AEROSOLIZED TYLOXAPOL IN THE AIRWAYS DISEASE OF CYSTIC FIBROSIS

这是一个由调查员构思、编写和实施的协议。首席调查员提交了支持这项研究的RO1申请。马歇尔博士获得了FDA的批准，可以推进这项研究。发现实验室随后授权了这种孤儿药物的技术，马歇尔博士向该公司提交了他的NIH申请，该公司同意支持该项目。到目前为止，已经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14天的泰洛沙普治疗，从0.125%开始，逐渐增加到5.0%。三名受试者出现无效咳嗽。然后，最大剂量降至2.5%。一名受试者在服用这种剂量时出现咳嗽。停药后咳嗽消失了。然后，另外10名正常志愿者在较低剂量的泰洛沙普进行了较长时间的测试。这些剂量是耐受性良好的。在两组正常志愿者中，均未出现临床显著的实验室异常。这项研究的下一阶段，即在囊性纤维化患者身上测试药物，刚刚开始。

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.