Publications by authors named "Mike Pauley"
A computational tool to optimize clinical trial parameter selection in Duchenne muscular dystrophy: A practical guide and case studies.
CPT Pharmacometrics Syst Pharmacol
November 2024
Article Synopsis
- * A new clinical trial simulator (CTS) has been developed using five models that track changes in functional test performances, offering a way to optimize trial parameters and endpoints for DMD studies.
- * This report illustrates the CTS’s functionality through two case studies, showcasing how it can enhance trial design while maintaining statistical significance, ultimately preventing unnecessary increases in trial size and duration.
Whereas islet autoantibodies (AAs) are well-established risk factors for developing type 1 diabetes (T1D), there is a lack of biomarkers endorsed by regulators to enrich clinical trial populations for those at risk of developing T1D. As such, the development of therapies that delay or prevent the onset of T1D remains challenging. To address this drug development need, the Critical Path Institute's T1D Consortium (T1DC) acquired patient-level data from multiple observational studies and used a model-based approach to evaluate the utility of islet AAs as enrichment biomarkers in clinical trials.
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