The DIAN-TU Next Generation Alzheimer's prevention trial: Adaptive design and disease progression model

被引：215

作者：

Bateman, Randall J. ^{[1
]}

Benzinger, Tammie L. ^{[2
]}

Berry, Scott ^{[3
]}

Clifford, David B. ^{[1
]}

Duggan, Cynthia ^{[1
]}

Fagan, Anne M. ^{[1
]}

Fanning, Kathleen ^{[1
]}

Farlow, Martin R. ^{[4
]}

Hassenstab, Jason ^{[1
]}

McDade, Eric M. ^{[1
]}

Mills, Susan ^{[1
]}

Paumier, Katrina ^{[1
]}

Quintana, Melanie ^{[3
]}

Salloway, Stephen P. ^{[5
]}

Santacruz, Anna ^{[1
]}

Schneider, Lon S. ^{[6
]}

Wang, Guoqiao ^{[7
]}

Xiong, Chengjie ^{[7
]}

机构：

[1] Washington Univ, Dept Neurol, St Louis, MO 63130 USA

[2] Washington Univ, Dept Radiol, St Louis, MO USA

[3] Berry Consultants, Austin, TX USA

[4] Indiana Univ, Indiana Alzheimer Dis Ctr, Indianapolis, IN 46204 USA

[5] Butler Hosp, Brown Med Sch, Memory & Aging Program, Providence, RI 02906 USA

[6] Univ Southern Calif, Keck Sch Med, Alzheimers Dis Res Ctr, Los Angeles, CA USA

[7] Washington Univ, Dept Biostat, St Louis, MO USA

来源：

ALZHEIMERS & DEMENTIA | 2017年 / 13卷 / 01期

关键词：

Alzheimer's disease; Alzheimer's prevention trial; Adaptive clinical trial; Biomarkers; Disease progression model; Cognitive composite; Dose adjustment; DIAN-TU; Amyloid; Tau; Autosomal dominant Alzheimer's disease; CEREBROSPINAL-FLUID BIOMARKER; A-BETA; COGNITIVE DECLINE; AMYLOID-BETA; TAU; BAPINEUZUMAB; NEUROGRANIN; PATHOLOGY; PLASMA; BRAIN;

D O I：

10.1016/j.jalz.2016.07.005

中图分类号：

R74 [神经病学与精神病学];

学科分类号：

摘要：

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. (C) 2016 the Alzheimer's Association. Published by Elsevier Inc. All rights reserved.

引用

页码：8 / 19

页数：12

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