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

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.

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.

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).

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.

http://www.chinadrugtrials.org.cn; Unique identifier: CTR20192127; http://www.chictr.org.cn; Unique identifier: ChiCTR2400088887.