Work

Safety and Feasibility of YC‐6 in Patients With Large Hemispheric Infarction: A Randomized Clinical Trial

Year: 2025

Type: article

Study designs: Randomized Controlled Trial, Clinical Trial

Abstract: Background Malignant cerebral edema is a critical cause of poor outcomes in large hemispheric infarction and novel neuroprotective agents are urgently needed. We aimed to assess the safety and preliminarily explored the feasibility of YC‐6 (5α‐androst‐3β,5,6β‐triol for injection) in large hemispheric infarction. Methods This study was a prospective, randomized, double‐blind, placebo‐controlled, proof‐of‐concept trial. Patients with large hemispheric infarction in middle cerebral artery within 12 hours of symptom onset were enrolled. Patients were assigned to receive either 300 mg YC‐6 or placebo twice a day for 10 days, randomly. The primary safety outcomes were serious adverse events and all‐cause death. The primary efficacy outcome was the distribution of the modified Rankin Scale score at 90 days. Results The incidence of serious adverse events (52.2% and 43.5%) and all‐cause death (39.1% and 40.0%) within 90 days were similar between YC‐6 and placebo group. There was no statistical difference between the groups in the distribution of modified Rankin Scale score (common odds ratio 0.73, 95% CI: 0.25 to 2.18). The proportion of a modified Rankin Scale score of 0 to 4 was 56.5% in YC‐6 group compared with 35.0% in placebo group ( P =0.16). In addition, YC‐6 treatment numerically alleviated edema volume at 72 hours and 10 days (RD −15.02, 95% CI: −40.94 to 10.90; RD −10.35, 95% CI: −46.69 to 25.98) and decreased midline shift at 10 days (RD −0.6, 95% CI: −4.17 to 2.97). Conclusions This study preliminarily explored the safety and feasibility of YC‐6 in patients with large hemispheric infarction. Based on this trial, the phase 2 trial of YC‐6 was worthy of anticipation. Registration http://www.chinadrugtrials.org.cn ; Unique identifier: CTR20192127; http://www.chictr.org.cn ; Unique identifier: ChiCTR2400088887.

Source: Journal of the American Heart Association

DOI: 10.1161/jaha.124.039157