Pediatric Brain Tumor Consortium (PBTC)

描述(由申请人提供)：到目前为止，人们必须得出结论，在疾病控制和保护儿童大脑的功能完整性和生活质量方面的进展速度 肿瘤仍然低于大多数其他主要类型的儿童癌症。在将多种创新疗法从实验室转化为儿童中枢神经系统疾病患者的早期临床测试时，我们未来的PBTC临床试验将更多地专注于了解与这些肿瘤的治疗相关的基因组学，以及PBTC第二阶段试验中的药物基因组学。OBC正在将支持PBTC的计算基础设施转换为Medidata Rave系统，并肯定会在新的授权期开始时将其到位。我们已经开始利用NCI赞助的癌症试验支持单位(CTSU)提供监管支持。在新的授权期间启动的所有PBTC试验的患者注册将通过CTSU肿瘤学患者登记网络(OPEN)进行。因此，这些变化将促进与使用Medidata Rave的COG和CTSU的更紧密合作。这将有助于向组委会提供来自PBTC试验的数据，以便实施后续试验。此外，这些系统提供了一种手段，便于选定的COG成员机构参与PBTC试验，这些试验需要非常罕见的中枢神经系统肿瘤患者的收益，就像针对具有特定路径激活或特定基因异常的肿瘤的新药物试验所需的那样。PBTC的使命是根据不断发展的知识，通过创新的、多学科的、多机构的临床试验，快速有效地为了解和治疗儿童中枢神经系统肿瘤做出贡献 脑肿瘤生物学和使用研究设计和分析基于最先进的统计科学。PBTC致力于将临床试验与相关的药理学、生物学、神经成像和基因组学研究相结合。PBTC调查人员认识到，他们有深厚的责任来研究他们的患者和家人的特殊需求，因为他们在治疗期间和治疗后都面临着巨大的挑战。

DESCRIPTION (provided by applicant): To date, one must conclude that the pace of progress in disease control and preservation of functional integrity and quality of life for pediatric brain tumors remains below that of most other major types of childhood cancers. In translating multiple innovative therapies from the laboratory to early phase clinical testing in pediatric patients with CNS diseases, our future PBTC clinical trials will focus more on understanding genomics related to the treatment of these tumors, as well as pharmacogenomics in PBTC phase II trials. The OBC is in the process of converting the computing infrastructure that supports the PBTC to the MediData Rave system and will definitely have it in place by the time the new grant period starts. We have already begun to utilize the NCI-sponsored Cancer Trials Support Unit (CTSU) for regulatory support. Patient registration for all PBTC trials initiated in the new grant period will be via the CTSU Oncology Patient's Enrollment Network (OPEN). Thus, these changes will facilitate closer collaborations with the COG, which is using MediData Rave and the CTSU. This will facilitate providing data from PBTC trials to the COG for the implementation of subsequent trials. In addition, these systems provide a means for facilitating selected COG member institutions to participate in PBTC trials that require accrual of patients with very rare CNS tumors, as may be required for trials of new agents that target tumors with specific pathways activated or with specific genetic abnormalities. The Mission of the PBTC is to contribute rapidly and effectively to the understanding and cure of pediatric CNS tumors through innovative, multidisciplinary, multi-Institutional clinical trials based upon evolving knowledge of brain tumor biology and using study designs and analyses based on state-of-the-art statistical science. The PBTC is committed to integrating clinical trials with correlative pharmacology, biology, neuroimaging and genomics studies. PBTC investigators recognize their profound responsibility to study the special needs of their patients and families, as they face enormous challenges during and after therapy.