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Publication Detail
Evaluating many treatments and biomarkers in oncology: a new design.
  • Publication Type:
    Journal article
  • Publication Sub Type:
    Journal Article
  • Authors:
    Kaplan R, Maughan T, Crook A, Fisher D, Wilson R, Brown L, Parmar M
  • Publication date:
    20/12/2013
  • Pagination:
    4562, 4568
  • Journal:
    J Clin Oncol
  • Volume:
    31
  • Issue:
    36
  • Status:
    Published
  • Country:
    United States
  • PII:
    JCO.2013.50.7905
  • Language:
    eng
  • Keywords:
    Antineoplastic Agents, Biomarkers, Tumor, Clinical Trials as Topic, Colorectal Neoplasms, Evidence-Based Medicine, Humans, Molecular Targeted Therapy, Neoplasms, Predictive Value of Tests, Prognosis, Research Design, Sample Size, Treatment Outcome
Abstract
There is a pressing need for more-efficient trial designs for biomarker-stratified clinical trials. We suggest a new approach to trial design that links novel treatment evaluation with the concurrent evaluation of a biomarker within a confirmatory phase II/III trial setting. We describe a new protocol using this approach in advanced colorectal cancer called FOCUS4. The protocol will ultimately answer three research questions for a number of treatments and biomarkers: (1) After a period of first-line chemotherapy, do targeted novel therapies provide signals of activity in different biomarker-defined populations? (2) If so, do these definitively improve outcomes? (3) Is evidence of activity restricted to the biomarker-defined groups? The protocol randomizes novel agents against placebo concurrently across a number of different biomarker-defined population-enriched cohorts: BRAF mutation; activated AKT pathway: PI3K mutation/absolute PTEN loss tumors; KRAS and NRAS mutations; and wild type at all the mentioned genes. Within each biomarker-defined population, the trial uses a multistaged approach with flexibility to adapt in response to planned interim analyses for lack of activity. FOCUS4 is the first test of a protocol that assigns all patients with metastatic colorectal cancer to one of a number of parallel population-enriched, biomarker-stratified randomized trials. Using this approach allows questions regarding efficacy and safety of multiple novel therapies to be answered in a relatively quick and efficient manner, while also allowing for the assessment of biomarkers to help target treatment.
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