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、私人基金会和工业界的合作，有效地将神经科学的进步转化为治疗患有神经性疾病的成人和儿童。基础神经科学的最新发现直接导致了对神经系统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几个组织和后勤挑战，包括监管和机构批准效率低下，招募和留住不同研究参与者的障碍，以及缺乏受过培训成为多中心临床研究试验领导者的个人。NeuroNEXT(NN)的一个中心目标是通过提供高效的方法、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记物多点研究，通过提供有效的方法学、组织、统计和后勤支持，作出明确的通过/不通过的决定。NeuroNEXT还将作为进行超罕见神经疾病基因治疗的早期临床试验的载体。如果被研究人群的性质所保证，如罕见疾病，将使用适应性阶段2-3试验和平台试验。NeuroNEXT由25个临床站点、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创建一个协作和可访问的CCC和临床研究网络，以有效地在罕见和常见的成人和儿童神经疾病中进行高质量的第二阶段临床试验、生物标记物研究和基因治疗试验。目标2是扩大有经验的临床研究人员和研究人员的人才库和多样性，他们准备成为多中心临床研究试验的领导者。目标3是与临床试验中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组织神经疾病的高影响临床试验。NeuroNEXT的创新将包括建立一个试验网络，该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都需要一个主合同程序，集中的药房和实验室测试，一个用于安全审查的单一数据安全监测委员会，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，以及使用公共数据元素进行数据收集。寻求使用该网络的申请者可以是新手或经验丰富的试验者，从而极大地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人员的池和多样性。将通过几种新的外联办法积极寻求新的议定书。在NN的头11年里，有11项研究涉及到与产业界、基金会、学术界和政府的伙伴关系。CCC将在这些先前成果的基础上进一步推进和加快神经疾病的治疗开发。

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.