Clinical Translational Core (CTC)

临床转化核心（CTC）

基本信息

批准号：
10239465
负责人：
MUSTAFA SAHIN
金额：
$ 22.86万
依托单位：
BOSTON CHILDREN'S HOSPITAL
依托单位国家：
美国
项目类别：
财政年份：
2021
资助国家：
美国
起止时间：
2021-07-22 至 2026-05-31
项目状态：
未结题

来源：
https://reporter.nih.gov/project-details/10239465
关键词：
Adaptive Behaviors Animal Behavior Area Basic Science Behavioral Biological Biological Assay Biological Markers Biology Biometry Blood Blood specimen Boston Cell Line Cells Child Care Child Psychiatry Clinic Clinical Clinical Research Clinical Trials Clinical Trials Design Cognitive Collaborations Collection Complex Consultations DNA Data Analyses Development Diagnosis Discipline Discrimination Disease Down Syndrome Drug Screening Electroencephalography Ensure Environment Equipment Event-Related Potentials Face Family Fibroblasts Fostering Fragile X Syndrome Future Generations Genetic Goals Growth Human Human Resources Image Improve Access Individual Infrastructure Institutional Policy Intellectual and Developmental Disabilities Research Centers Intellectual functioning disability Intervention Intervention Trial Language Mus Natural History Neurodevelopmental Disorder Neurofibromatoses Neuronal Differentiation Neurons Neurophysiology - biologic function Neuropsychological Tests Neuropsychology Neurosciences Parents Participant Patient Care Patient Recruitments Patients Pediatric Hospitals Pediatric Neurology Phenotype Physiological Physiology Population Positioning Attribute Pre-Clinical Model Preparation Process Registries Research Research Personnel Research Support Resources Rett Syndrome Sampling Scientist Services Siblings Technical Expertise Testing Therapeutic Tissues Training Translating Translational Research Tuberous sclerosis protein complex Work base bench to bedside biobank biomarker development biomarker performance cellular imaging clinical care clinical phenotype cohort community engagement density design genetic analysis high throughput screening human disease induced pluripotent stem cell mouse model multidisciplinary neurobehavioral neurophysiology next generation novel novel diagnostics novel therapeutic intervention patient advocacy group pre-clinical pre-clinical research programs protocol development rare genetic disorder screening social success tool translational neuroscience translational study treatment trial

项目摘要

CLINICAL TRANSLATIONAL CORE ABSTRACT The objectives of the Clinical Translational Core (CTC) of our IDDRC are to accelerate the translation of research discoveries into new treatments for neurodevelopmental disorders, through collaboration with basic scientists and clinicians, as well as to train future leaders in translational neuroscience. Services offered by the Clinical Translational Core to support translational work include three sub-cores: 1) Patient-based Preclinical Services, comprised of a Biorepository and the induced pluripotent stem cell (iPSC) based Human Neuron Unit, 2) Clinical Phenotyping Services, consisting of the Neurobehavioral Unit and the Neurophysiology Unit, and 3) Clinical Trial and Regulatory Services, containing the Regulatory Unit and Research Participant Registry. The suite of services offered by the CTC enables investigators to pursue an integrated approach to therapeutic discovery for neurodevelopmental disorders, from patient derived samples in preclinical research to biomarker development to natural history and interventional clinical trials. Sub-core 1 - Patient Based Preclinical Services - maintains the regulatory approval to collect patient samples that can be used for primary research or for the generation of iPSC lines and neuronal differentiation within the Human Neuron Unit. Sub- core 2 - Clinical Phenotyping Services - provides expertise and access to high density EEG equipment as well as direct phenotyping through neuropsychological assessment and neurophysiological recording. Sub-core 3 - Clinical Trial and Regulatory Services - provides expert consultation in the regulatory framework supporting clinical research which enables both the services provided within the Core and IDDRC investigators of all backgrounds. To facilitate engagement in clinical research by basic science researchers and young investigators, the sub-core assists in the preparation of regulatory documents, the training of study staff, patient recruitment and data analysis. Patient recruitment is supported by the Research Participant Registry, a collection of over 30,000 individuals willing to participate in clinical research, including patients with a myriad of neurodevelopmental diagnoses, their parents and siblings, and typically developing controls. To ensure that the efforts of the Core are focused in areas that will produce meaningful benefits for the intellectual and developmental disability population, the Core is strongly connected to clinical care centers and patient advocacy groups, both of which advise the activity and development of the group. Through these efforts, the Core has a proven record of success in designing and launching preclinical translational and clinical projects for IDDRC investigators.

临床翻译核心抽象的我们IDDRC的临床翻译核心（CTC）的目标是加速翻译通过与基本的合作，研究神经发育障碍的新疗法的研究发现科学家和临床医生，以及培训未来的转化神经科学领导者。由支持翻译工作的临床翻译核心包括三个子核：1）基于患者的临床前由生物座和诱导多能干细胞（IPSC）的人类神经元组成的服务单位，2）临床表型服务，由神经行为单元和神经生理单元组成， 3）包含监管单位和研究参与者的临床试验和监管服务注册表。 CTC提供的服务套件使调查人员能够采取综合方法神经发育障碍的治疗发现，从临床前研究中的患者衍生样品到生物标志物开发自然史和介入的临床试验。亚核1-基于患者临床前服务 - 维持监管部门的批准，以收集可用于主要的患者样本研究或为人类神经元单元内的IPSC线和神经元分化的产生。亚核心2-临床表型服务 - 还提供专业知识和访问高密度的脑电图设备通过神经心理学评估和神经生理记录进行直接表型。子核3- 临床试验和监管服务 - 在支持的监管框架中提供专家咨询临床研究使所有人的核心和IDDRC调查人员提供了两个服务背景。促进基础科学研究人员和年轻人参与临床研究调查人员，亚核心协助制备监管文件，学习人员的培训，患者招聘和数据分析。患者招聘得到了研究参与者注册表的支持，收集了30,000多名愿意参加临床研究的人，包括无数的患者神经发育诊断，他们的父母和兄弟姐妹，通常会发展控制。确保这一点核心的努力集中在将为知识分子带来有意义利益的领域和发育障碍人群，核心与临床护理中心和患者密切相关倡导团体，这两者都建议该小组的活动和发展。通过这些努力，核心在设计和启动临床前翻译和临床项目方面取得了成功的记录用于IDDRC调查人员。