physiotherapy. Thus, there is a great need to reduce the treatment burden (time commitment) of this therapy to improve the quality of life of CF patients. Parion Science's goal is to develop therapies that provide major improvements in efficacy over current treatments for CF. Parion is developing a specialized device of overnight administration of HS that will reduce daytime treatment requirements for CF patients. Parion has developed a novel nasal aerosol delivery device designed to administer a therapeutic dose of HS overnight that is comfortable, quiet, and compatible with sleep. The device was tested in a clinical deposition study where it achieved a higher than expected pulmonary deposition of HS aerosol, confirming that the device will be able to achieve a sufficiently high deposited pulmonary dose of HS to support future clinical trials. Based on the success of the deposition study, Parion will continue the development program by evaluating the tolerability and efficacy of HS delivery via the device in a Phase 2 clinical study. If successful, we believe that this approach will improve the quality of life of CF patients by decreasing the treatment burden associated with daily HS treatments and improve compliance of this therapy.