在肌缩侧面硬化症中,RNS60的扩展访问协议
Grace Addy1, Erica Scirocco1, Dario Gelevski1
1Sean M. Healey and AMG Center for ALS and the Neurological Clinical Research Institute, Massachusetts General Hospital, Harvard Medical School, Boston, Massachusetts, USA.
Muscle & nerve
|March 21, 2025
概括
针对肌缩性侧面硬化症 (ALS) 进行的RNS60在扩展访问协议中显示出有利的安全概况. 在不符合临床试验条件的患者中,每天两次雾化得到了很好的耐受.
科学领域:
- 神经科学是一个神经科学.
- 临床药理学 临床药理学
- 肺部医学 肺部医学
背景情况:
- 肌缩侧面硬化症 (ALS) 是一种进展性神经退行性疾病.
- RNS60是一种针对ALS的研究疗法,此前在小鼠模型中显示出疾病减缓效果.
- 以前的研究表明,RNS60在ALS患者中是安全的,并且耐受良好.
研究的目的:
- 评估RNS60的安全性和耐受性,通过每天两次的雾化在ALS患者中进行.
- 为无法参加受控临床试验的患者提供RNS60访问.
- 评估晚期ALS的长期剂量可行性.
主要方法:
- 一个正在进行的扩展访问协议 (EAP) 治疗了84名患有ALS的参与者.
- 参与者通过每天两次的家庭雾化获得RNS60,长达48个月.
- 通过不良事件和实验室测试来监测安全性.
主要成果:
- 与治疗相关的最常见的不良事件是分泌量增加 (32%).
- 严重的不良事件和死亡不被认为与RNS60治疗有关.
- 这项研究证明了长期RNS60的可行性.
结论:
- 每天两次雾化RNS60在ALS患者中表现出良性副作用.
- 扩展访问协议是对晚期ALS临床试验的一种可行的补充方法.
- 在传统的试验环境之外,RNS60对ALS患者的长期治疗有希望.
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