针对阿波利波蛋白的抗意义疗法:一个随机,双盲,安慰剂控制的第一阶段研究
Sotirios Tsimikas1, Nicholas J Viney2, Steven G Hughes2
1University of California San Diego, La Jolla, CA, USA; Isis Pharmaceuticals, Carlsbad, CA, USA.
Lancet (London, England)
|July 27, 2015
概括
在剂量上,ISIS-APO (a) Rx有效降低脂蛋白 (a) (Lp (a)) 的水平. 这项研究支持其作为心血管疾病和动脉狭窄的潜在治疗方法的持续发展.
科学领域:
- 心血管药理学
- 药物开发
- 脂肪新陈代谢
背景情况:
- 升高的脂蛋白 (Lp[a]) 是心血管疾病和动脉狭窄的重要危险因素.
- 目前没有有效的治疗方法可以降低血Lp
- ISIS-APO(a) Rx是一种针对肝脏中阿波脂蛋白 (apo[a]) 合成的新型抗感应药物.
研究的目的:
- 评估ISIS-APO的安全性,药理动力学和药理动力学.
- 在健康成年人中评估ISIS- APO (a) Rx降低血Lp (a) 度的有效性.
主要方法:
- 这是一项随机,双盲,安慰剂控制的第一阶段研究,涉及18至65岁的健康成年人,Lp< a>升高.
- 参与者接受了单次皮下注射或多次剂量ISIS- APO ((a) Rx (50- 400毫克) 或安慰剂.
- 主要终点是从基线30或36天Lp (a) 度的百分比变化;安全性和耐受性也被评估.
主要成果:
- 多剂量ISIS- APO ((a) Rx (100- 300毫克) 显示了血Lp ((a) 度的剂量依赖性降低,降低高达77. 8%的300毫克 (p< 0. 001).
- 还观察到与apoB-100和apo(a) 相关的氧化脂的减少.
- 轻微的注射部位反应是最常见的不良事件.
结论:
- 通过ISIS-APO (a) Rx可以实现强效,剂量依赖和选择性的血Lp (a) 降低.
- 该药物的安全性和耐受性支持其进一步的临床开发.
- ISIS-APO ((a) Rx 是一种有前途的治疗药物,可降低高Lp ((a)) 患者的心血管疾病和动脉缩风险.
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