Patient Stratification and Enrichment in Alzheimer's Disease Clinical Trials: Multi-Biomarker Enrichment and Disease Modelling Strategies
Presented by: Robin Wolz, Ph.D, Director of Translational Sciences
Disappointing results from large clinical trials in Alzheimer’s disease (AD) that failed over the last years highlight the importance of careful patient selection. Following these observations, trials evaluating anti-amyloid therapy now typically include only Amyloid positive subjects as measured through CSF A-Beta or Amyloid PET. More recently presented results suggest that patient selection based on multiple biomarkers can help further to select those patients that show a high likelihood to progress on primary endpoints and therefore increase power to detect treatment effects.
In this complimentary webinar we present several multi-biomarker enrichment strategies and discusses their operational impact on AD clinical trials. Enrichment strategies include the implementation of recent NIA-AA diagnostic criteria considering both Amyloid positivity and neurodegeneration. The second part of the webinar discusses the concept of recently emerging longitudinal AD disease models and their application to patient selection in trials. Following a hypothetical model of the development of biomarkers and clinical scales over the course of AD, more recent approaches were presented that build statistical models to describe clinical scales longitudinally for a sample population. We present results how this concept can be further extended to build individualized models of disease progression for individual subjects and how the model-predicted change in a primary endpoint can be used for efficient patient selection in clinical trials.
About your presenter
Robin Wolz, Ph.D.
Director of Translational Sciences, IXICO
Robin Wolz finished a Diploma (MSc equiv.) in Computer Science and Engineering at Hamburg University of Technology in 2008 and a Ph.D. in Medical Image Analysis at Imperial College London in 2011. He has a strong track record in medical image analysis with >80 publications, >900 citations and multiple patents in the field. During his work at Imperial College, he co-developed LEAP, a method for the automated analysis of structural MRI. LEAP is now commercially used to analyse clinical trial data and has served as one of four exemplar methods in the qualification process of low hippocampal volume as enrichment biomarker in AD clinical trials with regulatory bodies.
Robin gained experience in technology transfer through different roles at Philips Healthcare before joining IXICO full-time in 2014 as Director of Translational Science where he oversees both the internal scientific development as well as the research activities in grant-funded, external projects.