Flexible Study Design
A Flexible Study Design with Randomization to Placebo
The maximum tolerated dose of a new drug, supposed to alleviate some adverse effects of anti‑cancer chemotherapy, was to be established in a phase I trial. To assess the safety of the drug, information on the background toxicity rate in the enrolled sample had to be collected. By using state‑of‑the‑art methodology, a flexible study design was proposed for the trial. It combined the use of the continual reassessment method on a continuous dose scale with a concurrent randomization to placebo. The properties of the design were investigated by conducting a simulation study, which allowed to fine-tune the final form of the trial design. The trial has been completed successfully.