高剂量乌比奎诺补充剂在多系统缩中:一个多中心,随机,双盲,安慰剂控制的第二阶段试验
Jun Mitsui1, Takashi Matsukawa1, Yukari Uemura2
1Department of Molecular Neurology, Graduate School of Medicine, The University of Tokyo, Tokyo, Japan.
EClinicalMedicine
|May 31, 2023
概括
在临床试验中,高剂量的ubiquinol补充剂显著减缓了多重系统缩 (MSA) 的进展,通过改善统一多重系统缩评分表 (UMSARS) 第2部分在48周内的得分.
科学领域:
- 神经科学是一个神经科学.
- 生物化学 生物化学
- 临床试验 临床试验
背景情况:
- 对于辅酶Q10 (CoQ10) 生物合成至关重要的酶COQ2的遗传变异与多个系统缩 (MSA) 有关.
- 在MSA患者中观察到降低的CoQ10水平,这表明CoQ10在疾病的发病过程中可能发挥作用.
- COQ2基因突变和低CoQ10水平突出显示了MSA的潜在治疗标.
研究的目的:
- 在多系统性缩 (MSA) 患者中研究高剂量乌比奎诺 (一种CoQ10) 的疗效和安全性.
- 为了确定是否ubiquinol补充剂可以通过统一多重系统缩评分表 (UMSARS) 测量疾病进展的速度.
主要方法:
- 一个多中心,随机,双盲,安慰剂控制的第二阶段试验,涉及139名MSA患者.
- 患者在48周内每天接受1500毫克乌比奎诺或安慰剂.
- 主要疗效终点是48周后UMSARS第2部分得分的变化.
主要成果:
- 该研究达到其主要疗效结局,与安慰剂相比,ubiquinol组的UMSARS第2部分得分明显下降 (最小平方平均差异:-1.7;P=0.023).
- 二次疗效结局,包括巴特尔指数和亚达克西亚评级尺度,也有利于乌比奎诺尔组.
- 在ubiquinol (23.8%) 和安慰剂 (30.9%) 组之间,不良事件的发生率是可比的,这表明耐受性良好.
结论:
- 高剂量的乌比奎诺在MSA患者中耐受良好.
- 乌比金补充剂在减缓MSA患者功能状态下降方面显示出显著的益处.
- 这些发现支持了CoQ10作为多系统性缩症治疗剂的潜力.
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