Our research - Modernizing AD clinical trial design and heterogeniety testing methods

Our research focuses on developing and applying advanced heterogeneity testing methods to better understand variable treatment responses in Alzheimer’s disease (AD) clinical trials and patient cohorts. Recognizing that AD is inherently heterogeneous, we investigate strategies to identify true responders within randomized controlled trials (RCTs), addressing a critical gap where one-size-fits-all treatments often fail. Using the I-CONECT RCT as a model, we have implemented an individual-level treatment response (ITR) approach to quantify heterogeneity and its association with delayed cognitive decline. Our findings highlight significant variation in treatment effects, with the top 30% of responders demonstrating a meaningful delay in cognitive deterioration.

In addition, our research develops novel analytical pipelines employing tree-based algorithms and sophisticated statistical models to build responder profiles. These tools aim to guide enrichment strategies in clinical trials, improving their efficiency and impact. Beyond non-pharmacological interventions, we also explore pharmacological approaches, such as assessing the cognitive and biomarker effects of Nicotinamide Riboside (TruNiagen®, NR) through integrated trial designs. Employing combined parallel and crossover designs, this work enhances statistical power and reveals promising biomarker changes linked to NR supplementation.

Contact me if you are interested in novel analytics and trial methods!