在RASopathies中改善突触可塑性和认知功能 - 一个单中心,随机,双盲,并行组,安慰剂控制,交叉临床试验 (SynCoRAS)
Nikolai H Jung1, Silvia Egert-Schwender2, Beate Schossow2
1Social Pediatrics, School of Medicine, Technical University of Munich, Munich, Germany. nikolai.jung@tum.de.
Trials
|June 6, 2023
概括
这项临床试验调查了洛瓦斯塔丁和拉莫特里金是否可以通过增强突触可塑性来改善RAS病的认知功能. 早期结果表明,这些药物可能会改善这些罕见遗传疾病患者的认知和警觉.
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.
- 遗传学 是一个遗传学.
背景情况:
- 认知障碍在RASopathies (例如NF1,NS) 中普遍存在,与突触可塑性受损有关.
- 动物研究表明,洛瓦斯塔丁 (LOV) 和拉莫特里金 (LTG) 改善了突触可塑性和认知能力.
- 这项试验旨在将这些发现转化为患有RAS病的人类患者.
研究的目的:
- 评估LOV和LTG对诺南综合征 (NS) 和神经纤维素瘤1型 (NF1) 患者的突触可塑性和认知功能的影响.
- 评估LTG对NS患者突触可塑性和警觉性的影响.
- 检查LOV对NS患者突触可塑性和警觉性的影响.
主要方法:
- 第二期a,随机,双盲,安慰剂控制,交叉试验 (SynCoRAS) 涉及28名患者 (NS和NF1).
- 参与者每天服用300毫克LTG或安慰剂 (NS/NF1) 或200毫克LOV或安慰剂 (NS) 4天,并在7天后进行交叉.
- 通过跨磁刺激 (TMS;qTBS) 测量突触可塑性;使用注意力表现测试 (TAP) 评估注意力.
主要成果:
- 在NF1患者的初步发现表明,LOV改善了突触可塑性和认知能力.
- 该试验调查了这些积极影响是否延伸到NS患者.
- 二级终点包括注意力 (TAP) 和短间隔皮质抑制 (SICI).
结论:
- 这项研究涉及突触可塑性和认知缺陷,这是RASopathies中的关键问题.
- LTG在改善突触可塑性和认知功能方面表现有前途.
- 预计LOV和LTG都会增强突触可塑性和警觉性,可能改善认知.
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