Pilot Core

试点和可行性计划 摘要 试点和可行性计划将是儿科卓越中心的核心和凝聚力部分。这个 试点和可行性方案的总体目标是发展需要获得 初步数据为未来肾脏疾病儿童临床试验的设计提供信息。飞行员和 可行性计划将与管理核心、设计/分析核心、临床 表型核心和学习健康系统核心，以宣传、吸引、审查和资助提案。这个 试点和可行性计划将促进与儿童医院以外的研究人员的合作 费城和我们在约翰·霍普金斯大学的地区学术合作伙伴。我们将资助2-4个试点项目 在获奖期间，每个项目每年获得50,000美元的直接支持，最多两个 好几年了。至少有一项提案将从研究基地以外的地方获得资金。此外，该部还 费城儿童医院的儿科已经慷慨地同意资助一项额外的试验性研究，费用为5万美元 每年，为期两年。所有试点和可行性项目都将通过同行评审进行有价值的选择 科学咨询委员会。试点和可行性计划将提供指导，通过形成 小组委员会，为那些在整个提交过程中需要对其研究项目提供投入的调查人员提供 和审查过程。为实现以下具体目标和完成本研究所的总体研究目标 儿科英才中心，每个试点和可行性研究都将利用AT提供的资源 中心的三个核心中至少有一个。试点和可行性方案的具体目标是：1)刺激 并征集新的项目想法，以支持儿科卓越中心的总体研究目标-即 减少对肾病儿童进行临床试验的障碍；2)鼓励 初级调查员提交试点项目提案，并指导这些调查员修订 根据需要审查他们的建议；3)审查提交的试点项目的科学价值、创新水平、 以及与儿科卓越中心的总体研究目标和三个核心的凝聚力；以及4) 推动发展新的研究方向，获得初步数据，这些数据将成为 申请未来临床试验的独立研究支持。试点和可行性计划将建立 根据费城儿童医院强大的儿科肾病研究经验， 来自宾夕法尼亚大学更广泛的当地科学界的杰出人才，以及地区 约翰霍普金斯大学提供的专业知识。我们预计选定的试点项目将提供 调查人员有机会生成初步数据，这些数据将成为未来有竞争力的资金的基础 旨在预防、治疗或减缓儿童肾脏疾病进展的临床试验中的建议，以 为未来的治疗确定新的靶点，并更好地了解肾脏疾病的机制。 1

PILOT AND FEASIBILITY PROGRAM ABSTRACT The Pilot and Feasibility program will be a central and cohesive piece of the Pediatric Center of Excellence. The overall goal of the Pilot and Feasibility Program is to develop the science of investigators who need to obtain preliminary data to inform the design of future clinical trials in children with kidney disease. The Pilot and Feasibility Program will work closely with the Administrative Core, the Design/Analysis Core, the Clinical Phenotyping Core, and the Learning Health System Core to publicize, elicit, review, and fund proposals. The Pilot and Feasibility program will promote collaboration with investigators outside The Children's Hospital of Philadelphia and our regional academic partners at Johns Hopkins University. We will fund 2-4 pilot projects during the award period, with each project receiving $50,000 in direct support per year for a maximum of two years. At least one proposal will be funded from outside the research base. Additionally, the Department of Pediatrics at the Children’s Hospital of Philadelphia has generously agreed to fund an extra Pilot study at $50,000 per year for two years. All pilot and feasibility projects will be chosen meritoriously through peer review by a Scientific Advisory Committee. The Pilot and Feasibility Program will offer mentoring, through the formation of sub-committees, for those investigators who need input on their research projects throughout the submission and review process. To achieve the following specific aims and accomplish the overall research goals of the Pediatric Center of Excellence, each Pilot and Feasibility study will make use of the resources provided by at least one of the Center’s three Cores. The Specific Aims of the Pilot and Feasibility Program are: 1) To stimulate and solicit new project ideas that support the overall research goal of the Pediatric Center of Excellence–namely to decrease the barriers to implementing clinical trials in children with kidney disease; 2) To encourage submission of pilot project proposals by junior investigators and to mentor these investigators in the revision of their proposals, as needed; 3) To review pilot project submissions for their scientific merit, level of innovation, and cohesion with the overall research goals and three Cores of the Pediatric Center of Excellence; and 4) To promote the development of new research directions, acquiring preliminary data that will be the foundation for applications for future independent research support in clinical trials. The Pilot and Feasibility Program will build upon the strong pediatric nephrology research experience at the Children’s Hospital of Philadelphia, the outstanding talent from the broader local scientific community at the University of Pennsylvania, and the regional expertise offered by Johns Hopkins University. We anticipate that the selected pilot projects will provide investigators the opportunity to generate preliminary data that will form the basis for future, competitive funding proposals in clinical trials designed to prevent, treat, or slow the progression of kidney disease in children, to identify novel targets for future therapies, and to better understanding the mechanisms of kidney disease. 1