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. Author manuscript; available in PMC: 2019 Apr 15.
Published in final edited form as: Clin Cancer Res. 2018 Feb 6;24(8):1795–1804. doi: 10.1158/1078-0432.CCR-17-3351

Figure 4. Model clinical trial scheme testing an investigational drug in patients with HER2-positive BCBMs.

Figure 4

A clinical trial testing a rationally chosen drug in patients with HER2-positive BCBMs should consist of two cohorts: Cohort A, an open-label, single-arm, two-stage, phase II cohort: the ‘efficacy cohort’; and, Cohort B, a pre-surgical window cohort: the ‘biomarker evaluation / discovery’ cohort (see text for details). Patients are treated with a trastuzmab (T) backbone in combination with drug X. For Cohort A, the primary endpoint would be CNS objective response rate as measured by RANO-BM criteria. For Cohort B, the primary endpoint would be inhibition of drug-specific pharmacodynamic markers in resected brain tumor tissue. Secondary clinical endpoints will include other pharmacodynamic biomarkers e.g. change in cfDNA with treatment, in addition to clinical outcomes.