肌萎缩性脊髓侧索硬化症 HEALEY 适应性平台试验的启示。

IF 17 Q1 CELL BIOLOGY Nature aging Pub Date : 2024-11-01 DOI:10.1038/s43587-024-00740-x
Sabrina Paganoni, Brittney Harkey, Elisa Giacomelli, Merit Cudkowicz, on behalf of the HEALEY ALS Platform Trial Study Group
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引用次数: 0

摘要

与独立的临床试验相比,平台试验具有潜在的优势。在此,我们介绍了 HEALEY 肌萎缩侧索硬化症平台试验的运行框架,该框架可实现适应性强的运行基础设施、四种不同治疗方案的同时入组,以及在初始试验启动后加快新治疗方案的启动时间。
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Lessons from the HEALEY adaptive platform trial in amyotrophic lateral sclerosis
Platform trials present potential advantages over stand-alone clinical trials. Here, we describe the operational framework of the HEALEY Amyotrophic Lateral Sclerosis Platform Trial, which enables an adaptable operational infrastructure, concurrent enrolment into four distinct regimens and an accelerated start-up time for a new regimen added after initial trial launch.
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