NCT04828486

Futibatinib and Pembrolizumab for Treatment of Advanced or Metastatic FGF19 Positive BCLC Stage A, B, or C Liver Cancer

Phase II Study of FGFR Inhibitor Futibatinib in Combination With Anti-PD-1 Antibody Pembrolizumab in Patients With Advanced or Metastatic Hepatocellular Carcinoma With FGF19 Expression After First Line Therapy

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 14 (actual)
• Sponsor: Mayo Clinic · Academic / Other
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

This phase II trial studies the effect of futibatinib and pembrolizumab in treating patients with FGF19 positive BCLC stage A, B, or C liver cancer that has spread to other parts of the body (advanced or metastatic). Futibatinib may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth. Immunotherapy with monoclonal antibodies, such as pembrolizumab, may help the body's immune system attack the cancer, and may interfere with the ability of tumor cells to grow and spread. Giving futibatinib and pembrolizumab may help treat patients with FGF19 positive liver cancer.

Detailed description

PRIMARY OBJECTIVE: I. Determine the efficacy of combination of futibatinib and pembrolizumab in patients with advanced hepatocellular carcinoma (HCC) and high FGF19 expression who has received at least one line of therapy using progression free survival (PFS) at 6 months. SECONDARY OBJECTIVES: I. Assess the safety and tolerability of futibatinib and pembrolizumab combination through adverse event monitoring. II. Determine the overall objective response rate (ORR) and overall survival (OS) of patients with advanced HCC treated with futibatinib and pembrolizumab combination. III. Assess change in overall health-related quality of life, as measured by the global health domain of the European Organization for Research and Treatment of Cancer Quality of Life Questionnaire (EORTC-QLQ-C30) between baseline and at time of first-restaging scan. EXPLORATORY OBJECTIVES: I. To evaluate the prognostic effect of baseline number of circulating tumor cells (CTCs). II. To determine whether the change in number of CTCs post 2 months of treatment from baseline is associated with PFS and OS. III. To evaluate the prognostic effect of baseline circulating cell-free deoxyribonucleic acid (cfDNA). IV. To determine whether the change in cfDNA at 2 months of treatment from baseline is associated with PFS and OS. V. To correlate drug response in patient derived organoids with clinical response and characterize the tumor microenvironment. OUTLINE: Patients receive futibatinib orally (PO) once daily (QD) on days 1-21 for cycles 1-9, and days 1-42 for subsequent cycles and pembrolizumab intravenously (IV) over 30 minutes on day 1. Cycles repeat every 21 days for cycles 1-9 and every 42 days for subsequent cycles for up to 2 years in the absence of disease progression or unacceptable toxicity. After completing study treatment, patients are followed up at 30 days, every 9 weeks for up to 18 months, and then every 6 months for up to 5 years.

Conditions

• Advanced Hepatocellular Carcinoma
• BCLC Stage A Hepatocellular Carcinoma
• BCLC Stage B Hepatocellular Carcinoma
• BCLC Stage C Hepatocellular Carcinoma
• Metastatic Hepatocellular Carcinoma

Interventions

Type	Name	Description
DRUG	Futibatinib	Given PO
BIOLOGICAL	Pembrolizumab	Given IV
OTHER	Quality-of-Life Assessment	Ancillary studies

Timeline

• Start date: 2021-08-17
• Primary completion: 2024-09-18
• Completion: 2025-08-31
• First posted: 2021-04-02
• Last updated: 2025-11-18
• Results posted: 2025-10-29

Locations

1 site across 1 country: United States

Regulatory

• FDA-regulated drug study