What is dose expansion and why is a dose expansion trial performed?

Dose expansion trials are clinical trials that evaluate the safety, pharmacokinetics, pharmacodynamics, and clinical activity of a new drug or treatment in a larger group of patients than in the initial dose escalation trial. These trials are typically performed after the initial trial has shown promising results and the researchers want to further investigate the potential benefits and risks of the drug or treatment in a broader patient population.The primary goal of a dose expansion trial is to assess the efficacy and safety of a new treatment in a larger number of patients with the same disease or condition as those studied in the original trial. This helps to confirm the findings from the earlier study and provides additional information about the drug's effectiveness and side effects in a more diverse patient population. In addition to testing the drug's efficacy and safety, dose expansion trials may also explore how the drug affects biomarkers such as tumor size, blood markers, or imaging studies. These data can help to inform future clinical trials and guide the development of personalized medicine approaches.