AEROSOLIZED TYLOXAPOL IN THE AIRWAYS DISEASE OF CYSTIC FIBROSIS

这是一个由研究者构思、编写和实现的协议。首席研究员提交了一份RO1申请以支持本研究。马歇尔博士获得了FDA的批准，可以继续进行这项研究。随后，探索实验室获得了这种孤儿药的技术许可，马歇尔博士将他的NIH申请提交给了该公司，后者同意支持该项目。迄今为止，研究人员对两组正常志愿者进行了研究。第一组10人以剂量递增的方式接受泰洛沙波尔治疗14天，从0.125%开始，逐渐增加到5.0%。3名受试者出现非生产性咳嗽。然后将最大剂量降至2.5%。一名受试者在服用这一剂量后出现咳嗽。停药后咳嗽就好了。另外10名正常志愿者在较低剂量的泰洛沙波尔下接受了更长时间的测试。这些剂量耐受性良好。在两组正常志愿者中，均未发现有临床意义的实验室异常。这项研究的下一阶段，即在囊性纤维化患者身上测试药物，已经开始进行。

This is an investigator-conceived, -written, and -implemented protocol. An RO1 application to support this study was submitted by the Principal Investigator. Dr. Marshall had approval from the FDA to move forward with this study. Discovery Laboratories then licensed the technology for this orphan drug, and Dr. Marshall brought his NIH application to the company, who agreed to support the project. To date, two groups of normal volunteers have been studied. The first group of 10 received tyloxapol for 14 days in a dose-escalating fashion, starting at 0.125% and escalating to 5.0%. A non-productive cough developed in three subjects. The maximum dose was then decreased to 2.5%. One subject developed a cough on this dose. The cough resolved upon discontinuation of the drug. Ten additional normal volunteers were then tested at lower doses of tyloxapol and for longer periods of time. These doses were well-tolerated. In both groups of normal volunteers, no clinically significant laboratory abnormalities developed. The next phase of this investigation, namely testing the drug on patients with cystic fibrosis, has just gotten under way.