Phase I-II clinical trial design: a state-of-the-art paradigm for dose finding.

ABSTRACT:

Background

Conventional phase I algorithms for finding a phase-2 recommended dose (P2RD) based on toxicity alone is problematic because the maximum tolerated dose (MTD) is not necessarily the optimal dose with the most desirable risk-benefit trade-off. Moreover, the increasingly common practice of treating an expansion cohort at a chosen MTD has undesirable consequences that may not be obvious.

Patients and methods

We review the phase I-II paradigm and the EffTox design, which utilizes both efficacy and toxicity to choose optimal doses for successive patient cohorts and find the optimal P2RD. We conduct a computer simulation study to compare the performance of the EffTox design with the traditional 3 + 3 design and the continuous reassessment method.

Results

By accoun

SUBMITTER: Yan F 

PROVIDER: S-EPMC5888967 | biostudies-literature | 2018 Mar

REPOSITORIES: biostudies-literature