ViKTriY-PC Consortium Phase II Supplement

 描述（由申请人提供）：药物开发的新时代代表了如何考虑早期研究相对于不考虑潜在肿瘤基因组学、生物学和免疫学的“常规”治疗方法的范式转变。翻译终点，包括目标表达水平，参与和下游效应物的调节正在尽可能早地进行评估，并且越来越重视早期患者选择，利用新型生物标志物测定和分子表征来识别最有可能应答的患者。随着这些终点变得越来越常见，将临床试验分为不同的1期或2期研究的传统分类往往是不够的。相反，这些研究已经被混合到“早期临床试验”分类中，并且需要更广泛的专业知识来进行这些研究。NCI实验治疗-临床试验网络（ET-CTN）的最终目的是确定开发新型抗癌药物的更好方法，这些药物利用分子表征肿瘤的能力，并找到合适的生物标志物来选择最有可能对特定药物产生反应的患者。虽然最初的ET-CTN UM 1资助重点是1期，但通过将2期临床试验合并到网络中来扩大活动范围，可以更好地实现这一目标。本申请是对我们的母公司ETCTN UM 1奖（1UM 1CA 186689）的修订，旨在描述我们进行II期临床试验的能力。我们已经增加了2个机构，我们原来的合作伙伴关系，并已命名为范德比尔特-英格拉姆，Karmanos，TGen研究所，耶鲁大学-太平洋海岸（加州大学圣地亚哥分校和加州大学旧金山弗朗西斯科）（ViKTriY - PC）。随着II期能力的增加，我们的团队的目标是：1）利用机构科学，在罕见癌症、常见癌症和常见癌症的不常见变体中使用CTEP组合开发II期试验; 2）将血清、组织和成像生物标志物纳入新型II期试验，以更好地了解新型药物单独或联合使用的效果; 3）培养研究员和初级教师，他们在1期和2期范围内进行早期临床试验方面知识渊博并精通，并帮助他们将有希望的试验推进到3期。该团队的成员拥有一套独特的互补专业知识，以及关于合作研究和指导下一代癌症研究人员的类似理念。该ViKTriY-PC团队致力于利用其专业领域并最大限度地发挥其合作关系，在ET-CTN内开展尖端I期和II期试验，最终使命是改善癌症患者的结局。

 DESCRIPTION (provided by applicant): The new era in drug development represents a paradigm shift in how early phase studies are considered relative to the "conventional" approach of treating without consideration to underlying tumor genomics, biology and immunology. Translational endpoints, including levels of target expression, engagement, and modulation of downstream effectors are being assessed as early as possible, and increasing emphasis is being placed on early patient selection, utilizing novel biomarker assays, and molecular characterization to identify patients most likely to respond. As these endpoints have become more common, the traditional classification of clinical trials as distinct Phase 1 or Phase 2 studies is often insufficient. Rather, these studies have become blended into an "early clinical trial" classification, and a wider range of expertise is necessary to conduct them. The ultimate purpose of the NCI Experimental Therapeutics-Clinical Trials Network (ET-CTN) is to define better approaches for the development of novel anticancer agents that capitalize on the ability to characterize tumors molecularly and find appropriate biomarkers to select patients most likely to respond to specific agents. While the original ET-CTN UM1 grant had a Phase 1 emphasis, this goal can be better achieved by expanding the scope of activities by merging Phase 2 clinical trials into the Network. This application is a revision to our parent ETCTN UM1 award (1UM1CA186689) with the purpose of describing our capabilities to conduct Phase 2 clinical trials. We have added 2 institutions to our original partnership, and have termed it Vanderbilt-Ingram, Karmanos, TGen Research Institute, Yale - Pacific Coast (University of California-San Diego and University of California-San Francisco) (ViKTriY - PC). As Phase 2 capabilities are added, our team aims to 1) leverage institutional science to develop Phase 2 trials using the CTEP portfolio in rare cancers, common cancers, and uncommon variants of common cancers; 2) incorporate serum, tissue and imaging biomarkers into novel Phase 2 trials to better understand the effects of novel agents either alone or in combination; 3) and develop fellows and junior faculty who are knowledgeable and proficient in conducting early phase clinical trials across the Phase 1 and 2 spectrum, and help them advance promising trials to Phase 3. The members of this team have a unique set of complementary expertise and a similar philosophy regarding collaborative research and mentorship of the next generation of cancer investigators. This ViKTriY-PC team is committed to utilizing their areas of expertise and maximizing their collaborative relationships to conduct cutting- edge Phase 1 and 2 trials within the ET-CTN with the ultimate mission of improving outcomes for patients with cancer.