Shinkai T, Saijo N
Department of Internal Medicine, National Cancer Center Hospital, Japan.
Nihon Kyobu Shikkan Gakkai Zasshi. 1993 Dec;31 Suppl:232-44.
The goal of phase II clinical trials is to determine whether a new drug has activity against particular cancers. This trial, therefore, is the most important study in the process of development of investigational new agents and frequently may give misleading results. The design of phase II clinical trials of new anticancer agents in lung cancer has been reviewed based on the Guidelines for the Clinical Evaluation of Antineoplastic Agents notified by the Ministry of Health and Welfare in Japan. These guidelines present general approaches to clinical trials and should be adhered to in the development of investigational drugs in humans, although they are not directly mandated by law or regulations. We considered the policies of these guidelines and instituted them based on the actual circumstances in Japan, in order to develop a scientific and rational approach for clinical phase II trials. In addition, we introduced new active agents having a novel mechanism of action that show clinical activity in the treatment of lung cancer, such as irinotecan (CPT-11), a topoisomerase I inhibitor, and paclitaxel (taxol) and taxotere, targeting the microtubules.
II期临床试验的目的是确定一种新药是否对特定癌症具有活性。因此，该试验是研究性新药开发过程中最重要的研究，但常常可能给出误导性结果。基于日本厚生省发布的《抗肿瘤药物临床评价指南》，对肺癌新型抗癌药物的II期临床试验设计进行了综述。这些指南提出了临床试验的一般方法，尽管并非由法律法规直接强制要求，但在人类研究性药物的开发中应予以遵循。我们考虑了这些指南的政策，并根据日本的实际情况制定了这些政策，以便为临床II期试验制定科学合理的方法。此外，我们引入了具有新作用机制且在肺癌治疗中显示出临床活性的新型活性药物，如拓扑异构酶I抑制剂伊立替康（CPT-11），以及靶向微管的紫杉醇（泰素）和多西他赛。
