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Publication Detail
Evaluating many treatments and biomarkers in oncology: a new design.
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Publication Type:Journal article
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Publication Sub Type:Journal Article
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Authors:Kaplan R, Maughan T, Crook A, Fisher D, Wilson R, Brown L, Parmar M
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Publication date:20/12/2013
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Pagination:4562, 4568
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Journal:J Clin Oncol
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Volume:31
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Issue:36
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Status:Published
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Country:United States
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PII:JCO.2013.50.7905
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Language:eng
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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
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Author URL:
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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