Pediatric Brain Tumor ConsortiumA multi-institutional consortium devoted to novel phase I and II clinical evaluations of experimental treatment approaches for pediatric CNS tumors.

小儿脑肿瘤联盟是一个多机构联盟，致力于小儿中枢神经系统肿瘤实验治疗方法的新型 I 期和 II 期临床评估。

• 批准号: 10055934
• 负责人: Ira J Dunkel
• 金额: $ 481.08万
• 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 2025-03-31
• 项目状态: 未结题
• 来源: https://reporter.nih.gov/project-details/10055934
• 关键词: Adult; Adverse event; Area; BRAF gene; Basic Science; Biological; Biology; Biometry; Biotechnology; Brain Neoplasms; Cancer Therapy Evaluation Program; Cancer Vaccines; Central Nervous System Neoplasms; Cessation of life; Child; Childhood; Childhood Brain Neoplasm; Childhood Central Nervous System Neoplasm; Clinical; Clinical Trials; Collaborations; Committee Members; Contracts; Convection; Data; Development; Diagnosis; Diffuse intrinsic pontine glioma; Eligibility Determination; Ependymoma; Focused Ultrasound; Future; Genomics; Geography; Glioma; Goals; Image; Imaging Techniques; Immune checkpoint inhibitor; Immunologics; Immunotherapeutic agent; Immunotherapy; Infant; Institution; Intervention; Intrinsic factor; Laboratories; Laboratory Study; Lead; Local Therapy; MAP Kinase Gene; Magnetic Resonance Imaging; Malignant Neoplasms; Measures; Mediation; Mission; Modality; Molecular Target; Monitor; Morbidity - disease rate; Mutation; National Cancer Institute; Neuraxis; Neurosurgeon; Oncologist; Oncolytic viruses; Pathway interactions; Pediatric Brain Tumor Consortium; Pediatric Neoplasm; Pediatric Oncology Group; Pediatrics; Pharmaceutical Preparations; Pharmacologic Substance; Pharmacology; Phase; Phase I Clinical Trials; Phase II Clinical Trials; Prognostic Factor; Quality of life; Radiation Oncologist; Radiation Oncology; Recurrence; Refractory; Research; Research Design; Research Personnel; Safety; Saint Jude Children' s Research Hospital; Science; Series; Signal Transduction; Site; Solid Neoplasm; Structure; System; Therapeutic; Tissues; Toxic effect; Translating; Translational Research; Treatment Failure; Tumor Biology; anticancer research; appropriate dose; base; data acquisition; data management; drug development; early phase clinical trial; epigenomics; experience; high risk; improved; innovation; medulloblastoma; member; mortality; multidisciplinary; neuroimaging; neuropathology; novel; novel strategies; operation; pediatric patients; pharmacokinetics and pharmacodynamics; phase 2 study; prognostic; programs; prospective; protocol development; quality assurance; research clinical testing; response; scientific organization; success; survival outcome; targeted agent; translational clinical trial; translational scientist; treatment response; treatment strategy; trial design; tumor; working group

PBTC Project Summary/Abstract The primary goal of the Pediatric Brain Tumor Consortium (PBTC) is to rapidly and effectively contribute to the understanding and cure of pediatric central nervous system (CNS) tumors through innovative, multidisciplinary and multi-institutional prospective clinical trials. PBTC clinical trial development is not only based upon known prognostic factors but also upon an evolving understanding of pediatric brain tumor biology. The PBTC will continue to utilize innovative study designs and analyses based on state-of-the-art statistical science. Novel clinical trials will incorporate relevant correlative pharmacology, biology, neuroimaging, immunologic and genomics studies in an effort to define and integrate these modalities into a better understanding of pediatric CNS tumors. These goals will be accomplished through a close and continued partnership with the member institutions' basic and translational scientists as well as the clinical trial experts. In addition, the PBTC welcomes partnerships with investigators outside the consortium providing scientific, laboratory and regulatory support for innovative and high priority clinical trials. The PBTC will continue to maintain and grow its relationship with the National Cancer Institute (NCI) and its large network of adult and pediatric groups as well as with pharmaceutical and biotechnology companies. One of the most important goals of the PBTC is to successfully complete focused phase I and II clinical trials of novel agents and treatment approaches that can be then be translated into large prospective phase II and III studies overseen by the Children's Oncology Group (COG). The overarching long-term goal is to improve survival outcome and quality of life for children with CNS tumor diagnoses that so often lead to high morbidity and mortality such as diffuse intrinsic pontine gliomas, high-grade gliomas, infant embryonal tumors, refractory medulloblastoma, ependymoma, and low-grade gliomas. To this end, the PBTC will continue to focus upon introducing novel molecularly-targeted agents via innovative trials to assess the presence of the target in tumor and surrogate tissue, evaluate the ability of the agent to down-regulate its signaling target and better correlate pharmacokinetic and pharmacodynamic endpoints with toxicities and clinical responses. The PBTC will also continue to explore therapeutic opportunities for pediatric brain tumors through immunotherapeutic interventions as well as via local delivery of treatments. By focusing on the most common solid tumor in children which also accounts for the highest number of cancer-related deaths in pediatrics, the PBTC will continue to support the NCI's mission of helping all people live longer, healthier lives through its contributions to cancer research and identification of better treatments.

PBTC项目摘要/摘要 小儿脑肿瘤联盟（PBTC）的主要目标是快速有效地 有助于理解和治愈小儿中枢神经系统（CNS）肿瘤 通过创新，多学科和多机构的前瞻性临床试验。 PBTC临床 试验开发不仅基于已知的预后因素，而且还基于不断发展的 了解小儿脑肿瘤生物学。 PBTC将继续使用创新研究 基于最先进的统计科学设计和分析。新颖的临床试验将 结合相关的相关药理学，生物学，神经影像学，免疫学和 基因组学研究是为了将这些方式定义和整合到更好的理解中 小儿中枢神经系统肿瘤。这些目标将通过近距离持续实现 与会员机构的基本和转化科学家以及临床的合作伙伴关系 审判专家。此外，PBTC欢迎与外面的调查员建立合作伙伴关系 财团为创新和高优先事项提供科学，实验室和监管支持 临床试验。 PBTC将继续维持和发展其与国家的关系 癌症研究所（NCI）及其大型成人和小儿组的网络以及 药物和生物技术公司。 PBTC最重要的目标之一是 成功完成针对新药物和治疗的I期和II期临床试验 然后可以将可以翻译成大量的前瞻性II和III研究的方法监督 由儿童肿瘤学组（COG）。总体长期目标是提高生存 CNS肿瘤诊断儿童的结果和生活质量经常会导致高 发病率和死亡率，例如弥漫性固有的蓬托神经胶质瘤，高级神经胶质瘤，婴儿 胚胎肿瘤，难治性的髓母细胞瘤，室膜瘤和低度神经胶质瘤。对此 最后，PBTC将继续专注于通过 创新试验以评估肿瘤中靶标的存在和替代组织的存在，评估 代理下调其信号靶标的能力并更好地相关的药代动力学和 具有毒性和临床反应的药效终点。 PBTC也将继续 通过免疫治疗探索儿科脑肿瘤的治疗机会 干预措施以及通过本地提供治疗。通过专注于最常见的固体 儿童的肿瘤也是癌症相关死亡人数最多的儿童 儿科，PBTC将继续支持NCI的使命，即帮助所有人寿命更长的时间， 通过对癌症研究的贡献和对更好治疗的识别的贡献，更健康的生活。