Abstract

Background

The β-D-Mannuronic acid (M2000) as a novel immunosuppressive drug, patented (PCT/EP2017/067920), has shown positive effects in experimental model of multiple sclerosis (MS). In this study, our aim was to assess efficacy and safety outcomes in MS treated patients with mannuronic acid compared to the conventional drug.

Methods

In a 6-month, randomized controlled, phase II trial, we enrolled patients who had secondary progressive multiple sclerosis (SPMS), were 21–54 years of age, with a score of 1–7 on the Expanded Disability Status Scale (EDSS), and who had at least one relapse in the previous 6 months. Patients were administered orally 1000 mg/day (two 500 mg/capsule daily) of M2000. Endpoints included changes in brain magnetic resonance imaging (MRI) measures and the EDSS score, as compared to the conventional drug (interferon beta-1a, interferon beta-1b).

Results

A total of 25 (92.5%) of the M2000 treated patients and 25 conventionally treated patients completed the study. M2000 had better performance compared to the conventional drug regarding to MRI-related measurements, however, the differences between groups were not statistically significant. M2000 decreased the disability progression over the 6-month period. The EDSS score was decreased in the M2000 treated group in the sixth month versus the conventional drug (p < 0.009). Furthermore, we did not observe any short-term side effects.

Conclusions

As compared with the conventional drug, mannuronic acid (M2000) improved the rate of disability progression. This clinical trial demonstrated the efficacy and safety of mannuronic acid in patients with SPMS. (Registered Clinical Trials number, IRCT2016111313739N6).

中文翻译：

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一项用于评估甘露糖醛酸在继发性进展型多发性硬化症中的疗效和安全性的对照、随机 II 期临床试验

摘要

背景

β-D-甘露糖醛酸（M2000）作为一种新型免疫抑制药物，已获得专利（PCT/EP2017/067920），在多发性硬化症（MS）实验模型中显示出积极作用。在这项研究中，我们的目的是评估与传统药物相比，使用甘露糖醛酸治疗 MS 的患者的疗效和安全性结果。

方法

在一项为期 6 个月的随机对照 II 期试验中，我们招募了 21-54 岁的继发性进行性多发性硬化症 (SPMS) 患者，扩展残疾状态量表 (EDSS) 得分为 1-7 ，并且在过去 6 个月内至少复发过一次。患者口服 1000 毫克/天（每天两次 500 毫克/胶囊）的 M2000。与传统药物（干扰素β-1a、干扰素β-1b）相比，终点包括脑磁共振成像（MRI）测量和EDSS评分的变化。

结果

共有 25 名 (92.5%) 接受 M2000 治疗的患者和 25 名接受常规治疗的患者完成了研究。与常规药物相比，M2000 在 MRI 相关测量方面具有更好的性能，但组间差异无统计学意义。M2000 在 6 个月期间减少了残疾进展。与常规药物相比，M2000 治疗组第 6 个月的 EDSS 评分降低（p  < 0.009）。此外，我们没有观察到任何短期副作用。

结论

与传统药物相比，甘露糖醛酸（M2000）提高了残疾进展率。该临床试验证明了甘露糖醛酸对 SPMS 患者的疗效和安全性。（注册临床试验编号，IRCT2016111313739N6）。