AEROSOLIZED TYLOXAPOL IN THE AIRWAYS DISEASE OF CYSTIC FIBROSIS

雾化泰洛沙泊治疗气道囊性纤维化疾病

• 批准号: 6218447
• 负责人: BRUCE C MARSHALL
• 金额: $ 0.16万
• 依托单位: UNIVERSITY OF UTAH
• 依托单位国家: 美国
• 财政年份: 1998
• 资助国家: 美国
• 起止时间: 1998-12-01 至 1999-11-30
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/6218447
• 关键词: aerosols; bioassay; clinical research; cystic fibrosis; detergents; drug adverse effect; drug screening /evaluation; experimental designs; human subject; particle; volunteer

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.

这是一个由制造商构思、编写和实施的方案。 主要研究者提交了支持本研究的RO1申请。马歇尔博士已获得FDA的批准，可以继续进行这项研究。 然后，探索实验室批准了这种孤儿药的技术，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，已经研究了两组正常志愿者。 第一组10人以剂量递增方式接受泰洛沙泊14天，从0.125%开始递增至5.0%。 3例受试者出现干咳。 然后将最大剂量降至2.5%。 1例受试者在该剂量下发生咳嗽。 停药后咳嗽消退。另外10名正常志愿者在较低剂量的泰洛沙泊和较长的时间内进行了测试。 这些剂量耐受良好。在两组正常志愿者中，均未出现具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，刚刚开始。