Children's National Medical Center Application for NEXT

国家儿童医疗中心 NEXT 申请

• 批准号: 8241362
• 负责人: Roger Packer
• 金额: $ 34.4万
• 依托单位: CHILDREN'S RESEARCH INSTITUTE
• 依托单位国家: 美国
• 财政年份: 2011
• 资助国家: 美国
• 起止时间: 2011-09-30 至 2018-08-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/8241362
• 关键词: Adult; Affect; Agreement; Area; Biological Markers; Caring; Child; Clinical; Clinical Research; Clinical Trials; Core Facility; Department of Defense; Development; Developmental Disabilities; Diagnostic; Disease; Doctor of Philosophy; Drug Kinetics; Ensure; Epilepsy; Foundations; Funding; Generations; Genetic; Grant; Institution; Institutional Review Boards; Intellectual functioning disability; International; Leadership; Medical center; Molecular; Multi-Institutional Clinical Trial; Muscular Dystrophies; National Center for Research Resources; National Institute of Arthritis and Musculoskeletal and Skin Diseases; National Institute of Child Health and Human Development; Neurocutaneous Syndromes; Neurofibromatoses; Neurologic; Neurologist; Neuromuscular Diseases; Neurosciences; Neurosciences Research; Office Management; Patients; Pediatric Brain Tumor Consortium; Pediatric Hospitals; Pediatrics; Pharmacodynamics; Pharmacology; Rehabilitation Research; Rehabilitation therapy; Research; Research Infrastructure; Research Personnel; Resources; Series; Site; Support Groups; TNFRSF5 gene; Testing; Therapeutic; Toxicology; United States National Institutes of Health; Work; base; clinical infrastructure; design; experience; member; metropolitan; nervous system disorder; neuro-oncology; neuromuscular; programs; research study; urea cycle; young adult

DESCRIPTION (provided by applicant): The Children's National Medical Center (CNMC) will utilize its extensive experience in neuroscience clinical trial development, its proven ability to accrue patients on neurologic clinical trials, its large patient base, and its clinical trial infrastructure to participate in the Network of Excellence in Neuroscience Clinical Trials (NEXT). CNMC will collaborate with other investigators within NEXT, as it has done in other neurologic clinical trials consortia over the past decade, to develop and ensure timely completion of biologically based translational/clinical research studies in both children and adults. CNMC will leverage synergistic support for the NEXT consortium through a series of pre-existing NIH- and DOD-funded clinical infrastructure grants and programs, including the only NIH CTSA hosted site within a children's hospital. CNMC also has highly developed core facilities, including third-generation sequencing and molecular diagnostic capabilities, biomarker discovery and testing facilities, and pharmacokinetic/pharmacodynamic experience, which will be available for CNMC activities within NEXT. CNMC has a strong leadership team, led by Roger J. Packer, MD (PI) and Eric Hoffman, PhD, who are experienced clinical trialists in neuroscience research and have not only led clinical trials, but have been instrumental in the development of national and international clinical trial consortia (including those for neuromuscular diseases). A host of experienced child neurologists, neuro-intensivists, clinical trialists, molecular geneticists, expets in pharmacokinetics and pharmacodynamics, and neurogeneticists are in place at CNMC to participate in trials opened through NEXT. The institution has a large patient base in a multitude of different neurologic diseases from which to accrue patients on NEXT studies. In addition to the over 18,000 unique patients with neurologic conditions seen at CNMC each year, CNMC works closely with multiple foundations/support groups, which have pledged to support NEXT efforts and refer patients to CNMC for open trials. CNMC'S Clinical Research Management Office is designed specifically to support and facilitate clinical trial completion. CNMC already has mechanisms in place through the Institutional Review Board, including agreement to participate in a federated IRB, to facilitate trial approval. PUBLIC HEALTH RELEVANCE: The inclusion of CNMC within NEXT will provide NEXT with a site that has specialized expertise in diverse areas of neuroscience and can accrue significant numbers of patients. It will provide NEXT with an excellent site to carry out clinical trials in th DC metropolitan region, as well as providing NEXT with a pediatrics-focused resource experienced in developing neurologic consortia. CNMC also has highly evolved genetic and biomarker-discovery core facilities to support NEXT studies.

描述（由申请人提供）：儿童国家医学中心（CNMC）将利用其在神经科学临床试验开发中的丰富经验，其在神经系统临床试验中累积的患者的证明能力，大型患者基础以及其临床试验基础设施来参与神经科学临床临床试验的卓越网络（下一项）。 CNMC将与其他研究人员合作，就像在过去十年中其他神经系统临床试验联盟中所做的那样，以开发并确保及时完成儿童和成人的基于生物学的转化/临床研究。 CNMC将通过一系列已有的NIH和DOD资助的临床基础设施赠款和计划来利用对下一个财团的协同支持，其中包括唯一的NIH CTSA在儿童医院内托管了现场。 CNMC还拥有高度开发的核心设施，包括第三代测序和分子诊断能力，生物标志物的发现和测试设施以及药代动力学/药物动力学经验，下一步将用于CNMC活动。 CNMC拥有一支强大的领导团队，由医学博士Roger J. Packer（PI）和Eric Hoffman博士领导，他们是神经科学研究领域的临床试验者，不仅领导了临床试验，而且在国家和国际临床试验的发展中发挥了重要作用（包括用于神经肌肉疾病的疾病）。 CNMC有许多经验丰富的儿童神经病学家，神经培训学家，临床试验者，分子遗传学家，药代动力学和药效学方面的预期以及神经遗传学家在CNMC中都有参与接下来通过的试验。该机构在多种不同的神经系统疾病中拥有庞大的患者基础，下一步研究患者。除了每年在CNMC处看到的18,000多名具有神经系统疾病的独特患者外，CNMC还与多个基础/支持组密切合作，这些基础/支持组已承诺支持下一项工作，并将患者推荐给CNMC进行开放试验。 CNMC的临床研究管理办公室专门旨在支持和促进临床试验完成。 CNMC已经通过机构审查委员会已经建立了机制，包括同意参加联合IRB的协议，以促进审判批准。 公共卫生相关性：接下来将CNMC纳入下一步将提供一个在神经科学不同领域具有专业知识的网站，并且可以吸引大量患者。下一步将为在DC大都会地区进行临床试验的出色地点，并为下一步提供以儿科为重点的资源来开发神经系统财团。 CNMC还具有高度发展的遗传和生物标志物发现核心设施，以支持下一项研究。