ABSTRACT NUMBER: ESOC2026OT115 CLINICAL SAFETY AND EFFICACY OF ALLOGENIC HUMAN INDUCED PLURIPOTENT STEM CELLS-DERIVED EXOSOMES IN PATIENTS WITH ACUTE ISCHEMIC STROKE

Abstract Background and aims Cell-based therapies represent a promising therapeutic strategy for enhancing neurologic recovery after stroke. Preclinical studies have demonstrated the efficacy of stem cell-derived exosomes in animal models of ischemic stroke. Methods We aim to evaluate the safety and efficacy of allogeneic human induced pluripotent stem cell (iPSC)-derived exosomes in patients with acute ischemic stroke. Results This is a multicenter, prospective, phase I/II clinical trial conducted in China between October 2023 and June 2026. Eligible participants will be patients with a diagnosis of cortical ischemic stroke, a baseline National Institutes of Health Stroke Scale (NIHSS) score of 6-20, and enrollment within 1-7 days after symptom onset. Stage 1 is a dose-escalation study including three different dose levels (n=3 per dose). Stage 2 is an expanded safety and preliminary efficacy phase with a randomized, double-blind, placebo-controlled design. In Stage 2, 20 patients will be randomized 1:1 to receive the highest safe dose identified in Stage 1 or placebo. Conclusions The primary endpoint is the incidence of serious adverse events (SAEs) within 90 days. Secondary endpoints include the proportion of modified Rankin Scale (mRS) score of 0-2 at 90 days, infarct volume, degree of cerebral edema, and single-cell RNA sequencing of peripheral blood. Conflict of interest