尼多西兰在PH1中的安全性和有效性:PHYOX3的临时分析
Jaap Groothoff1, Anne-Laure Sellier-Leclerc2, Lisa Deesker1
1Department of Pediatric Nephrology, Emma Children's Hospital, Amsterdam UMC, University of Amsterdam, Amsterdam, The Netherlands.
Kidney international reports
|May 6, 2024
概括
在30个月的时间里,每月一次的尼多西兰治疗显著降低了1型原发性高氧化尿患者的尿氧酸. 在PHYOX3试验中,RNAi疗法证明了持续的疗效和良好的安全性.
科学领域:
- 生物化学 生物化学
- 遗传学 遗传学 是一个
- 药理学 药理学是指药理学的学科.
背景情况:
- 初级高氧化尿1型 (PH1) 是一种罕见的遗传性疾病,影响肝脏氧酸盐代谢.
- 尼多西兰是一种RNA干扰 (RNAi) 疗法,已被批准用于PH1治疗.
- 该PHYOX3试验评估了PH1患者每月服用尼多西兰的长期疗效和安全性.
研究的目的:
- 评估PH1.1患者30个月内每月服用一次尼多西兰的持续疗效和安全性.
- 评估内多西兰对24小时尿液中酸盐 (Uox) 排泄的影响.
- 为了监测不良事件和整体耐受性,nedoosiran治疗.
主要方法:
- 目前正在进行的PHYOX3试验的临时分析.
- 患有PH1的参与者继续从之前的尼多西兰试验 (PHYOX1) 继续.
- 评估了30个月内每月服用尼多西兰的安全性和有效性,包括尿液中氧沙酸盐水平和不良事件.
主要成果:
- 在整个30个月的研究期间,平均估计的淋巴球过率 (eGFR) 保持稳定.
- 平均24小时尿液中酸盐 (Uox) 排泄显示,在所有访问中,从基线持续减少≥60%.
- 76.9%的参与者在第二个月达到正常或接近正常的Uox分泌水平,结果持续.
- 副作用大多为轻度至中度,主要是注射部位事件;没有发生与治疗相关的严重不良事件或停药.
结论:
- 尼多西兰耐受性良好,并且在PH1患者中至少30个月减少Uox分泌的持续有效性.
- 在研究期间,没有发现任何重大安全问题.
- 在PHYOX3研究中,继续收集PH1的长期数据.
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