Efficacy and Safety of Mildronate for Acute Ischemic Stroke: A Randomized, Double-Blind, Active-Controlled Phase II Multicenter Trial

Background and Objective Mildronate, an inhibitor of carnitine-dependent metabolism, is considered to be an anti-ischemic drug. This study is designed to evaluate the efficacy and safety of mildronate injection in treating acute ischemic stroke. Methods We performed a randomized, double-blind, multicenter clinical study of mildronate injection for treating acute cerebral infarction. 113 patients in the experimental group received mildronate injection, and 114 patients in the active-control group received cinepazide injection. In addition, both groups were given aspirin as a basic treatment. Modified Rankin Scale (mRS) score was performed at 2 weeks and 3 months after treatment. National Institutes of Health Stroke Scale (NIHSS) score and Barthel Index (BI) score were performed at 2 weeks after treatment, and then vital signs and adverse events were evaluated. Results A total of 227 patients were randomized to treatment ( n = 113, mildronate; n = 114, active-control). After 3 months, there was no significant difference for the primary endpoint between groups categorized in terms of mRS scores of 0–1 and 0–2 ( p = 0.52 and p = 0.07, respectively). There were also no significant differences for the secondary endpoint between groups categorized in terms of NIHSS scores of >5 and >8 ( p = 0.98 and p = 0.97, respectively) or BI scores of >75 and >95 ( p = 0.49 and p = 0.47, respectively) at 15 days. The incidence of serious adverse events was similar between the two groups. Conclusion Mildronate injection is as effective and safe as cinepazide injection in treating acute cerebral infarction.