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

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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.

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Acknowledgments

This study was funded by Ge-Lin Biopharmaceutical Co. Ltd., Shenyang, China. The authors have no potential conflicts of interest that are directly relevant to the content of this study. The Steering Committee for this study consisted of Prof. Su Xiuchu, Prof. Xu Dezhong, Prof. Wu Baoren, and Dr. Fan Fulin. The Safety Board consisted of Prof. Huang Yuangui, Prof. Li Huanzhang, and Prof. Li Kaizong.

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Authors and Affiliations

  1. Department of Neurology, Xijing Hospital, The Fourth Military Medical University, Xi’an, Shaanxi, China

    Yi Zhu, Guangyun Zhang, Jun Zhao, Deshuai Li, Juanfang Liu, Xuedong Liu, Haibo Zhao, Xiao Zhang & Gang Zhao

  2. Department of Orthopaedics, Tangdu Hospital, Fourth Military Medical University, Xi’an, Shaanxi, China

    Xiaodong Yan

  3. Department of Health Statistics, Fourth Military Medical University, Xi’an, Shaanxi, China

    Jielai Xia

  4. First Affiliated Hospital of Xi’an Jiaotong University, Xi’an, Shaanxi, China

    Zhengyi Li

  5. Second Affiliated Hospital, Zhejiang University School of Medicine, Zhejiang, China

    Baorong Zhang

  6. People’s Hospital of Hebei Province, Hebei, China

    Zongcheng Guo

  7. Bethune International Peace Hospital of People’s Liberation Army, Hebei, China

    Lianyuan Feng

  8. First Hospital of China Medical University, Beijing, China

    Zhaodong Zhang

  9. General Hospital of Shenyang Military Region, Shenyang, China

    Fang Qu

Authors
  1. Yi Zhu
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  2. Guangyun Zhang
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  3. Jun Zhao
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  13. Zongcheng Guo
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  15. Zhaodong Zhang
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  16. Fang Qu
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  17. Gang Zhao
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Corresponding author

Correspondence to Gang Zhao.

Additional information

Y. Zhu and G. Zhang contributed equally to this work.

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Zhu, Y., Zhang, G., Zhao, J. et al. Efficacy and Safety of Mildronate for Acute Ischemic Stroke: A Randomized, Double-Blind, Active-Controlled Phase II Multicenter Trial. Clin Drug Investig 33, 755–760 (2013). https://doi.org/10.1007/s40261-013-0121-x

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