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

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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.