Enrichment of clinical trials in MCI due to AD using markers of amyloid and neurodegeneration

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2016-09-20
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American English
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American Academy of Neurology (AAN)
Abstract

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.

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Cite As
Wolz, R., Schwarz, A. J., Gray, K. R., Yu, P., & Hill, D. L. G. (2016). Enrichment of clinical trials in MCI due to AD using markers of amyloid and neurodegeneration. Neurology, 87(12), 1235–1241. https://doi.org/10.1212/WNL.0000000000003126
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0028-3878
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Neurology
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PMC
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