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Advanced Hepatocellular CarcinomaPhase 2NCT04828486

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

A Phase 2 study testing Futibatinib, Pembrolizumab and Quality-of-Life Assessment in people with Advanced Hepatocellular Carcinoma and BCLC Stage A Hepatocellular Carcinoma, run by Mayo Clinic at 1 site in United States.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
Search all trials

Trial 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). Read more about this study

Who is it for?
Ages 18 years and older
What is being tested?
Futibatinib, Pembrolizumab and Quality-of-Life Assessment
Type of treatment
Injection or infusion
Could I receive a placebo?
No, everyone receives an active treatment
How many people
14 participants
Where
1 siteUnited States
Study dates
17 Aug 2021to 31 Aug 2025
Phase
Phase 2
Are there results?
Yes, posted in the registrySummary in the doctors' view

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 18 years and older
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • 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)
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  • 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)
  • Calcium =< 1.5 x ULN (=< 15 days prior to registration)
  • Prothrombin time/international normalized ratio/activated partial thromboplastin time (PT/INR/aPTT) =< 1.5 x ULN OR if patient is receiving anticoagulant therapy then INR or aPTT is within target range of therapy (=< 15 days prior to registration)
  • Calculated creatinine clearance >= 40 ml/min using the Cockcroft-Gault formula (=< 15 days prior to registration)
  • Child-Pugh scores of =< 7 (Child-Pugh A or B7)
  • Barcelona Clinic Liver Cancer Stage (BCLC) stage A, B, or C
  • Negative pregnancy test done =< 7 days prior to registration, for persons of childbearing potential only
  • Willing to use an adequate method of contraception from registration through 120 days after the last dose of study medication
  • NOTE: Only for a) persons of childbearing potential or b) persons able to father a child with partners of childbearing potential
  • Able to swallow oral medication
  • Provide written informed consent
  • Willingness to provide mandatory blood specimens for correlative research
  • Willingness to provide mandatory tissue specimens for correlative research
  • Willingness and the ability to comply with scheduled visits (including geographical proximity), treatment plans, laboratory tests, and other study procedures
  • Ability to complete questionnaires by themselves or with assistance

Who cannot join

  • 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
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  • 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:
  • =< 2 weeks prior to registration for nonmyelosuppressive agents or
  • =< 4 weeks prior to registration for myelosuppressive agents
  • History and/or current evidence of any of the following disorders:
  • Retinal or corneal disorder confirmed by retinal/corneal examination and considered clinically significant in the opinion of the Investigator
  • Pneumonitis or interstitial lung disease within =< 3 years prior to registration
  • Active central nervous system (CNS) metastasis and/or carcinomatous meningitis
  • NOTE: Patients with previously treated brain metastases that are clinically and radiologically stable (for at least 4 weeks prior to enrollment) are eligible
  • Corrected QT interval using Fridericia's formula (QTcF) > 480 msec
  • NOTE: Patients with an atrioventricular pacemaker or other condition (for example, right bundle branch block) that renders the QT measurement invalid are an exception and the criterion does not apply
  • Co-morbid systemic illnesses or other severe concurrent disease which, in the judgment of the investigator, would make the patient inappropriate for entry into this study or interfere significantly with the proper assessment of safety and toxicity of the prescribed regimens
  • Receiving any other investigational agent which would be considered as a treatment for the primary neoplasm
  • History or risk of autoimmune disease, including, but not limited to, systemic lupus erythematosus, rheumatoid arthritis, inflammatory bowel disease, vascular thrombosis associated with antiphospholipid syndrome, Wegener's granulomatosis, Sjogren's syndrome, Guillain-Barre syndrome, multiple sclerosis, autoimmune thyroid disease, vasculitis, or glomerulonephritis. Notes:
  • Patients with a history of autoimmune hypothyroidism on a stable dose of thyroid replacement hormone are eligible.
  • Patients with controlled Type 1 diabetes mellitus on a stable insulin regimen are eligible.
  • Patients with eczema, psoriasis, lichen simplex chronicus of vitiligo with dermatologic manifestations only (e.g., patients with psoriatic arthritis would be excluded) are permitted provided that they meet the following conditions:
  • Must not have ocular manifestations
  • Rash must cover less than 10% of body surface area (BSA)
  • Disease is well controlled at baseline and only requiring low potency topical steroids (e.g., hydrocortisone 2.5%, hydrocortisone butyrate 0.1%, flucinolone 0.01%, desonide 0.05%, aclometasone dipropionate 0.05%)
  • No acute exacerbations of underlying condition within the last 12 months (not requiring psoralen plus ultraviolet A radiation [PUVA], methotrexate, retinoids, biologic agents, oral calcineurin inhibitors; high potency or oral steroids)
  • Known active human immunodeficiency virus (HIV) infection (defined as patients who are not on anti-retroviral treatment and have detectable viral load and CD4+ < 500/ml)
  • NOTE: HIV-positive patients who are well controlled on anti-retroviral therapy are allowed to enroll
  • History and/or current evidence of any of the following disorders:
  • Non-tumor related alteration of the calcium-phosphorus homeostasis that is considered clinically significant in the opinion of the Investigator
  • Ectopic mineralization/calcification, including but not limited to soft tissue, kidneys, intestine, or myocardia and lung, considered clinically significant in the opinion of the Investigator
  • Retinal or corneal disorder confirmed by retinal/corneal examination and considered clinically significant in the opinion of the Investigator
  • Uncontrolled intercurrent illness including, but not limited to:
  • Ongoing or active severe infection
  • NOTE: Must be afebrile > 7 days to be eligible. Patient may be eligible if fever is present and infection has been ruled out or fever is related to tumor
  • Psychiatric illness/social situations that would limit compliance with study requirements
  • Clinically significant or uncontrolled cardiac disease, including unstable angina, acute myocardial infarction within 6 months from day 1 of study treatment administration, New York Heart Association class III and IV congestive heart failure, and uncontrolled arrhythmia
  • NOTE: Participants with pacemaker or with atrial fibrillation and well controlled heart rate are allowed
  • Other active malignancy <6 months prior to pre-registration
  • EXCEPTIONS: Non-melanotic skin cancer, papillary thyroid cancer, or carcinoma-in-situ of the cervix, or others curatively treated and now considered to be at less than 30% risk of relapse are eligible
  • Prior organ transplantation

Trial locations and contacts

1 site in 1 country.

United States1 site

  • Mayo Clinic in Rochester

    Rochester, Minnesota, 55905

Other trials in United States

About this study

Official title: 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

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.

Read the detailed description

PRIMARY OBJECTIVE:

  1. 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:

  1. 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:

  1. 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.

  1. 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.

Being tested
Futibatinib, Pembrolizumab, Quality-of-Life Assessment
Medical subject headings
Carcinoma, Hepatocellular

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • FutibatinibDrug

    Also called Lytgobi, TAS-120

    Given PO

  • PembrolizumabBiological

    Also called Keytruda, Lambrolizumab, MK-3475, SCH 900475

    Given IV

  • Quality-of-Life AssessmentOther

    Also called Quality of Life Assessment

    Ancillary studies

Study groups in NCT04828486
Study groupRoleWhat they receive
Treatment (futibatinib, pembrolizumab)ExperimentalPatients 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.
Receives: Drug: Futibatinib; Biological: Pembrolizumab; Other: Quality-of-Life Assessment

What is being measured

The primary outcome is the main question the study is designed to answer.

  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).

    Measured: 6 months

Secondary outcomes (4)

  1. Overall Response Rate (ORR)

    ORR defined as the number of evaluable patients achieving a response [partial response or complete response per Response Evaluation Criteria in Solid Tumors (RECIST) v1.1] during treatment. Complete Response (CR): All of the following must be true: a. Disappearance of all target lesions. b. Each target lymph node must have reduction in short axis to <1.0 cm. c. Normalization of tumor biomarkers. Partial Response (PR): At least a 30% decrease in PBSD (sum of the longest diameter for all target lesions plus the sum of the short axis of all the target lymph nodes at current evaluation) taking as reference the BSD (baseline sum of dimensions).

    Measured: 3 years

  2. Overall Survival (OS)

    OS is defined as the time from registration to death due to any cause. Median OS time and 95% CI will be reported.

    Measured: 4 years

  3. Incidence of Adverse Events

    Adverse events will be evaluated per Common Terminology Criteria for Adverse Events (CTCAE) version 5.0 for each patient. Number of participants experiencing one or more grade 3+ adverse events will be reported.

    Measured: 4 years

  4. Change in Quality of Life (QOL)

    As measured by the global health domain of the European Organization for Research and Treatment of Cancer Quality of Life Questionnaire (EORTC-QLQ-C30), version 3. Change in score between baseline and first re-staging will be calculated for each individual. Twenty-eight (28) questions are answered on a scale of 1-4 where 1=not at all, 2=a little, 3=quite a bit, and 4= very much. The final two questions are answered on a scale of 1-7 where 1= very poor and 7=excellent. Raw scores were then scored according to EORTC guidelines (Fayers PM, Aaronson NK, Bjordal K, Groenvold M, Curran D, Bottomley A on behalf of the EORTC Quality of Life Group. EORTC QLQ-C30 Scoring Manual (3rd edition). Brussels: EORTC, 2001.), so that scores are 0-100, with a higher score indicating a better quality of life.

    Measured: 3 years

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted2 Apr 2021
  2. Study start17 Aug 2021
  3. Primary completion18 Sep 2024
  4. Study completion31 Aug 2025

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 2
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
14 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT04828486
Sponsor's protocol ID
MC200402
Other IDs
NCI-2021-02444, 20-007235
Status last verified
Oct 2025
First submitted
30 Mar 2021
Last updated
18 Nov 2025
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing Futibatinib, Pembrolizumab and Quality-of-Life Assessment in people with Advanced Hepatocellular Carcinoma and BCLC Stage A Hepatocellular Carcinoma, run by Mayo Clinic at 1 site in United States.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 29 inclusion and 49 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 18 Nov 2025. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 29 Oct 2025). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Progression-free Survival (PFS) (time frame: 6 months). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT04828486.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 18 Nov 2025.

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