A Phase I, Open-Label, Dose-Escalation Study to Evaluate the Safety and Tolerability of Intravenous UMC119-06 in Patients with Acute Ischemic Stroke

This first-in-human, open-label, dose-escalation study (NCT04097652) evaluates UMC119-06, a mesenchymal stem cell product derived from human umbilical cord, for the treatment of acute ischemic stroke (AIS). The study includes two dosing cohorts: 1×10^6 cells/kg and 5×10^6 cells/kg, with three participants enrolled in each cohort. The primary objective is to assess the safety, tolerability, and maximum feasible dose of UMC119-06 in AIS patients, while the secondary objective focuses on evaluating long-term safety and clinical efficacy following a single administration. The study conducted safety assessments across both dosing cohorts, demonstrating that UMC119-06 is well-tolerated, with no adverse events (AEs) classified as possibly, probably, or definitely related to the product. Despite the small sample size of six patients, trends toward positive outcomes were observed in the modified Rankin Scale, the National Institutes of Health Stroke Scale, the Barthel Index, and the infarct volume on brain magnetic resonance imaging. Additionally, changes in biomarkers such as IL-6, TNF-α, VEGF, and HGF suggest a potential impact of UMC119-06 on inflammation, angiogenesis, and tissue repair processes. This Phase I study provides preliminary evidence regarding the safety and potential efficacy of UMC119-06 in AIS patients. Larger-scale studies with control groups are warranted to validate these findings and further explore the therapeutic potential of UMC119-06 for AIS.