Assessing activity in early-phase trials in oncology: current designs for expansion cohorts and phase 2
December 2, 2021
Be updated on different types of Bayesian and frequentist designs that may be used for expansion cohorts in phase 1 trials and in single-arm and randomized phase 2 trials in oncology.
Historically, single-arm phase 2 trials have been used in oncology in order to confirm tolerability and safety observed in phase 1, and to obtain evidence of activity in specific histological types. Randomized phase 2 trials became increasingly common over the past two decades and have mainly aimed at improving go/no-go decisions to move to phase 3. In parallel, the advent of precision medicine led to two major changes in the landscape of phase 2 trials. The first has been the introduction of master protocols, such as umbrella, basket and phase 2 platform trials, which have their design focused not only on the experimental treatment, but also on the biomarkers. The second major change has been the increasing ability to assess activity in early-phase trials, given the expected low toxicity and high selectivity of treatments and the focus on targeted populations. This led to expansion cohorts, which are often designed using principles that are applicable to phase 2 trials.
In this webinar, our goal is to review different types of Bayesian and frequentist designs that may be used for expansion cohorts in phase 1 trials and in single-arm and randomized phase 2 trials in oncology.
– We will start by reviewing the recent evolution and general trends in the design of early-phase trials to investigate the activity of targeted agents and immunotherapy.
– We will then summarize the most salient features of Bayesian and frequentist designs in this setting.
– Finally, we will cover different statistical caveats, including early consideration of randomization, issues related to sample-size calculation and the efficient use of biomarkers.
- Be updated on current trends in expansion cohorts and phase-2 designs in oncology
- Gain insights into the advantages and disadvantages of various Bayesian and frequentist approaches to the design of early-phase trials to assess activity