Using circulating tumor DNA as a novel biomarker of efficacy for dose-finding designs in oncology.

ABSTRACT: Dose-finding trials are designed to identify a safe and potentially effective drug dose and schedule during the early phase of clinical trials. Historically, Bayesian adaptive dose-escalation methods in Phase I trials in cancer have mainly focussed on toxicity endpoints rather than efficacy endpoints. This is partly because efficacy readouts are often not available soon enough for dose escalation decisions. In the last decade, 'liquid biopsy' technologies have been developed, which may provide a readout of treatment response much earlier than conventional endpoints. This paper develops a novel design that uses a biomarker, circulating tumour DNA (ctDNA), with toxicity and activity outcomes in dose-finding studies. We compare the proposed approach based on repeated ctDNA measurement with ex

SUBMITTER: Chen X 

PROVIDER: S-EPMC12365363 | biostudies-literature | 2025 Aug

REPOSITORIES: biostudies-literature