美国肿瘤药物开发过程中的0期临床试验

Phase 0 clinical trials in the development of oncology new drugs in USA

0期临床研究,在美国又被称为探索性新药临床研究（IND）研究,欧洲被称为探索性临床试验,指：在传统Ⅰ期剂量递增、安全性和毒性研究之前开展的,非常有限的人体暴露（〈30个患者,通常10~15个,≤7 d时间）并且没有治疗或者诊断意图的首次人体试验。本文从0期临床试验特点、探索性IND指导、肿瘤药物开发实践经验等方面介绍0期临床试验对美国肿瘤药物开发的影响。

Phase 0 clinical trials, also called exploratory IND in USA and exploratory clinical trials in EU, is performed prior to traditional phase 1 dose escalation, safety and toxicity studies. In these trials, human exposure is very limited （ 〈 30 subjects, always 10 - 15 subjects,≤7 d） and there is no therapeutic or diagnostic intent of first in human trials. Features of phase 0 clinical trials, exploratory IND guidance, and oncology drug development experience in the USA were reviewed in this article.