Abstract / Summary
Purpose: TAPUR is a phase II basket trial evaluating the antitumor activity of commercially available targeted agents in patients with advanced cancer and genomic alterations. Results of two cohorts of patients with various solid tumors with FGFR1 or FGFR2 alterations treated with sunitinib are reported.
Methods: Eligible patients had measurable disease, Eastern Cooperative Oncology Group performance status 0-2, adequate organ function, and no remaining standard treatment options. The primary end point was disease control (DC), defined as objective response (OR) or stable disease (SD) of at least 16 weeks duration (SD16+). Low accruing histology-specific cohorts with FGFR1 or FGFR2 alterations treated with sunitinib were collapsed into two histology-pooled cohorts for this analysis. The hypothesized null DC rate of 15% was evaluated by a 1-sided exact binomial test (alpha .10; 82% power) per cohort. Secondary end points included OR, progression-free survival, overall survival, duration of response, duration of SD, and safety.
Results: Sixty-four patients with 20 different cancer types with FGFR1 (N = 36) or FGFR2 (N = 28) alterations were enrolled from April 2016 to June 2024 and collapsed into two histology-pooled cohorts (FGFR1 and FGFR2) for analysis. Eight patients (all in the FGFR1 cohort) were not evaluable for efficacy. DC rates for the FGFR1 and FGFR2 cohorts were 21% (one-sided 90% CI, 12 to 100) and 39% (one-sided 90% CI, 27 to 100), respectively. The null hypothesis was not rejected for the FGFR1 cohort (P = .24) but was rejected for the FGFR2 cohort (P = .0015). Twenty patients had at least one treatment-related grade 3-5 adverse event (AE) or serious AE.
Conclusion: Sunitinib met the prespecified criteria to declare a signal of activity in patients with various solid tumors and FGFR2 alterations but not FGFR1 alterations.