DEVOTE研究探讨在脊柱肌肉缩中使用更高剂量的Nusinersen:研究设计和A部分结果
Richard S Finkel1, John W Day2, Samuel Ignacio Pascual Pascual3
1Center for Experimental Neurotherapeutics, St. Jude Children's Research Hospital, Memphis, TN, USA.
Journal of neuromuscular diseases
|July 2, 2023
概括
在DEVOTE研究中,研究人员研究了DEVOTE.
科学领域:
- 神经学 神经学
- 药理学 药理学是指药理学的学科.
- 临床试验 临床试验
背景情况:
- 药理动力学/药理动力学建模表明,较高的努辛森剂量可能会提高疗效.
- 努辛森的批准剂量为12毫克.
研究的目的:
- 为了评估高剂量的nusinersen的安全性,耐受性和有效性.
- 描述三个部分的DEVOTE临床研究 (NCT04089566) 的设计.
主要方法:
- 部分A评估了儿科参与者的高剂量nusinersen的安全性和耐受性.
- 部分B和C分别涉及疗效评估和剂量过渡评估.
- 该研究在A部分招募了6名年龄在6.1-12.6岁之间的参与者.
主要成果:
- 部分A完成没有安全问题;大多数治疗出现的不良事件是轻微的和与程序相关的.
- 脑脊液的nusinersen水平与模型预测保持一致.
- 大多数参与者表现出运动功能的稳定或改善,尽管A部分并非以疗效为重点.
结论:
- 部分A的发现支持持续开发更高的nusinersen剂量.
- 目前正在进行B部分和C部分的进一步调查.
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