Acute ischemic stroke (AIS) is the second leading cause of death and disability worldwide. While reperfusion therapies have advanced substantially, many patients—particularly in low- and middle-income countries—remain unable to access them, and even with successful reperfusion, outcomes are often suboptimal.
Oxidative stress plays a critical role in brain injury and persists for weeks and months after a ischemic stroke. Edaravone, a lipophilic free radical scavenger with high blood-brain barrier permeability, has been approved for AIS treatment in China and Japan. However, in previous trials edaravone was administrated intravenously within 48 hours after stroke onset for up to 14 days. Whether earlier initiation and prolonged edaravone treatment could yield better outcomes was unknown.
The ERASE-STROKE trial, a randomized, double-blind, placebo-controlled phase 3 study conducted at 40 sites in China, enrolled 614 AIS patients with disabling anterior circulation stroke of non-cardioembolic origin who did not receive reperfusion therapy. Patients were randomized within 24 hours of symptom onset to receive oral edaravone (TTYP01) 60 mg or placebo twice daily for 28 days .
Key findings:
Primary outcome (mRS 0–1 at 90 days): 65.4% in TTYP01 group vs. 47.1% in placebo group (OR 2.12, 95% CI 1.53–2.94, P <0.001).
Safety outcomes: No significant differences in symptomatic intracranial hemorrhage, recurrent stroke, or serious adverse events between groups.
The treatment effect was consistent across all predefined subgroups.
The study demonstrates that oral edaravone given early and for a prolonged duration is safe and significantly improves functional recovery in AIS patients who do not receive reperfusion therapy.
These findings support the neuroprotective properties of edaravone and offer a potential therapeutic option for the substantial population of stroke patients who cannot access reperfusion therapies. Further research is needed to confirm these results in broader populations and in conjunction with reperfusion therapy.
The trial is registered at ClinicalTrials.gov (NCT06648304) and the Chinese Drug Clinical Trial Registry (CTR20230707).
Science Bulletin
Randomized controlled/clinical trial