Efficacy and safety of mildronate for acute ischemic stroke: a randomized, double-blind, active-controlled phase II multicenter trial

Clin Drug Investig. 2013 Oct;33(10):755-60. doi: 10.1007/s40261-013-0121-x.

Abstract

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.

Publication types

  • Clinical Trial, Phase II
  • Multicenter Study
  • Randomized Controlled Trial
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Aged
  • Brain Ischemia / diagnosis
  • Brain Ischemia / drug therapy*
  • Brain Ischemia / physiopathology
  • Cardiovascular Agents / adverse effects
  • Cardiovascular Agents / therapeutic use*
  • Double-Blind Method
  • Female
  • Humans
  • Male
  • Methylhydrazines / adverse effects
  • Methylhydrazines / therapeutic use*
  • Middle Aged
  • Stroke / diagnosis
  • Stroke / drug therapy*
  • Stroke / physiopathology
  • Treatment Outcome

Substances

  • Cardiovascular Agents
  • Methylhydrazines
  • 3-(2,2,2-trimethylhydrazine)propionate