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

NCT04828486 | Completed Phase 2 Results DMC FDA Drug

• 3 other identifiers: MC200402NCI-2021-0244420-007235
• Study Type: interventional
• Target: 14
• Locations: 1 country
• Sites: 1

Brief 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.

Conditions

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

Outcome Measures

Primary Outcomes (1)

• Progression-free Survival (PFS)

  PFS is defined as the length of time from study registration until disease progression. For the purposes of this study, 6 months is defined as 27 weeks. Percent of patients alive and progression free will be reported, estimated using the method of Kaplan-Meier. Progression will be evaluated in this study using Response Evaluation Criteria in Solid Tumors (RECIST) guidelines (version 1.1).

  6 months

Secondary Outcomes (4)

• Overall Response Rate (ORR)

  3 years

• Overall Survival (OS)

  4 years

• Incidence of Adverse Events

  4 years

• Change in Quality of Life (QOL)

  3 years

Study Arms (1)

Treatment (futibatinib, pembrolizumab)

EXPERIMENTAL

Patients receive futibatinib PO QD on days 1-21 for cycles 1-9, and days 1-42 for subsequent cycles and pembrolizumab 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.

Drug: Futibatinib; Biological: Pembrolizumab; Other: Quality-of-Life Assessment

Interventions

Futibatinib DRUG

Given PO

Also known as: Lytgobi, TAS-120

Treatment (futibatinib, pembrolizumab)

Pembrolizumab BIOLOGICAL

Given IV

Also known as: Keytruda, Lambrolizumab, MK-3475, SCH 900475

Treatment (futibatinib, pembrolizumab)

Quality-of-Life Assessment OTHER

Ancillary studies

Also known as: Quality of Life Assessment

Treatment (futibatinib, pembrolizumab)

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Age \>= 18 years
• Adequate tissue for FGF19 testing by ribonucleic acid (mRNA) or immunohistochemistry (IHC)
• Disease characteristics:
• Radiologically or pathologically confirmed hepatocellular carcinoma (HCC) that is not eligible for curative resection, transplantation, or ablative therapies
• NOTE: Prior radiation, chemoembolization, radioembolization, or other local ablative therapies or hepatic resection are permitted
• Measurable disease by any imaging modality as defined by Response Evaluation Criteria in Solid Tumors (RECIST) 1.1 criteria in at least one site not previously treated with radiation or liver directed therapy (including bland, chemo- or radio-embolization, or ablation)
• NOTE: Tumor lesions in a previously irradiated area are not considered measurable disease; disease that is measurable by physical examination only is not eligible
• Eastern Cooperative Oncology Group (ECOG) performance status (PS) 0, 1 or 2
• Absolute neutrophil count (ANC) \>= 1500/mm\^3 (=\< 15 days prior to registration)
• Hemoglobin \>= 8.0 g/dL (=\< 15 days prior to registration)
• Platelet count \>= 75,000/mm\^3 (=\< 15 days prior to registration)
• Albumin \>= 2.5 g/dL (=\< 15 days prior to registration)
• Alanine aminotransferase (ALT) and aspartate transaminase (AST) =\< 2.5 x upper limit of normal (ULN) (or =\< 5 x ULN for patients with liver metastasis) (=\< 15 days prior to registration)
• Total bilirubin =\< 2 x ULN (=\< 15 days prior to registration)
• Phosphorus =\< 1.5 x ULN (=\< 15 days prior to registration)

You may not qualify if:

• Known standard therapy for the patient's disease that is potentially curative or definitely capable of extending life expectancy
• Eligible for first-line treatment with IMbrave150 or STRIDE regimens
• Any of the following because this study involves an investigational agent whose genotoxic, mutagenic and teratogenic effects on the developing fetus and newborn are unknown:
• Pregnant persons
• Nursing persons
• Persons of childbearing potential who are unwilling to employ adequate contraception
• Any of the following prior therapies:
• Surgery =\< 4 weeks prior to registration
• Radiotherapy for extended field =\< 4 weeks prior to registration or limited field radiotherapy =\< 2 weeks prior to registration
• Systemic anticancer therapy =\< 2 weeks prior to registration
• NOTE: Prior immunotherapy is allowed unless patient discontinued due to grade 4 adverse event (AE)
• Live vaccine =\< 30 days prior to registration
• Prior treatment with FGFR inhibitor
• Received strong inhibitors and inducers and sensitive substrates of CYP3A4 =\< 2 weeks prior to registration
• Received a drug that has not received regulatory approval for any indication as follows:

Sponsors & Collaborators

• Mayo Clinic: lead

Study Sites (1)

Mayo Clinic in Rochester

Rochester, Minnesota, 55905, United States

Location

Related Links

• Mayo Clinic Clinical Trials

MeSH Terms

Conditions

Carcinoma, Hepatocellular

Interventions

futibatinib; pembrolizumab

Condition Hierarchy (Ancestors)

Adenocarcinoma; Carcinoma; Neoplasms, Glandular and Epithelial; Neoplasms by Histologic Type; Neoplasms; Liver Neoplasms; Digestive System Neoplasms; Neoplasms by Site; Digestive System Diseases; Liver Diseases

Results Point of Contact

• Title: Nguyen H Tran MD MPH
• Organization: Mayo Clinic

Tran.Nguyen@mayo.edu

507-284-2511

Study Officials

• Nguyen H. Tran, M.D.

  Mayo Clinic in Rochester

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: No

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: March 30, 2021
• First Posted: April 2, 2021
• Study Start: August 17, 2021
• Primary Completion: September 18, 2024
• Study Completion: August 31, 2025
• Last Updated: November 18, 2025
• Results First Posted: October 29, 2025

Record last verified: 2025-10

Locations

US (1)