Minocycline for acute basilar artery occlusion stroke undergoing endovascular treatment: a randomized, evaluator-blinded, open-label, pilot trial

Abstract Background Minocycline is a promising anti-inflammatory agent for reducing futile recanalization, but whether it improves clinical outcomes in basilar-artery stroke patients treated with endovascular therapy (EVT) remains unclear. In this study, we aimed to assess the efficacy and safety of oral minocycline combined with EVT on outcomes in patients who had basilar-artery stroke. Methods The Minocycline for acute Ischemic Stroke undergoing endovascular Treatment due to Basilar artery occlusion (MIST-B) trial was a multicenter, randomized, evaluator-blinded, open-label, pilot trial in 12 Chinese hospitals. Adults with basilar-artery stroke no more than 24 h after onset were randomized (1:1) to receive oral minocycline (200 mg loading, then 100 mg every 12 h for 5 days) plus EVT or EVT alone. The primary efficacy outcome was the expanded National Institutes of Health Stroke Scale (e-NIHSS) score at day 5; the primary safety outcome was symptomatic intracranial hemorrhage (sICH) within 24 h. Participants were followed up 90 days after randomization. Modified intention-to-treat analysis examined treatment effect for the primary efficacy outcome using linear mixed models, with the baseline measurement of e-NIHSS score as a covariate. Results From March 4, 2023, to March 4, 2025, 90 patients were randomized, and 89 were analyzed (mean age 61.7 years, 31.5% women). At day 5, median e-NIHSS was 12.0 (IQR 3.5 to 30.0) in the minocycline group and 10.5 (IQR 2.0 to 35.0) in the control group; corresponding mean values were 15.0 (SD 13.5) and 17.2 (SD 16.9), respectively (adjusted mean difference − 2.83 [95% CI -7.86 to 2.20]; P = 0.27). sICH within 24 h occurred in 2/37 (5.4%) of participants in the minocycline group vs. 6/41 (14.6%) in the control group (adjusted risk ratio, 0.70; 95% CI, 0.11 to 4.67). Other secondary efficacy and safety outcomes, including 90-day modified Rankin Scale, mortality, and adverse events, showed no significant differences between groups. Conclusions Minocycline was well-tolerated in this population; however, these results did not provide evidence to support prescribing minocycline to improve early neurological outcomes. Larger trials of minocycline with extended treatment duration are warranted. Registration The trial was registered at ClinicalTrials.gov (NCT05512910) on August 23, 2022.