In the clinical trial setting, several biomarker-driven designs exist to detect heterogeneous effects. Adaptive enrichment designs initially enroll and randomize patients regardless of their biomarker …

Feb 17, 2022 · Abstract A popular design for clinical trials assessing targeted therapies is the two-stage adaptive enrichment design with recruitment in stage 2 limited to a biomarker-defined subgroup …

Jan 1, 2021 · Nevertheless, several limitations of adaptive enrichment designs have been noted. First, modification of trial participants during the mid-course may result in high uncertainty of the treatment …

Sep 27, 2024 · Background An adaptive design allows modifying the design based on accumulated data while maintaining trial validity and integrity. The final sample size may be unknown when designing …

Apr 25, 2025 · The ROCP in the R2 approach demonstrates robust performance across most effect sizes, although GSD retains superiority within the R2 approach due to its sample size boundary. …

Apr 28, 2023 · The typical maximum likelihood estimation (MLE) for the treatment effect in the fixed sample size trial may be biased in adaptive designs because of the trial design adaptation (e.g., …

Jun 5, 2023 · Flexibility of design is not a panacea, but adaptive clinical trial designs offer the potential for more efficient research Randomised controlled trials, when appropriately designed and …

Using numerical optimization and a dynamic programming approach, we determine optimal single and two-stage designs optimizing the total sample size of the trial, the prevalence of the subgroup in the …

Reviews are given of adaptive signature designs; a Bayesian design that uses a random partition to identify treatment-sensitive biomarker subgroups and assign treatments; and designs that enrich …

The challenge was especially acute, since the specific disease is known to have substantial heterogeneity driving a difference in potential response to the treatment. PhaseV developed an …