Early and prolonged oral edaravone for neuroprotection in acute ischemic stroke: the ERASE-STROKE randomized controlled phase 3 trial

Abstract

The optimal timing and duration of neuroprotective therapy for acute ischemic stroke (AIS) remain unclear. Previous studies showed benefits of edaravone treatment when started within 48 h of AIS onset for 14 d, but it remains unknown whether earlier initiation (within 24 h of onset) and longer treatment (28 d) can yield better outcomes. In ERASE-STROKE, oral edaravone (TTYP01) was demonstrated to improve functional outcomes in patients with AIS. Here, to evaluate the neuroprotective effects of TTYP01, a phase 3, multicenter, double-blind trial was conducted among patients with disabling anterior circulation stroke who did not undergo reperfusion therapy. Within 24 h of symptom onset, 614 eligible patients were randomly assigned to receive TTYP01 60 mg or placebo twice daily for 28 d plus standard care. The primary endpoint was excellent functional outcome (mRS score 0–1) at 90 d. Among 614 patients in the intention-to-treat population, excellent outcome was achieved in 65.4% with TTYP01 vs. 47.1% with placebo (odds ratio 2.12; 95% CI 1.53–2.94; P < 0.001). Rates of symptomatic intracranial hemorrhage, mortality, and serious adverse events did not differ significantly. In patients with disabling anterior circulation stroke not receiving reperfusion, 28-d TTYP01 is safe and improves functional recovery. ClinicalTrials.gov: NCT06648304; Chinese Drug Clinical Trial Registry: CTR20230707.