MOLECULAR ANALYSIS OF TUMORS FOR TREATMENT SELECTION IN CLINICAL TRIALS- PHASE II

美国国家癌症研究所(NCI)正在继续推行将基因组学纳入不断发展的精准医学国家战略的临床试验概念。最近对肿瘤分子异常的认识取得了进展，这些异常可以促进增殖或促进生存，导致了许多针对这些异常的治疗药物的开发。研究将筛选可能预测对具有特定作用机制的靶向制剂的反应的分子特征。在某些情况下，研究设计将使用“伞形方案”，即分子筛查的单一入口点然后将患者分配到多个单臂2/3期试验。该系列试验的一个例子是Match，于2015年7月向患者开放登记。其他试验将使用浓缩设计，其中特定的分子特征是资格标准，治疗仅限于此类患者。

The National Cancer Institute (NCI) is continuing to pursue clinical trials concepts that incorporate genomics into the evolving national strategy for precision medicine. Recent advances in knowledge of the molecular abnormalities in tumors that either drive proliferation or promote survival have led to the development of many therapeutic agents that target those abnormalities. Studies will screen for molecular features that may predict response to a targeted agent with a specific mechanism of action. In some cases, the study designs will use an "umbrella protocol" whereby a single entry point for molecular screening will then distribute patients to multiple, single-arm phase 2/3 trials. One example of the trials in the series, MATCH, was open for patient enrollment in July 2015. Other trials will use enrichment designs where the particular molecular characteristic is an eligibility criterion and the therapy is limited to such patients.