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

项目摘要 神经科学临床试验卓越网络（NeuroNEXT）计划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，将神经科学的进步有效地转化为患有神经系统疾病的成人和儿童的治疗。基础神经科学的最新发现直接导致了对神经系统疾病的疾病过程的理解的重大突破，其中许多疾病给个人、家庭和社区带来了巨大的负担。然而，在多中心临床试验中测试这些新疗法存在一些组织和后勤挑战，包括监管和机构批准效率低下，招募和保留不同研究参与者的障碍，以及缺乏经过培训的多中心临床研究试验领导者。NeuroNEXT（NN）的中心目标是从最初的概念到最终的分析，通过提供有效的方法，组织，统计和后勤支持，促进高质量的早期临床试验和生物标志物多中心研究，并提供明确的去/不去决策。NeuroNEXT还将作为超罕见神经系统疾病基因治疗早期临床试验的载体。如果研究人群的性质（如罕见病）需要，将使用适应性2-3期试验和平台试验。 NeuroNEXT由25个临床研究中心、一个临床协调中心（CCC）和一个数据协调中心（DCC）组成。目标1是与DCC建立一个协作和可访问的CCC和临床研究网络，以有效地进行罕见和常见成人和儿童神经系统疾病的高质量2期临床试验，生物标志物研究和基因治疗试验。目标2是扩大有经验的临床研究者和研究人员的储备和多样性，他们准备成为多中心临床研究试验的领导者。目标3是与临床试验的关键利益相关者形成战略合作，包括政府组织，学术界，基金会，行业合作伙伴和以患者为中心的团体，通过NN组织高影响力的神经系统疾病临床试验。NeuroNEXT的创新将包括建立一个试验网络，需要使用一个单一的机构审查委员会，所有网络资助的研究的主合同流程，集中的药房和实验室检测，一个单一的数据安全监测委员会进行安全审查，广泛共享标准操作程序和知识，最先进的早期开发试验设计方法，以及使用共同数据元素进行数据收集。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，从而大大扩大了训练有素并准备领导多中心试验的经验丰富的临床研究者的人才库和多样性。将通过若干新的外联办法积极寻求新的协议。在NN的前11年里，有11项涉及与工业界、基金会、学术界和政府合作的研究得到了资助。CCC将扩大这些先前的成就，以进一步推进和加速神经系统疾病的治疗开发。

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.