Trial results for the Phase 2 study (NCT04828486) investigating the combination of futibatinib and pembrolizumab for the treatment of advanced or metastatic FGF19 positive BCLC Stage A, B, or C liver cancer were posted on ClinicalTrials.gov on 2025-10-29. The study reported an Overall Response Rate (ORR) of 0 participants among the enrolled patients.
Background
This study investigated the combination of futibatinib and pembrolizumab in patients with FGF19 positive BCLC stage A, B, or C liver cancer that had spread to other parts of the body (advanced or metastatic). Futibatinib is described as potentially stopping the growth of cancer cells by blocking enzymes, while pembrolizumab, an immunotherapy with monoclonal antibodies, may help the body's immune system attack cancer and interfere with tumor cell growth and spread.
Trial design
The study (NCT04828486) was a Phase 2 trial with an enrollment of 14 participants. The conditions investigated included Advanced Hepatocellular Carcinoma, BCLC Stage A Hepatocellular Carcinoma, BCLC Stage B Hepatocellular Carcinoma, BCLC Stage C Hepatocellular Carcinoma, and Metastatic Hepatocellular Carcinoma. Participants received treatment with both futibatinib and pembrolizumab.
Key results
The trial reported the following key measurements for the treatment group (Futibatinib, Pembrolizumab):
- Progression-free Survival (PFS) was 23.1 percentage of participants.
- Overall Response Rate (ORR) was 0 Participants.
- Overall Survival (OS) had a median of 55.7 weeks.
- The Incidence of Adverse Events was observed in 9 Participants.
- The Change in Quality of Life (QOL) showed a median of -10.68 score on a scale.
What this means
The results from this Phase 2 trial indicate that the combination of futibatinib and pembrolizumab did not achieve an objective response in any of the 14 participants with advanced or metastatic FGF19-positive liver cancer, as evidenced by the 0% Overall Response Rate. While a Progression-free Survival of 23.1% and a median Overall Survival of 55.7 weeks were observed, the lack of objective responses suggests limited efficacy of this specific combination in this patient population. Additionally, 9 out of 14 participants experienced adverse events, and there was a negative median change in quality of life, indicating potential tolerability challenges.
Source
The information regarding these trial results was obtained from ClinicalTrials.gov, a public database of clinical studies. The results for study NCT04828486, titled "Futibatinib and Pembrolizumab for Treatment of Advanced or Metastatic FGF19 Positive BCLC Stage A, B, or C Liver Cancer," were posted on 2025-10-29 on clinicaltrials.gov.