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