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Enrichment factors for clinical trials in mild-to-moderate Alzheimer's disease.


ABSTRACT: Introduction:Heterogeneity of outcomes in Alzheimer's disease (AD) clinical trials necessitates large sample sizes and contributes to study failures. This analysis determined whether mild-to-moderate AD populations could be enriched for cognitive decline based on apolipoprotein (APOE) ?4 genotype, family history of AD, and amyloid abnormalities. Methods:Modeling estimated the number of randomized patients needed to detect a 2-point treatment difference on the AD Assessment Scale-Cognitive subscale using placebo data from three randomized, double-blind trials (ClinicalTrials.gov Identifiers: NCT01955161, NCT02006641, and NCT02006654). Results:An 80% power to detect a 2-point treatment effect required the randomization of 148 amyloid-positive patients; 178 ?4 homozygous or amyloid-positive patients; and 231 ?4 homozygous, family history-positive, or amyloid-positive patients, compared with 1619 unenriched patients (per arm). Discussion:Enrichment in mild-to-moderate AD clinical trials can be achieved using combinations of biomarkers/risk factors to increase the likelihood of observing potential treatment effects.

SUBMITTER: Ballard C 

PROVIDER: S-EPMC6527908 | biostudies-literature | 2019

REPOSITORIES: biostudies-literature

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Enrichment factors for clinical trials in mild-to-moderate Alzheimer's disease.

Ballard Clive C   Atri Alireza A   Boneva Neli N   Cummings Jeffrey L JL   Frölich Lutz L   Molinuevo José Luis JL   Tariot Pierre N PN   Raket Lars Lau LL  

Alzheimer's & dementia (New York, N. Y.) 20190520


<h4>Introduction</h4>Heterogeneity of outcomes in Alzheimer's disease (AD) clinical trials necessitates large sample sizes and contributes to study failures. This analysis determined whether mild-to-moderate AD populations could be enriched for cognitive decline based on apolipoprotein (<i>APOE</i>) ε4 genotype, family history of AD, and amyloid abnormalities.<h4>Methods</h4>Modeling estimated the number of randomized patients needed to detect a 2-point treatment difference on the AD Assessment  ...[more]

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