奥克特雷奥提德皮下存储用于巨:一个随机的,双盲的,安慰剂控制的第三阶段试验,ACROINNOVA 1
Diego Ferone1, Pamela Freda2, Laurence Katznelson3
1Endocrinology Unit, Department of Internal Medicine, IRCCS Ospedale Policlinico San Martino, 16131 Genova, Italy.
The Journal of clinical endocrinology and metabolism
|October 8, 2024
概括
与安慰剂相比,CAM2029是一种新型的八二基储存,证明了对壮症的优越生化控制. 这种自我治疗改善了生活质量和患者的满意度.
科学领域:
- 内分泌学 在内分泌学.
- 药理学 药理学是指药理学的学科.
背景情况:
- 巨症是一种由过度增长激素 (GH) 和胰岛素类增长因子-1 (IGF-1) 引起的疾病,影响生活质量 (QoL) 和死亡率.
- 标准的护理 (SoC) 治疗巨,如长效重复性奥克胺或兰胺自凝,通常需要医疗保健提供者的管理.
- CAM2029是一种使用FluidCrystal技术增强生物可用性的新型皮下八胺储存剂,可自给药和室温储存.
研究的目的:
- 评估CAM2029与安慰剂相比在壮症患者中实现生化控制方面的优越性.
- 评估CAM2029在维持IGF-1水平在正常的上限 (ULN) 内的有效性.
主要方法:
- 在10个国家的45个地点进行了为期24周的多国,随机,双盲,第三阶段试验 (NCT04076462).
- 在SoC上有72名生物化学控制的壮病患者被随机分为2:1接受每月一次的CAM2029 (n=48) 或安慰剂 (n=24).
- 主要终点:IGF-1 ≤ULN在22/24.周的患者比例. 关键的二次终点包括IGF-1 ≤ULN与剂量减少反应者和联合IGF-1/GH控制.
主要成果:
- 在22/24.2周,CAM2029对IGF-1控制 (72.2%对37.5%,P=.0018) 和联合IGF-1/GH控制 (70.0%对37.5%,P=.0035) 的反应率更高.
- 与安慰剂和基线相比,接受CAM2029治疗的患者经历了良好的症状控制,改善了QoL,并增加了治疗满意度.
- CAM2029的安全性概况与SoC治疗一致,并且耐受性良好.
结论:
- CAM2029提供了一种方便而有效的自我治疗壮症的治疗选择.
- 该研究表明,与安慰剂相比,CAM2029具有更好的生化控制.
- 与基线SoC相比,CAM2029改善了症状控制,QoL和患者满意度.
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