Children's National Medical Center Application for NEXT

描述（由申请人提供）：国家儿童医学中心 (CNMC) 将利用其在神经科学临床试验开发方面的丰富经验、其在神经系统临床试验中招募患者的成熟能力、庞大的患者基础以及临床试验基础设施来参与神经科学临床试验卓越网络 (NEXT)。 CNMC 将与 NEXT 内的其他研究人员合作，就像过去十年在其他神经临床试验联盟中所做的那样，开发并确保及时完成针对儿童和成人的基于生物学的转化/临床研究。 CNMC 将通过一系列现有的 NIH 和 DOD 资助的临床基础设施拨款和计划，包括儿童医院内唯一的 NIH CTSA 托管站点，为 NEXT 联盟提供协同支持。 CNMC还拥有高度发达的核心设施，包括第三代测序和分子诊断能力、生物标志物发现和测试设施以及药代动力学/药效学经验，这些将可用于NEXT内的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经验丰富的临床试验专家，不仅领导过临床试验，而且在国家和国际临床试验联盟（包括神经肌肉疾病临床试验联盟）的发展中发挥了重要作用。 CNMC 拥有一批经验丰富的儿童神经科医生、神经重症监护医生、临床试验专家、分子遗传学家、药代动力学和药效学专家以及神经遗传学家，参与通过 NEXT 开展的试验。该机构在多种不同的神经系统疾病方面拥有大量患者基础，可以从中招募患者参加 NEXT 研究。除了 CNMC 每年收治超过 18,000 名患有神经系统疾病的独特患者外，CNMC 还与多个基金会/支持团体密切合作，这些基金会/支持团体已承诺支持 NEXT 工作并将患者转介至 CNMC 进行公开试验。 CNMC 的临床研究管理办公室专门为支持和促进临床试验的完成而设计。 CNMC 已经通过机构审查委员会建立了机制，包括同意参加联合 IRB，以促进试验批准。 公共卫生相关性：CNMC 纳入 NEXT 将为 NEXT 提供一个在神经科学不同领域拥有专业知识的网站，并且可以吸引大量患者。它将为 NEXT 提供一个在华盛顿特区开展临床试验的绝佳场所，并为 NEXT 提供在发展神经病学联盟方面经验丰富的儿科资源。 CNMC 还拥有高度进化的遗传和生物标志物发现核心设施来支持 NEXT 研究。

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.