TY  - JOUR
TI  - Enrichment of clinical trials in MCI due to AD using markers of amyloid and neurodegeneration
AV  - restricted
Y1  - 2016/09/20/
EP  - 1241
KW  - Aged
KW  -  Alzheimer Disease
KW  -  Amyloid beta-Peptides
KW  -  Biomarkers
KW  -  Brain
KW  -  Clinical Trials as Topic
KW  -  Cognitive Dysfunction
KW  -  Cohort Studies
KW  -  Female
KW  -  Humans
KW  -  Male
KW  -  Mental Status Schedule
KW  -  Nerve Degeneration
KW  -  Positron-Emission Tomography
N2  - OBJECTIVE: To investigate the effect of enriching mild cognitive impairment (MCI) clinical trials using combined markers of amyloid pathology and neurodegeneration. METHODS: We evaluate an implementation of the recent National Institute for Aging-Alzheimer's Association (NIA-AA) diagnostic criteria for MCI due to Alzheimer disease (AD) as inclusion criteria in clinical trials and assess the effect of enrichment with amyloid (A+), neurodegeneration (N+), and their combination (A+N+) on the rate of clinical progression, required sample sizes, and estimates of trial time and cost. RESULTS: Enrichment based on an individual marker (A+ or N+) substantially improves all assessed trial characteristics. Combined enrichment (A+N+) further improves these results with a reduction in required sample sizes by 45% to 60%, depending on the endpoint. CONCLUSIONS: Operationalizing the NIA-AA diagnostic criteria for clinical trial screening has the potential to substantially improve the statistical power of trials in MCI due to AD by identifying a more rapidly progressing patient population.
ID  - discovery10073276
IS  - 12
N1  - This version is the version of record. For information on re-use, please refer to the publisher?s terms and conditions.
SP  - 1235
VL  - 87
JF  - Neurology
A1  - Wolz, R
A1  - Schwarz, AJ
A1  - Gray, KR
A1  - Yu, P
A1  - Hill, DLG
A1  - Alzheimer's Disease Neuroimaging Initiative
UR  - https://doi.org/10.1212/WNL.0000000000003126
SN  - 1526-632X
ER  -