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Abstract Details

Machine Learning Models Can Improve Efficiency of Alzheimer’s Disease Clinical Trials.
Aging, Dementia, and Behavioral Neurology
P16 - Poster Session 16 (5:30 PM-6:30 PM)
10-012
To investigate if machine learning (ML) models can effectively predict clinical disease progression in mild-to-moderate Alzheimer’s disease (AD) patients during the timeframe of a phase 3 clinical trial.
In AD trials active treatment is intended to slow down the rate of cognitive decline. Enrollment of participants who will not show cognitive decline reduce the chance of detecting therapeutic effects. One strategy to boost the power of trials is to enroll individuals likely to progress using data-driven predictive models.

We used data from 202 participants from the Alzheimer’s Disease Neuroimaging Initiative (ADNI) and 77 participants from the placebo arm of the phase 3 trial of semagacestat for AD (LFAN) with diagnosis of mild-to-moderate AD at baseline. K-Nearest Neighborhood (kNN) models were trained using ADNI data to classify participants to 1) cognitive decliners, change of >0 from baseline in the Alzheimer’s Disease Assessment Scale cognitive subscale (ADAS-cog) score; and 2) Stable cognition, change of ≤0 from baseline ADAS-cog score, after 12 months of follow-up. Trained models were applied to data from LFAN trial (validation sample) to predict disease progression in 12 months. Feature-set in all models included demographics, Apo4 status, neurocognitive tests, and volumetric MRI.

kNN classifier had a sensitivity of 77.9% and specificity of 35.4% for identifying decliners in ADNI sample. In LFAN sample, the model showed an overall accuracy of 63.6%, sensitivity of 66.6%, and specificity of 50% in identifying decliners at the 12 months of follow-up. The model had a positive predictive value of 85.7%, which is 22.7% more than the base prevalence of 63% for decliners.
Machine learning predictive models can be effectively used to boost the power of clinical trials by reducing the sample size.
Authors/Disclosures
Ali Ezzati, MD (University of California, Irvine)
PRESENTER
The institution of Dr. Ezzati has received research support from NIA. The institution of Dr. Ezzati has received research support from Alzheimer's Association. The institution of Dr. Ezzati has received research support from Cure Alzheimer's Fund.
Richard B. Lipton, MD, FÂé¶¹´«Ã½Ó³»­ (Albert Einstein College of Medicine) Dr. Lipton has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Allergan/Abbvie. Dr. Lipton has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Amgen. Dr. Lipton has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Biohaven. Dr. Lipton has received personal compensation in the range of $50,000-$99,999 for serving as a Consultant for Eli Lilly. Dr. Lipton has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Lundbeck. Dr. Lipton has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for GlaxoSmithKline. Dr. Lipton has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Teva. Dr. Lipton has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Vedanta. Dr. Lipton has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Merck. Dr. Lipton has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Grifols. Dr. Lipton has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Pfizer. Dr. Lipton has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Axon. Dr. Lipton has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Satsuma. Dr. Lipton has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Genentech. Dr. Lipton has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Cool Tech. Dr. Lipton has received personal compensation in the range of $500-$4,999 for serving as a Consultant for BDSI. Dr. Lipton has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Linpharma. Dr. Lipton has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Axsome. Dr. Lipton has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Clexio. Dr. Lipton has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Shiratronics. Dr. Lipton has received personal compensation in the range of $5,000-$9,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Allergan/Abbvie. Dr. Lipton has received personal compensation in the range of $10,000-$49,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Biohaven. Dr. Lipton has received personal compensation in the range of $10,000-$49,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Eli Lilly. Dr. Lipton has received personal compensation in the range of $10,000-$49,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Lundbeck. Dr. Lipton has or had stock in Biohaven.Dr. Lipton has or had stock in Manistee.Dr. Lipton has or had stock in Axon.Dr. Lipton has or had stock in CoolTech. The institution of Dr. Lipton has received research support from Teva. The institution of Dr. Lipton has received research support from Amgen. The institution of Dr. Lipton has received research support from Allergan/Abbvie. The institution of Dr. Lipton has received research support from Gammacore. The institution of Dr. Lipton has received research support from Axsome. The institution of Dr. Lipton has received research support from Charleston Labs. The institution of Dr. Lipton has received research support from Eli Lilly. The institution of Dr. Lipton has received research support from Satsuma. The institution of Dr. Lipton has received research support from NIH . The institution of Dr. Lipton has received research support from Veterans Administration. Dr. Lipton has received publishing royalties from a publication relating to health care.