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


CONCLUSIONS
Mildronate injection is as effective and safe as cinepazide injection in treating acute cerebral infarction.
Department of Neurology, Xijing Hospital, The Fourth Military Medical University, Xi'an, Shaanxi, China.
Yi
Y
Zhu
Guangyun
G
Zhang
Jun
J
Zhao
Deshuai
D
Li
Xiaodong
X
Yan
Juanfang
J
Liu
Xuedong
X
Liu
Haibo
H
Zhao
Jielai
J
Xia
Xiao
X
Zhang
Zhengyi
Z
Li
Baorong
B
Zhang
Zongcheng
Z
Guo
Lianyuan
L
Feng
Zhaodong
Z
Zhang
Fang
F
Qu
Gang
G
Zhao
D000368:Aged; D002545:Brain Ischemia / Q000175:diagnosis / Q000188:drug therapy* / Q000503:physiopathology; D002317:Cardiovascular Agents / Q000009:adverse effects / Q000627:therapeutic use*; D004311:Double-Blind Method; D005260:Female; D006801:Humans; D008297:Male; D008763:Methylhydrazines / Q000009:adverse effects / Q000627:therapeutic use*; D008875:Middle Aged; D020521:Stroke / Q000175:diagnosis / Q000188:drug therapy* / Q000503:physiopathology; D016896:Treatment Outcome
D002317:Cardiovascular Agents; D008763:Methylhydrazines; C050147:3-(2,2,2-trimethylhydrazine)propionate