一个随机的2期动力学试验,评估SAGE-324/BIIB124在基本震的个体
Rodger J Elble1, William G Ondo2, Kelly E Lyons3
1Department of Neurology, Southern Illinois University School of Medicine, Springfield, Illinois, USA.
Movement disorders : official journal of the Movement Disorder Society
|February 15, 2024
概括
一种GABAA受体调节剂SAGE-324/BIIB124在第二阶段试验中显示出基本震 (ET) 症状的显著改善. 支持进一步开发这种潜在的ET治疗.
科学领域:
- 神经科学是一个神经科学.
- 药理学 药理学是指药理学的学科.
背景情况:
- SAGE-324/BIIB124是一种正在试验中的阳性全调节器,向GABAA受体.
- 基本震 (ET) 是一种神经疾病,其特征是不自发的节奏震.
研究的目的:
- 评估SAGE-324/BIIB124在患有基本震 (ET) 的人群中的疗效和安全性.
- 该KINETIC研究 (NCT04305275) 是一个第二阶段,双盲,随机,安慰剂控制的试验.
主要方法:
- 69名年龄在18至80岁的参与者被随机分配给口服SAGE-324/BIIB124 (60毫克) 或安慰剂,每天服用一次,持续28天.
- 主要终点是与基线的变化,基本震评级量表表现分量表 (TETRAS-PS) 第4项 (上肢震) 在第29天.
主要成果:
- SAGE-324/BIIB124在第29天 (P=0.0491) 与安慰剂相比,显示上肢震的统计显著减少.
- 最常见的治疗出现的不良事件是嗜睡,头,疲劳和平衡障碍.
结论:
- 研究结果支持SAGE-324/BIIB124的持续发展,作为治疗基本震的潜在治疗选择.
- 需要进一步的研究,以充分确定SAGE-324/BIIB124在ET患者中的安全性和有效性.
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