Abstract TP320: A Randomized, Double-blind, Placebo-controlled, Phase I / IIa Clinical Trial for Evaluation of the Safety and Potential Therapeutic Effects After Intravenous Transplantation of Human Umbilical Cord-derived Mesenchymal Stem Cells in Patients With Cerebral Infarction

Abstract

Hypothesis: Cord has tremendous amount of stem cells that are easier and safer to obtain. The purpose of this study is to investigate the safety and the potential therapeutic effects per dose of human umbilical cord-derived mesenchymal stem cells(Cordstem-ST) IV transplantation in acute cerebral infarction. Methods: This study consist two treatment cohorts: Cohort 1 is one episode of IV Cordstem-ST 2.0 x 10^8 cells or placebo injection on day 0. Cohort 2 is two episodes of IV Cordstem-ST 2.0 x 10^8 cells or placebo injections on day 0 and day 7. Total 18 patients were enrolled and nine patients were allocated in each Cohort. Initially, we non-randomly non-blindly injected Cordstem-ST in initial 3 patients among 2 cohorts to investigate safety evaluation for KFDA requirement. And then we processed randomized, double-blind, placebo-controlled study in remaining 12 patients. Among 12 patients, the ratio of placebo and Cordstem-ST is 1:2 in each cohort. Patients were eligible for acute ischemic stroke patients within 7 days of stroke onset involving anterior circulation territory confirmed by the diffusion weighted MRI and NIHSS score between 5 and 20. Primary outcome measured by number of treatment related-adverse events during 6 months period. Secondary outcome measured improvement in clinical function as assessed by NIHSS, mRS, and BI compared to baseline at 6 months. Other studies including brain imaging (MR volumetry, tractogram, PET), immunochemistry (including biochemical markers), neuropsychological, and genetic studies were performed. Results: We finished this study for 18 patients. All patients had no serious adverse effects. Now, we are processing for rechecking final data and constructing about statistical analyzing methods before opening data randomization. Within 1-2 weeks, all data will be locked for blocking data correction. And then, randomization of data will be opening and we will evaluate primary and secondary outcomes according to schedule of pre-planned statistical analysis. We will present final results in 2018 ISC. Conclusions: We processed this study without any significant problems. Although, up to now, opening process of randomization has not been arrived, we are anticipating announcement of good results in 2018 ISC.