Preliminary results were presented from this phase 2 clinical trial by Lipika Goyal, MD, MPhil, Medical Oncologist, Tucker Gosnell Center for Gastrointestinal Cancers, Massachusetts General Hospital, Boston, at the 2020 ASCO annual meeting.
The goal of the study is to assess the results of futibatinib, an investigational FGFR inhibitor, for patients with intrahepatic cholangiocarcinoma (CCA) who have FGFR2 fusions or other FGFR2 rearrangements.
Patients with these characteristics were enrolled if they had locally advanced or metastatic, unresectable cancer, had disease progression (getting worse) after 1 or more lines of systemic chemotherapy, and had received no previous therapy with an FGFR inhibitor.
A total of 103 patients enrolled in the study were given 20 mg of futibatinib orally, once daily, until disease progression, drug intolerance, withdrawal of consent, or death.
Promising Results
The following data were available for 67 patients who had 6 months or longer of follow-up. Of these patients, 45% had had 1 previous chemotherapy treatment, 28% had 2 lines of chemotherapy, and 27% of the patients had 3 or more lines of chemotherapy.
Measuring change in tumor from baseline (the start of the study), the objective response rate in these patients was 37.3%, and the disease control rate was 82.1%. Responses were durable, with an average duration of response of 8.3 months.
Side Effects
Serious treatment-related side effects occurred in 10.4% of the patients. The most frequent treatment-related adverse events, each of these occurring in more than 20% of patients, included hyperphosphatemia (81%), diarrhea (37%), dry mouth (33%), alopecia (30%), dry skin (27%), and fatigue (24%). The safety profile was deemed consistent with this drug class of FGFR inhibitors.
The data support the efficacy and safety of futibatinib in patients with CCA and FGFR2 genetic alterations whose disease progressed after chemotherapy.
This clinical trial is closed to new enrollees but a phase 3 clinical trial of futibatinib versus chemotherapy is enrolling new patients at www.ClinicalTrials.gov (NCT04093362).