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、其在神经科学临床试验中吸引患者的公认能力、其庞大的患者基础以及其临床试验基础设施，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，就像它在过去十年中在其他神经学临床试验联盟中所做的那样，开发并确保及时完成儿童和成人的生物转化/临床研究。CNMC将通过一系列预先存在的NIH和国防部资助的临床基础设施赠款和项目，包括NIH CTSA在儿童医院内唯一托管的网站，为下一个财团提供协同支持。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、生物标记物发现和测试设施以及药代动力学/药效学经验，这些将在NEXT内提供给CNMC活动。CNMC拥有一个强大的领导团队，由罗杰·J·帕克医学博士(PI)和埃里克·霍夫曼博士领导，他们是神经科学研究方面经验丰富的临床试验专家，不仅领导过临床试验，而且在国内和国际临床试验财团(包括神经肌肉疾病的临床试验财团)的发展中发挥了重要作用。一大批经验丰富的儿童神经学家、神经强化专家、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都已在CNMC就位，参与到NEXT开始的试验。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，可以在下一步的研究中积累患者。除了每年在CNMC看到的18,000多名独特的神经系统疾病患者外，CNMC还与多个基金会/支持团体密切合作，这些基金会/支持团体承诺支持下一步的努力，并将患者转介给CNMC进行公开试验。CNMC的临床研究管理办公室是专门为支持和促进临床试验完成而设计的。CNMC已经通过机构审查委员会建立了机制，包括同意参与一个联合的IRB，以促进试验批准。

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.