The Allogeneic Human Mesenchymal Stem Cell (MSCs) injection in Patients with Hypoplastic Left Heart Sydrome: A Phase IIb Clinical Trial (ELPIS)

研究摘要 异基因人骨髓间充质干细胞注射治疗左心发育不良综合征 （HLHS）：IIb期临床试验（ELPIS），项目1需要数据协调中心（DCC） 协调，2）管理，3）数据管理和4）生物统计支持。ELPIS旨在 确定间充质干细胞（MSC）治疗经历II期HLHS患者的疗效 通过测定舒张期质量来确定右心室功能改善的手术 心脏磁共振成像（CMRI）。发展协调委员会将通过以下方式支持电子学习方案信息系统： 以下四个具体目标。具体目标1：提供项目协调：与临床 协调中心（CCC）PI和NHLBI，DCC将联合制定CCC、DCC和 临床中心，以衡量和指导所有ELPIS操作所需的熟练程度和进展， 研究的成功设计，执行，并根据项目里程碑完成。DCC还将 协调所有涉及CCC、NHLBI、数据和安全性监测委员会（DSMB）以及临床 中心，包括但不限于治理。（指导/执行），辅助/子研究，终点， 运营、出版和遵守小组委员会。具体目标2：提供项目管理： DCC将与CCC合作，为ELPIS创建并分发一份政策手册，其中包含所有必要的 确保试验安全进行并指导相互作用的规则和标准操作规程（SOP） 发展协调委员会和协调委员会之间的关系，以及这些机构、指导委员会、 执行委员会、临床中心和核心实验室。此外，DCC将开发一个 组织/沟通计划，将指导定期和频繁审查研究目标（例如， 招募和终点收集）、里程碑、时间表、研究和预算管理以及总体 战略具体目标3：提供项目数据管理：DCC将提供所有必要的统计数据 和后勤支持的CCC作为两个小组的工作，以跟踪试验的进展。招聘 发生率、不良事件、方案偏离、错过访视、依从率、失访等， 联合监测，集体讨论，并在双方努力执行的单一行动计划， 根据NHLBI的程序和政策。DCC将提供一个网站应用程序，其内容 符合CCC的需求和要求。具体目标4：提供项目生物统计支持：DCC 将以最新技术水平为开发和研究设计（特别是统计分析）做出贡献 对ELPIS的每个声明终点进行统计评价。当DCC满足其要求时， 通过与CCC的合作，我们将能够回答这个重要的研究问题，以确定 MSC对HLHS患者的疗效与该患者人群临床相关的主要终点。

ABSTRACT OF RESEARCH PLAN Allogeneic Human MEsenchymal Stem Cell (MSC) Injection in Patients with Hypoplastic Left Heart Syndrome (HLHS): a Phase IIb Clinical Trial (ELPIS), requires a Data Coordinating Center (DCC) for project 1) coordination, 2) administration, 3) data management, and 4) biostatistical support. ELPIS is designed to determine the efficacy of mesenchymal stem cell (MSC) treatment in HLHS patients undergoing the stage II operation by determining the improvement of right ventricular function as measured by the diastolic mass indexed on cardiac magnetic resonance imaging (CMRI). DCC support of ELPIS will be accomplished through the following four specific aims. Specific Aim 1: Provide Project Coordination: In consultation with the Clinical Coordinating Center (CCC) PI and NHLBI, the DCC will jointly develop metrics for the CCC, DCC and clinical centers to gauge and direct the proficiency and progress of all ELPIS operations required for the study's successful design, execution, and completion according to project milestones. DCC will also coordinate all meetings involving CCC, NHLBI, Data and safety Monitoring Board (DSMB), and the clinical centers, including but not restricted to governance. (Steering/Executive), ancillary/substudy, endpoints, operations, publications, and adherence subcommittees. Specific Aim 2: Provide Project Administration: The DCC, in alliance with the CCC, will create and distribute a policy manual for ELPIS containing all requisite rules and Standard Operating Procedures (SOPs) for the safe conduct of the trial and to guide interaction between the DCC and CCC, as well as the relationships between these bodies, the Steering Committee, the Executive Committee, the clinical centers and the core laboratories. In addition the DCC will develop an organization/communication plan that will guide the regular and frequent review of study goals (e.g., recruitment and endpoint collection), milestones, timelines, study and budget management, and overall strategy. Specific Aim 3: Provide Project Data Management: The DCC will provide all necessary statistical and logistical support for the CCC as the two groups work to follow the progress of the trial. Recruitment rates, adverse events, protocol deviations, missed visits, adherence rates, loss to follow-up, etc. will be jointly monitored, collectively discussed, and a single plan of action diligently prosecuted by both in accordance with NHLBI procedures and policies. The DCC will provide a website application whose content aligns with the CCC needs and requirements. Specific Aim 4: Provide Project Biostatistical Support: The DCC will contribute to the development and study design (particularly statistical analysis) with state of the art statistical evaluations for each of the declared endpoints of ELPIS. When the DCC fulfills its requirements in collaboration with the CCC, we will be able to answer this important research question to determine the efficacy of MSC's for HLHS patients for a primary endpoint with clinical relevance to this patient population.