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

左心发育不全综合征患者注射同种异体人间充质干细胞 (MSC)：IIb 期临床试验 (ELPIS)

• 批准号: 10053591
• 负责人: BARRY R DAVIS
• 金额: $ 33.57万
• 依托单位: UNIVERSITY OF TEXAS HLTH SCI CTR HOUSTON
• 依托单位国家: 美国
• 财政年份: 2020
• 资助国家: 美国
• 起止时间: 2020-09-15 至 2025-08-31
• 项目状态: 未结题
• 来源: https://reporter.nih.gov/project-details/10053591
• 关键词: Adherence; Adverse event; Allogenic; Anastomosis - action; Ancillary Study; Annual Reports; Big Data Methods; Biometry; Budgets; Cardiac; Case Report Form; Certification; Child; Clinical; Clinical Management; Clinical Trials; Clinical Trials Data Monitoring Committees; Collaborations; Collection; Common Data Element; Communication; Communities; Consultations; Data; Data Analytics; Data Collection; Data Coordinating Center; Data Set; Databases; Development; Documentation; Enrollment; Ensure; Evaluation; Event; Fontan Procedure; Goals; Human; Hypoplastic Left Heart Syndrome; Infant; Information Dissemination; Injections; Institutional Review Boards; Insurance; Intelligence; Laboratories; Licensing; Logistics; Magnetic Resonance Imaging; Manuals; Measures; Mesenchymal Stem Cells; Monitor; National Heart, Lung, and Blood Institute; Participant; Patient Care; Patients; Performance; Phase; Policies; Procedures; Process; Protocols documentation; Publications; Quality Control; Reporting; Research; Research Design; Research Personnel; Right Ventricular Function; Safety; Schedule; Secure; Statistical Data Interpretation; System; Time; TimeLine; Validation; Ventricular; Voting; Work; adherence rate; adverse event monitoring; authority; base; clinical center; clinically relevant; data management; data warehouse; design; electronic data; electronic data capture system; follow-up; improved; indexing; meetings; operation; patient population; payment; primary endpoint; recruit; stem cell therapy; symposium; visit adherence; web site; working group

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.

研究计划摘要 同种异体人间充质干细胞 (MSC) 注射治疗左心发育不全综合征患者 (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 旨在 确定间充质干细胞 (MSC) 治疗对 II 期 HLHS 患者的疗效 通过舒张期质量来确定右心室功能的改善情况来进行手术 心脏磁共振成像（CMRI）索引。 DCC 对 ELPIS 的支持将通过 以下四个具体目标。具体目标 1：提供项目协调：与临床咨询 协调中心 (CCC) PI 和 NHLBI，DCC 将共同制定 CCC、DCC 和 临床中心衡量和指导所有 ELPIS 操作的熟练程度和进度 根据项目里程碑，研究的成功设计、执行和完成。 DCC还将 协调涉及 CCC、NHLBI、数据和安全监测委员会 (DSMB) 以及临床试验的所有会议 中心，包括但不限于治理。 （指导/执行）、辅助/子研究、终点、 运营、出版物和遵守小组委员会。具体目标 2：提供项目管理： DCC 与 CCC 合作，将为 ELPIS 创建并分发一份政策手册，其中包含所有必需的内容 用于安全进行试验并指导互动的规则和标准操作程序 (SOP) DCC 和 CCC 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地审查学习目标（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 两个小组努力跟踪试验进展，并为 CCC 提供后勤支持。招聘 率、不良事件、方案偏差、漏诊、依从率、失访等将 双方共同监督、集体讨论并认真执行单一行动计划 符合 NHLBI 程序和政策。 DCC 将提供一个网站应用程序，其内容 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 将为最先进的开发和研究设计（特别是统计分析）做出贡献 对每个声明的 ELPIS 终点的统计评估。当 DCC 满足其要求时 与 CCC 合作，我们将能够回答这个重要的研究问题，以确定 MSC 对 HLHS 患者的疗效作为主要终点，与该患者群体具有临床相关性。