Pediatric Brain Tumor Consortium (PBTC)

描述（由申请人提供）：迄今为止，人们必须得出结论，在疾病控制和儿童大脑功能完整性和生活质量的保护方面的进展速度 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CNS疾病儿科患者的早期临床试验过程中，我们未来的PBTC临床试验将更多地关注与这些肿瘤治疗相关的基因组学，以及PBTC II期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，并将在新的授权期开始时将其安装到位。我们已经开始利用NCI-sponsored Cancer Trials Support Unit（CTSU）提供监管支持。在新的资助期内启动的所有PBTC试验的患者登记将通过CTSU肿瘤患者登记网络（OPEN）进行。因此，这些变化将促进与使用MediData Rave和CTSU的COG进行更密切的合作。这将有助于向委员会提供PBTC试验的数据，以便开展后续试验。此外，这些系统还提供了一种方法，便于选定的COG成员机构参与PBTC试验，这些试验需要招募非常罕见的CNS肿瘤患者，这可能需要用于靶向特定通路激活或特定遗传异常肿瘤的新药试验。PBTC的使命是通过基于以下方面不断发展的知识的创新、多学科、多机构临床试验，快速有效地为儿童CNS肿瘤的理解和治疗做出贡献： 脑肿瘤生物学和使用基于最先进的统计科学的研究设计和分析。PBTC致力于将临床试验与相关药理学、生物学、神经影像学和基因组学研究相结合。PBTC研究人员认识到他们有责任研究患者和家庭的特殊需求，因为他们在治疗期间和治疗后面临巨大的挑战。

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.