脊柱肌肉缩的治疗经验丰富的患者:一个3b期的开放标签试验
Jennifer M Kwon1, Francina Munell2, Laure Le Goff3
1American Family Children's Hospital, University of Wisconsin School of Medicine and Public Health, Madison, WI, USA. kwon@neurology.wisc.edu.
Nature medicine
|December 8, 2025
概括
门内胺基阿贝帕罗维克 (OAV101 IT) 在以前接受其他治疗的脊髓肌缩 (SMA) 的儿童和青少年中显示出可管理的安全性. 这项研究证实,OAV101 IT是OAV101 IT.
科学领域:
- 遗传学和基因治疗
- 神经学 神经学
- 儿科医学 儿科医学
背景情况:
- 脊髓肌肉缩 (SMA) 是一种罕见的遗传疾病,影响运动神经元.
- 内瘤基因abeparvovec (OAV101 IT) 为SMA提供了一个潜在的一次性基因转移疗法.
- 之前的治疗方法如nusinersen和risdiplam已被使用,但需要更广泛的治疗选择.
研究的目的:
- 为了评估OAV101 IT在先前用nusinersen或risdiplam治疗的儿科SMA患者的安全性和耐受性.
- 评估不良事件 (AE),严重不良事件 (SAE) 和特别感兴趣的不良事件 (AESI).
主要方法:
- 一项为期52周的第3b期单臂,开放标签,多中心研究 (STRENGTH,NCT05386680).
- 招募了27名年龄在2岁至18岁以下的SMA参与者,他们之前已经停止了SMA治疗.
- 主要目标是安全性和耐受性评估.
主要成果:
- 所有27名参与者都经历了至少一个AE;鼻炎 (55.6%),炎症 (51.9%) 和吐 (48.1%) 是最常见的.
- 13名参与者 (48.1%) 患有与治疗有关的副作用;吐 (22.2%) 是最常见的.
- 四名参与者 (14.8%) 经历了SAE,主要是呼吸道感染. 没有任何副作用导致死亡或停止治疗. 在AESI中,包括过渡性血小板缺血 (29.6%) 和肝毒性 (14.8%).
结论:
- 在接受过先前治疗的参与者中,OAV101 IT的安全性概况与以前未接受过治疗的患者的发现一致.
- OAV101 IT 似乎是对更广泛的 SMA 患者群体的一次性基因疗法选择,通常耐受性很好.
- 进一步的长期监测是必要的,以充分了解OAV101 IT在这个人群中的安全性和有效性.
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