TY - JOUR
T1 - The DIAN-TU Next Generation Alzheimer's prevention trial
T2 - Adaptive design and disease progression model
AU - DIAN-TU Pharma Consortium for the Dominantly Inherited Alzheimer Network
AU - Bateman, Randall J.
AU - Benzinger, Tammie L.
AU - Berry, Scott
AU - Clifford, David B.
AU - Duggan, Cynthia
AU - Fagan, Anne M.
AU - Fanning, Kathleen
AU - Farlow, Martin R.
AU - Hassenstab, Jason
AU - McDade, Eric M.
AU - Mills, Susan
AU - Paumier, Katrina
AU - Quintana, Melanie
AU - Salloway, Stephen P.
AU - Santacruz, Anna
AU - Schneider, Lon S.
AU - Wang, Guoqiao
AU - Xiong, Chengjie
N1 - Publisher Copyright:
© 2016 the Alzheimer's Association
PY - 2017/1/1
Y1 - 2017/1/1
N2 - 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.
AB - 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.
KW - Adaptive clinical trial
KW - Alzheimer's disease
KW - Alzheimer's prevention trial
KW - Amyloid
KW - Autosomal dominant Alzheimer's disease
KW - Biomarkers
KW - Cognitive composite
KW - DIAN-TU
KW - Disease progression model
KW - Dose adjustment
KW - Tau
UR - http://www.scopus.com/inward/record.url?scp=85002578779&partnerID=8YFLogxK
U2 - 10.1016/j.jalz.2016.07.005
DO - 10.1016/j.jalz.2016.07.005
M3 - Article
C2 - 27583651
AN - SCOPUS:85002578779
SN - 1552-5260
VL - 13
SP - 8
EP - 19
JO - Alzheimer's and Dementia
JF - Alzheimer's and Dementia
IS - 1
ER -