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

研究计划摘要 同种异体人骨髓间充质干细胞移植治疗左心发育不良综合征 (HLHS)：IIb期临床试验(ELPIS)，需要项目1的数据协调中心(DCC)) 协调，2)管理，3)数据管理，4)生物统计支持。Elpis的设计目的是 确定间充质干细胞(MSC)治疗II期HLHS患者的疗效 以舒张期质量衡量右室功能改善的手术 以心脏磁共振成像(CMRI)为索引。DCC对ELPIS的支持将通过以下方式完成 以下四个具体目标。具体目标1：提供项目协调：与临床医生协商 协调中心(CCC)PI和NHLBI，DCC将联合制定CCC、DCC和 临床中心衡量和指导所有ELPIS操作的熟练程度和进展 根据项目里程碑成功地设计、执行和完成研究。DCC还将 协调所有涉及CCC、NHLBI、数据和安全监控委员会(DSMB)以及临床的会议 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ELPI创建并分发一份政策手册，其中包含所有必要的 安全进行试验和指导互动的规则和标准操作程序(SOP) 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将制定一项 组织/沟通计划，将指导定期和频繁地审查研究目标(例如， 征聘和终点收集)、里程碑、时间表、研究和预算管理以及总体 策略。具体目标3：提供项目数据管理：数据中心将提供所有必要的统计数据 以及对CCC的后勤支持，因为这两个小组正在努力跟踪试验的进展。招聘 比率、不良事件、方案偏差、错过就诊、依从率、失访等 共同监督、集体讨论和共同努力执行的单一行动计划 符合NHLBI程序和政策。DCC将提供一个网站应用程序，其内容 与CCC的需求和要求保持一致。具体目标4：提供项目生物统计支助：DCC 将对最先进的开发和研究设计(特别是统计分析)做出贡献 对ELPIS的每一个申报终点进行统计评估。当DCC满足其要求时 与CCC合作，我们将能够回答这一重要的研究问题，以确定 MSC对主要终点的HLHS患者的疗效与这一患者群体的临床相关性。

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.