Presenter of 1 Presentation
SAFETY AND EFFICACY OF LT3001 IN PATIENTS WITH ACUTE ISCHEMIC STROKE WITHIN 24 HOURS AFTER SYMPTOMS ONSET: A PHASE 2, MULTI-CENTER, RANDOMIZED, DOUBLE-BLIND, PLACEBO-CONTROLLED STUDY
Abstract
Background and Aims
LT3001, a novel small molecule, designed to simultaneously act as a thrombolytic and reduce reperfusion injury, has been shown safe and efficacious in multiple animal stroke models. Here we assessed the safety of LT3001 in acute ischemic stroke patients within 24 hours after symptoms onset.
Methods
Eligible patients were last-known-normal ≤24h, had a NIHSS 4-30 and were not treated with IV-tPA and/or endovascular thrombectomy. Participants were 2:1 randomly assigned to receive a single dose of LT3001 or placebo. The primary outcome was the sICH rate ≤36h. Secondary outcomes included neurological/ functional outcome measures, mortality and other adverse events by Day-90 (NCT04091945).
Results
Twenty-four participants (16 in LT3001 group and 8 in placebo group) were enrolled at 10 centers in the US and Taiwan. Participants in the LT3001 and placebo groups were age of 62±13 and 68±9 years with median NIHSS of 6 (Range 4-24) and 5 (Range 4-17), respectively. Median times from onset-to-treatment were 21-hours for LT3001 and 18.5-hours for placebo. No sICH occurred and other safety measures appeared comparable between groups. Excellent functional outcome (90-day mRS(0-1)) was achieved in 3 (21%) LT3001 and 1 (14%) placebo-treated participants. Major neurological improvement (≥4-points NIHSS improvement from baseline to Day-30) occurred in 7 (47%) LT3001 and 1 (14%) placebo-treated participants. Among the 9 participants with baseline NIHSS≥6 treated with LT3001, 7 (78%) showed major neurological improvement.
Conclusions
Treatment with LT3001 within 24 hours after ischemic stroke onset appears to be safe and may be associated with better neurological and functional outcomes.