在患有脱髓性神经病症的患者中高剂量制药级生物素:一个2b期的开放标签,不受控制的试点研究
Alain Créange1,2,3, Emilie Hutin4,5, Frédéric Sedel6
1AP-HP, Hôpital Henri Mondor, Service de Neurologie, 94010, Créteil, France. alain.creange@aphp.fr.
BMC neurology
|October 30, 2023
概括
高剂量的生物素显示神经功能和神经退髓化神经病变的运动能力得到改善,尽管主要目标未实现. 治疗耐受性很好,这表明对这些疾病的潜在治疗益处.
科学领域:
- 神经学 神经学
- 临床药理学 临床药理学
背景情况:
- 研究了高剂量制药级生物素用于脱髓化神经病变.
- 对生物在神经疾病中的疗效进行概念验证研究.
研究的目的:
- 评估高剂量生物丁在患有脱髓化神经病变的患者中的疗效.
- 评估电生理学变量和临床结果的改善.
主要方法:
- 第二阶段b试点研究与15名患者在三个神经病变类型.
- 每天服用100毫克生物素口服三次,长达52周.
- 在4个电生理学变量中的2个中,评估的初级终点是10%的改善.
主要成果:
- 主要终点只有一个患者达到.
- 在二次终点中观察到的改善:MRC得分,INCAT感官总和得分,6分钟的步行距离,神经刺激性参数.
- 步态改善和神经兴奋度之间存在正相关性.
结论:
- 高剂量的生物素改善了脱髓化神经病变中的感觉和运动参数,步态和神经刺激能力.
- 治疗表现出令人满意的耐受性,没有与生物素相关的严重不良事件.
- 尽管未达到主要终点,但可能需要进一步的研究.
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