DIAN-TU 下一代阿尔茨海默病预防试验：适应性设计与疾病进展模型
The DIAN-TU Next Generation Alzheimer's prevention trial: Adaptive design and disease progression model.
作者信息
Bateman Randall J, Benzinger Tammie L, Berry Scott, Clifford David B, Duggan Cynthia, Fagan Anne M, Fanning Kathleen, Farlow Martin R, Hassenstab Jason, McDade Eric M, Mills Susan, Paumier Katrina, Quintana Melanie, Salloway Stephen P, Santacruz Anna, Schneider Lon S, Wang Guoqiao, Xiong Chengjie
机构信息
Department of Neurology, Washington University in St Louis, St Louis, MO, USA.
Department of Radiology, Washington University in St Louis, St Louis, MO, USA.
出版信息
Alzheimers Dement. 2017 Jan;13(1):8-19. doi: 10.1016/j.jalz.2016.07.005. Epub 2016 Aug 29.
INTRODUCTION
The Dominantly Inherited Alzheimer Network Trials Unit (DIAN-TU) trial is an adaptive platform trial testing multiple drugs to slow or prevent the progression of Alzheimer's disease in autosomal dominant Alzheimer's disease (ADAD) families. With completion of enrollment of the first two drug arms, the DIAN-TU now plans to add new drugs to the platform, designated as the Next Generation (NexGen) prevention trial.
METHODS
In collaboration with ADAD families, philanthropic organizations, academic leaders, the DIAN-TU Pharma Consortium, the National Institutes of Health, and regulatory colleagues, the DIAN-TU developed innovative clinical study designs for the DIAN-TU NexGen prevention trial.
RESULTS
Our expanded trial toolbox consists of a disease progression model for ADAD, primary end point DIAN-TU cognitive performance composite, biomarker development, self-administered cognitive assessments, adaptive dose adjustments, and blinded data collection through the last participant completion.
CONCLUSION
These steps represent elements to improve efficacy of the adaptive platform trial and a continued effort to optimize prevention and treatment trials in ADAD.
引言
显性遗传阿尔茨海默病网络试验单元（DIAN-TU）试验是一项适应性平台试验，旨在测试多种药物，以减缓或预防常染色体显性遗传阿尔茨海默病（ADAD）家族中阿尔茨海默病的进展。随着前两个药物组的入组工作完成，DIAN-TU现在计划在该平台上增加新药，即下一代（NexGen）预防试验。
方法
DIAN-TU与ADAD家族、慈善组织、学术带头人、DIAN-TU制药联盟、美国国立卫生研究院以及监管部门的同事合作，为DIAN-TU下一代预防试验制定了创新的临床研究设计。
结果
我们扩展后的试验工具包包括ADAD疾病进展模型、主要终点DIAN-TU认知表现综合指标、生物标志物开发、自我管理的认知评估、适应性剂量调整以及在最后一名参与者完成试验前进行的盲态数据收集。
结论
这些步骤是提高适应性平台试验疗效的要素，也是为优化ADAD预防和治疗试验而持续做出的努力。
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