A new phase 2 clinical trial testing futibatinib in patients with cholangiocarcinoma (CCA) is now open.

The open-label, multinational, randomized clinical trial aims to confirm the clinical benefits of 20 mg of futibatinib and evaluate the safety and efficacy of 16 mg of the treatment in patients who have FGFR2 gene fusions or other rearrangements and have previously been treated.

Read more about the subtypes of CCA 

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An estimated 120 patients, aged 18 years and above, will be recruited and randomly divided into 2 groups. Patients in each group will receive 1 of the 2 doses of the drug once a day for 21 days.

The primary outcome measure will be the patients’ overall response rate. Secondary outcome measures will include the duration of response, progression-free survival, overall survival, treatment-emergent adverse events, and any change in quality of life from baseline.

The trial sponsored by Taiho Oncology is not yet open for recruitment. It is estimated to start on April 14, 2023 and be completed in June 2026. 

Patients who have a history or current evidence of a calcium and phosphate homeostasis disorder, current evidence of a clinically significant retinal disorder, other serious illness, or a history of major surgery in the previous 4 weeks, among other criteria, are not eligible. A full list of inclusion and exclusion criteria can be found on the trial site. 

CCA is a rare cancer of the biliary tract. Depending on the anatomical origin, there are 2 subtypes of CCA: intrahepatic and extrahepatic. Extrahepatic CCA is further divided into 2 groups, distal and perihilar CCA. 

Around 20% of patients with intrahepatic CCA have a mutation in genes coding for a fibroblast growth factor receptor (FGFR), especially fusions of the FGFR2 gene.

Futibatinib is an irreversible FGFR inhibitor that can inhibit all 4 subtypes of FGFR. It is approved by the US Food and Drug Administration (FDA) for the treatment of adult patients with previously treated, unresectable, locally advanced or metastatic intrahepatic CCA with an FGFR2 gene fusion or other rearrangement. It is sold under the brand name Lytgobi®.


Study of futibatinib in patients with advanced cholangiocarcinoma with FGFR2 fusion or rearrangement (FOENIX-CCA4). ClinicalTrials.gov. February 14, 2023, Accessed March 1, 2023.