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Adaptive sequential design for phase II single-arm oncology trials: an expansion of Simon's design.

Ping GaoWeidong Zhang
Published in: Journal of biopharmaceutical statistics (2024)
Single-arm phase II trials are very common in oncology. A fixed sample trial may lack sufficient power if the true efficacy is less than the assumed one. Adaptive designs have been proposed in the literature. We propose a Simon's design based, adaptive sequential design. Simon's design is the most used fixed sample design for single-arm phase II oncology trials. A prominent feature of Simon's design is that it minimizes the sample size when there is no clinically meaningful efficacy. We identify Simon's trial as a special group sequential design. Established methods for sample size re-estimation (SSR) can be readily applied to Simon's design. Simulations show that simply adding SSR to Simon's design may still not provide desirable power. We propose some expansions to Simon's design. The expanded design with SSR can provide even more power.
Keyphrases
  • phase ii
  • clinical trial
  • open label
  • randomized controlled trial
  • systematic review
  • healthcare
  • study protocol
  • deep learning
  • molecular dynamics
  • health insurance