A mutation-specific, single-arm, phase 2 study of dovitinib in patients with advanced malignancies

Matthew H. Taylor, Ajjai S. Alva, Timothy Larson, Sebastian Szpakowski, Das Purkaystha, Alpesh Amin, Linda Karpiak, Sarina A. Piha-Paul

Research output: Contribution to journalArticlepeer-review

2 Scopus citations


Background: Receptor tyrosine kinases (RTKs) play key roles in tumorigenesis. The multi-RTK inhibitor dovitinib has demonstrated promising antitumor activity in multiple cancers. Patients and Methods: In this phase 2, open-label, single-arm study, patients with advanced malignancies with RTK-pathway genetic aberrations whose disease progressed on/following standard treatment received dovitinib (500 mg/day; 5-days-on/2-days-off). The primary endpoint was clinical benefit rate (CBR; complete response, partial response [PR], or stable disease [SD] for ≥ 16 weeks). Results: Of 80 patients enrolled, common tumors included gastrointestinal stromal tumors (GIST; 20.0%), colorectal cancer (CRC; 18.8%), and ovarian cancer (10.0%). Patients were heavily pretreated (median prior lines = 4; 67.5% had ≥ 3 prior lines). Genetic aberrations included cKIT (28.8%), FGFR3 (15.0%), and RET (15.0%). The CBR was 13.8%; one PR (GIST) and 10 SD (adenoid cystic [n = 3]; ovarian [n = 3]; GIST [n = 2]; CRC [n = 1]; gastroesophageal junction [n = 1]). The most common treatment-related adverse events were fatigue, diarrhea, nausea, and vomiting. Conclusions: In this heterogeneous patient population, the safety profile was acceptable for dovitinib therapy. A subset of patients with RTK pathway-activated tumors experienced clinical benefit. However, the primary endpoint was not met, suggesting further refinement of predictive biomarkers is required.

Original languageEnglish (US)
Pages (from-to)1235-1243
Number of pages9
Issue number14
StatePublished - Apr 1 2020


  • Advanced malignancies
  • Basket trial
  • Dovitinib
  • Histology-agnostic
  • Mutation-specific

ASJC Scopus subject areas

  • Oncology


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