Pediatric Brain Tumor Consortium (PBTC)

儿童脑肿瘤联盟 (PBTC)

• 批准号: 9919067
• 负责人: Ira J Dunkel
• 金额: $ 278.14万
• 依托单位: ST. JUDE CHILDREN'S RESEARCH HOSPITAL
• 依托单位国家: 美国
• 财政年份: 1999
• 资助国家: 美国
• 起止时间: 1999-04-01 至 2020-03-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/9919067
• 关键词: Antibodies; Biological; Biology; Brain Neoplasms; CTLA4 gene; Cancer Etiology; Cancer Trials Support Unit; Cancer Vaccines; Central Nervous System Diseases; Central Nervous System Neoplasms; Cessation of life; Characteristics; Child; Childhood; Childhood Brain Neoplasm; Childhood Central Nervous System Neoplasm; Clinical Trials; Collaborations; Data; Diffuse intrinsic pontine glioma; Enrollment; Ependymoma; Face; Family; Future; Genomics; Glioma; Goals; Grant; Immunotherapy; Infrastructure; Innovative Therapy; Institution; Intervention; Knowledge; Laboratories; Laboratory Study; Malignant Childhood Neoplasm; Measures; Medidata; Mission; Molecular; Molecular Abnormality; Monitor; Multi-Institutional Clinical Trial; Mutation; Neurosurgeon; New Agents; Oncologist; Oncolytic viruses; Pathway interactions; Patient-Focused Outcomes; Patients; Pediatric Brain Tumor Consortium; Pharmacogenomics; Pharmacologic Substance; Pharmacology; Phase; Pilot Projects; Process; Quality of life; Radiation Oncologist; Recurrent disease; Research; Research Design; Research Personnel; SLEB2 gene; Science; System; Time; Translating; Treatment Failure; Tumor Biology; base; disorder control; experience; innovation; medulloblastoma; member; multidisciplinary; neuroimaging; novel; oncology; pediatric patients; phase II trial; preservation; public health relevance; research clinical testing; response; success; targeted agent; tumor

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.

描述（由申请人提供）：迄今 肿瘤仍低于大多数其他主要类型的童年癌症。在将CNS疾病患者的多种创新疗法从实验室转化为早期临床测试时，我们的未来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的计算基础架构转换为Medidata Rave System，并且在新的赠款期开始时肯定会实现它。我们已经开始利用NCI赞助的癌症试验支持单元（CTSU）进行监管支持。在新赠款期间启动的所有PBTC试验的患者注册将通过CTSU肿瘤患者的入学网络（OPEN）。因此，这些更改将有助于与COG进行更紧密的合作，该COG使用Medidata Rave和CTSU。这将促进从PBTC试验到COG的数据，以实施随后的试验。此外，这些系统提供了一种促进选定的COG成员机构的方法，以参与需要非常罕见的CNS肿瘤患者应计的PBTC试验，这对于针对激活特定途径或特定遗传异常的新药物的试验可能需要进行。 PBTC的任务是通过创新的，多学科的，多机构的临床试验，迅速有效地为小儿中枢神经系统肿瘤的理解和治愈做出贡献，基于对不断发展的知识 脑肿瘤生物学以及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调查人员认识到他们在治疗期间和之后面临巨大挑战时，他们有责任研究患者和家庭的特殊需求。