Clinical Coordinating Center for the Network of Excellence in Neuroscience Clinical Trials (NEXT - CCC)

神经科学临床试验卓越网络临床协调中心 (NEXT - CCC)

• 批准号: 10741962
• 负责人: MERIT E CUDKOWICZ
• 金额: $ 283.53万
• 依托单位: MASSACHUSETTS GENERAL HOSPITAL
• 依托单位国家: 美国
• 财政年份: 2023
• 资助国家: 美国
• 起止时间: 2023-09-01 至 2028-08-31
• 项目状态: 未结题
• 来源: https://reporter.nih.gov/project-details/10741962
• 关键词: Academia; Acceleration; Achievement; Acute; Adult; Agreement; Area; Biological Markers; Bylaw; Child; Childhood; Chronic; Clinical; Clinical Investigator; Clinical Research; Clinical Trials; Clinical Trials Data Monitoring Committees; Clinical Trials Network; Collaborations; Common Data Element; Communication; Communities; Community Networks; Complement; Conceptions; Conduct Clinical Trials; Contracts; Data Collection; Data Coordinating Center; Decentralization; Development; Disease; Education; Ensure; Family; Foundations; Funding; Gene therapy trial; Goals; Government; Individual; Industry; Institution; Institutional Review Boards; Knowledge; Laboratories; Lead; Leadership; Link; Longevity; Methodology; Monitor; Multi-Institutional Clinical Trial; Multicenter Trials; Nature; Neurodevelopmental Disorder; Neurologic; Neurosciences; Participant; Patients; Periodicals; Persons; Pharmacy facility; Phase; Phase II Clinical Trials; Population; Privatization; Procedures; Process; Protocols documentation; Rare Diseases; Reporting; Research; Research Personnel; Safety; Site; Societies; Standardization; Teleconferences; Testing; Training; Training and Education; Translating; United States National Institutes of Health; Update; Work; biomarker validation; clinical biomarkers; clinical research site; clinical trial participant; cost; early phase clinical trial; equity, diversity, and inclusion; experience; gene therapy; gene therapy clinical trial; improved; industry partner; innovation; investigator training; nervous system disorder; novel; novel strategies; novel therapeutics; organizational structure; outreach; patient oriented; patient population; performance site; programs; protocol development; recruit; study population; symposium; therapeutic development; therapy development; trial design; Academia; Acceleration; Achievement; Acute; Adult; Agreement; Area; Biological Markers; Bylaw; Child; Childhood; Chronic; Clinical; Clinical Investigator; Clinical Research; Clinical Trials; Clinical Trials Data Monitoring Committees; Clinical Trials Network; Collaborations; Common Data Element; Communication; Communities; Community Networks; Complement; Conceptions; Conduct Clinical Trials; Contracts; Data Collection; Data Coordinating Center; Decentralization; Development; Disease; Education; Ensure; Family; Foundations; Funding; Gene therapy trial; Goals; Government; Individual; Industry; Institution; Institutional Review Boards; Knowledge; Laboratories; Lead; Leadership; Link; Longevity; Methodology; Monitor; Multi-Institutional Clinical Trial; Multicenter Trials; Nature; Neurodevelopmental Disorder; Neurologic; Neurosciences; Participant; Patients; Periodicals; Persons; Pharmacy facility; Phase; Phase II Clinical Trials; Population; Privatization; Procedures; Process; Protocols documentation; Rare Diseases; Reporting; Research; Research Personnel; Safety; Site; Societies; Standardization; Teleconferences; Testing; Training; Training and Education; Translating; United States National Institutes of Health; Update; Work; biomarker validation; clinical biomarkers; clinical research site; clinical trial participant; cost; early phase clinical trial; equity, diversity, and inclusion; experience; gene therapy; gene therapy clinical trial; improved; industry partner; innovation; investigator training; nervous system disorder; novel; novel strategies; novel therapeutics; organizational structure; outreach; patient oriented; patient population; performance site; programs; protocol development; recruit; study population; symposium; therapeutic development; therapy development; trial design

Project Summary The long-term objective of the Network of Excellence in Neuroscience Clinical Trials (NeuroNEXT) initiative is to efficiently translate advances in neuroscience into treatments for adults and children with neurological disorders through partnerships with government, academia, private foundations, and industry. Recent discoveries in basic neuroscience have directly led to major breakthroughs in the understanding of disease processes in neurological disorders, many of which impose an enormous burden on individuals, families, and communities. Yet there are several organizational and logistical challenges to testing these new therapies in multicenter clinical trials, including inefficiencies in regulatory and institutional approvals, barriers to recruitment and retention of diverse study participants, and a dearth of individuals trained to be the leaders of multicenter clinical research trials. A central goal of NeuroNEXT (NN) is to facilitate, from initial conception through final analysis, high-quality early phase clinical trials and biomarker multisite studies with clear go/no-go decisions by providing efficient methodological, organizational, statistical, and logistical support. NeuroNEXT will also serve as the vehicle for the conduct of early clinical trials of gene therapy for ultra-rare neurological disorders. If warranted by the nature of the studied population, such as rare diseases, adaptive phase 2-3 trials and platform trials will be used. NeuroNEXT is comprised of 25 clinical sites, a Clinical Coordinating Center (CCC), and a Data Coordinating Center (DCC). Aim 1 is to create a collaborative and accessible CCC and clinical research network with the DCC, to efficiently conduct high-quality Phase 2 clinical trials, biomarker studies, and gene therapy trials in rare and common adult and pediatric neurological disorders. Aim 2 is to expand the pool and diversity of experienced clinical Investigators and research staff who are prepared to be leaders of multicenter clinical research trials. Aim 3 is to form strategic collaborations with key stakeholders in clinical trials, including government organizations, academia, foundations, industry partners, and patient-centered groups to organize high-impact clinical trials through NN for neurological disorders. NeuroNEXT innovations will include establishing a trial network that requires the use of a single institutional review board, a master contract process for all network- funded studies, centralized pharmacy and laboratory testing, a single Data Safety Monitoring Board for safety review, broad sharing of Standard Operating Procedures and knowledge, state-of-the-art early development trial design approaches, and the use of the common data elements for data collection. Applicants who seek to use the network can be novice or experienced trialists, thereby greatly expanding the pool and diversity of experienced clinical investigators trained and prepared to lead multicenter trials. New protocols will be proactively sought through several new outreach approaches. Eleven studies involving partnerships with industry, foundations, academia, and the government were funded in the first 11 years NN. The CCC will expand on these prior achievements to further advance and accelerate therapeutic development for neurological disorders.

项目摘要 神经科学临床试验卓越网络（Neuronext）的长期目标是通过与政府，学术界，私人基金会和行业的合作伙伴关系，有效地将神经科学的进步转化为成人和神经疾病儿童的治疗方法。基本神经科学的最新发现直接导致了对神经系统疾病疾病过程的理解，其中许多人对个人，家庭和社区造成了巨大负担。然而，在多中心临床试验中测试这些新疗法的组织和后勤挑战，包括监管和机构批准的效率低下，招募和保留多样的研究参与者的障碍，以及被培训的个人缺乏成为多中心临床研究试验的领导者。 Neuronext（NN）的一个核心目标是从最初的概念到最终分析，高质量的早期临床试验和生物标志物多站点研究，并通过提供有效的方法论，组织，统计和后勤支持，从而具有明确的GO/NO-GO决策。 Neuronext还将作为进行超稀有神经系统疾病基因治疗的早期临床试验的载体。如果由研究人群的性质（例如罕见疾病）保证，则将使用自适应阶段2-3期试验和平台试验。 Neuronext由25个临床部位，一个临床协调中心（CCC）和数据协调中心（DCC）组成。 AIM 1是与DCC建立一个协作且可访问的CCC和临床研究网络，以有效地进行高质量的2期临床试验，生物标志物研究和基因治疗试验。目标2是扩大准备成为多中心临床研究试验领导者的经验丰富的临床研究人员和研究人员的池和多样性。 AIM 3是在临床试验中与主要利益相关者进行战略合作，包括政府组织，学术界，基金会，行业伙伴和以患者为中心的小组，以通过NN来组织神经系统疾病的高影响力临床试验。 Neuronext创新将包括建立一个试验网络，该试验网络需要使用单个机构审查委员会，所有网络资助研究的总合同流程，集中式药学和实验室测试，单个数据安全监控委员会，用于安全审查，标准操作程度和知识的广泛共享，最先进的早期开发试验方法以及用于数据元素的使用。寻求使用该网络的申请人可以是新手或经验丰富的试验者，从而大大扩大了经验丰富的临床调查人员的多样性，并准备领导多中心试验。新协议将通过几种新的外展方法积极寻求。在NN的头11年，资助了11项涉及与行业，基金会，学术界和政府合作的研究。 CCC将扩大这些先前的成就，以进一步促进和加速神经系统疾病的治疗发展。