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CholangiocarcinomaPhase 1/2NCT02052778

A Study of TAS-120 in Patients With Advanced Solid Tumors

A Phase 1/2 study testing Futibatinib in people with Cholangiocarcinoma and Urothelial Cancer, run by Taiho Oncology, Inc. at 58 sites in 13 countries.

Completed

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

Official record
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Trial summary

This is an open-label, nonrandomized, Phase 1/2 study for the fibroblast growth factor receptor (FGFR) inhibitor futibatinib (TAS-120). The purpose of the study is to evaluate the safety, tolerability, pharmacokinetic, pharmacodynamic, and anti-tumor activity of futibatinib in patients with advanced solid tumors with… Read more about this study

Who is it for?
Ages 18 years and older
What is being tested?
Futibatinib
Type of treatment
Pill or oral medicine
Could I receive a placebo?
No, everyone receives an active treatment
How many people
407 participants
Where
58 sitesin 13 countries
Study dates
21 Jul 2014to 22 Oct 2024
Phase
Phase 1/2
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

  • Provide written informed consent
  • Age ≥ 18 years of age
  • Has histologically or cytologically confirmed, locally advanced or metastatic cancer
  • The following specific criteria for each study portion
  • Phase 1 (Dose Escalation):
  • Patients with any type of solid tumor
Show all 26Show fewer
  • Disease progression following standard therapies or intolerant to prior standard therapies
  • Phase 1 (Dose Expansion)
  • Have at least one FGF/FGFR aberration
  • Disease progression following standard therapies or were intolerant to prior standard therapies (including prior FGFR inhibitors).
  • Have measurable disease as defined by Response Evaluation Criteria in Solid Tumors (RECIST) guidelines (version 1.1, 2009) for advanced solid tumors or Response Assessment in Neuro-Oncology criteria (2010) for brain tumors.
  • Patients with any of the following tumor types
  • Patients with intrahepatic or extrahepatic CCA harboring FGFR2 gene fusions or other FGFR2 aberrations
  • Patients with primary CNS tumors
  • Patients with advanced urothelial carcinoma with FGFR3 fusions or FGFR3 activating mutations
  • Patients with breast cancer or gastric cancer
  • Patients with other solid tumor types harboring FGFR gene fusions or activating mutations
  • Patients with solid tumor types and other FGF/FGFR alterations not listed above
  • Phase 2
  • Patients with iCCA and FGFR2 gene rearrangements (incl fusions)
  • Have been treated with at least one prior systemic gemcitabine and platinum-based chemotherapy
  • Must have documentation of radiographic progression of disease
  • No prior FGFR inhibitor
  • Measurable disease as defined by Response Evaluation Criteria in Solid Tumors (RECIST) guidelines (version 1.1, 2009)
  • Eastern Cooperative Oncology Group (ECOG) performance status 0 or 1
  • Adequate organ function.

Who cannot join

  • History and/or current evidence of clinically significant non-tumor related alteration of calcium-phosphorus homeostasis.
  • History and/or current evidence of clinically significant ectopic mineralization/calcification.
  • History and/or current evidence of clinically significant retinal disorder
  • A serious illness or medical condition(s)
  • Pregnant or breast-feeding female

Trial locations and contacts

58 sites in 13 countries.

58 sites in 13 countries

United States26 sites
  • New Mexico Cancer Care Alliancer

    Albuquerque, New Mexico, 87106

  • Massachusetts General Hospital

    Boston, Massachusetts, 02114

  • Dana Farber Cancer Institution

    Boston, Massachusetts, 02215

  • Roswell Park Cancer Institute

    Buffalo, New York, 14263

  • University of Virginia Cancer Center

    Charlottesville, Virginia, 22903

  • Mary Crowley Cancer Research - Medical City

    Dallas, Texas, 75230

  • Wayne State Universtity (Karmanos Cancer Institute)

    Detroit, Michigan, 48201

  • City of Hope National Medical Center

    Duarte, California, 91010

  • The University of Kansas Cancer Center

    Fairway, Kansas, 66205

  • Banner MD Anderson Cancer Center

    Gilbert, Arizona, 85234

  • Greenville Health System ITOR,Clinical Research Unit

    Greenville, South Carolina, 29605

  • The University of Texas MD Anderson Cancer Center

    Houston, Texas, 77030

  • University of Wisconsin Clinical Science Center

    Madison, Wisconsin, 53792

  • Medical College of Wisconsin

    Milwaukee, Wisconsin, 53226

  • Cancer Treatment Centers of America

    Newnan, Georgia, 30265

  • Hospital of the University of Pennsylvania

    Philadelphia, Pennsylvania, 19104

  • Sidney Kimmel Cancer Center at Jefferson

    Philadelphia, Pennsylvania, 19107

  • University of Pittsburgh Medical Center Hillman Cancer Center

    Pittsburgh, Pennsylvania, 15232

  • Mayo Clinic (MN)

    Rochester, Minnesota, 55905

  • University of Utah, Huntsman Cancer Hospital

    Salt Lake City, Utah, 84112

  • UCSF Helen Diller Family Comprehensive Cancer Center. Mission Bay

    San Francisco, California, 94158

  • Mayo Clinic (AZ)

    Scottsdale, Arizona, 85259

  • Virginia Mason Cancer Center

    Seattle, Washington, 98101

  • Spartanburg Medical Center

    Spartanburg, South Carolina, 29303

  • The University of Arizona Cancer Center - North Campus

    Tucson, Arizona, 85719

  • Cancer Treatment Centers of America Zion, IL

    Zion, Illinois, 60099

Other trials in United States

France6 sites
  • Institut Bergonie

    Bordeaux, 33076

  • Hospices Civils de Lyon

    Bron, 69677

  • Centre Léon Bérard Bât

    Lyon, Cedex, 69373

  • Pitié-Salpêtrière Hospital

    Paris, 75013

  • Rennes, Centre Eugène Marquis

    Rennes, 35042

  • Institute Goustave-Roussy-DITEP

    Villejuif, 94805

Other trials in France

Japan5 sites
  • Hokkaido University Hospital

    Hokkaido, 060-8648

  • Kyoto University Hospital, Department of Clinical Pharmacology and Therapeutics

    Kyoto, 606-8507

  • Tohoku University Hospital

    Miyagi, 980-8574

  • Osaka International Cancer Institute

    Osaka, 541-8567

  • National Cancer Center Hospital

    Tokyo, 104-0045

Other trials in Japan

South Korea4 sites
  • Yonsei University, Severance Hospital (Seoul)

    Seoul, 03722

  • ASAN Medical Center (Seoul)

    Seoul, 05505

  • Samsung Medical Center (Seoul)

    Seoul, 06351

  • Seoul National University Hospital

    Seoul, 110-744

Other trials in South Korea

Spain4 sites
  • Val D'Hebron University Hospital

    Barcelona, 08035

  • Hospital Clinic i Provincial de Barcelona,

    Barcelona, 08036

  • University Hospital Ramón y Cajal

    Madrid, 28034

  • Centro Integral Oncológico Clara Campal - Hospital Universitario Madrid Sanchinarro

    Madrid

Other trials in Spain

United Kingdom4 sites
  • Guy's and St Thomas' NHS Foundation Trust

    London, SE1 9RT

  • Sarah Cannon Research Institute

    London, W1G 6AD

  • University College London Hospital

    London, W1T 7HA

  • The Christie NHS Foundation Trust

    Manchester, M20 4BX

Other trials in United Kingdom

Italy2 sites
  • Fondazione I.R.C.C.S. Istituto Neurologico Carlo Besta

    Milan, 20133

  • UOC Oncologia Medica 1 I"V - Istituto Oncologico Veneto - IRCCS

    Padova, 35128

Other trials in Italy

Taiwan2 sites
  • Cheng Kung University Hospital

    Tainan, 704

  • National Taiwan University Hospital

    Taipei, 10048

Other trials in Taiwan

Australia1 site
  • Royal Melbourne Hospital

    Melbourne

Other trials in Australia

Canada1 site
  • Sunnybrook Research Institue

    Toronto, M4N3M5

Other trials in Canada

Germany1 site
  • University Hospital Essen, West German Cancer Center, Department of Medical Oncology

    Essen, 45147

Other trials in Germany

Hong Kong1 site
  • The Chinese University of Hong Kong

    Shatin

Other trials in Hong Kong

Netherlands1 site
  • University Hospital Jenna

    Jenna, 07740

Other trials in Netherlands

About this study

Official title: Phase 1/2 Study of TAS-120 in Patients With Advanced Solid Tumors Harboring FGF/FGFR Aberrations

This is an open-label, nonrandomized, Phase 1/2 study for the fibroblast growth factor receptor (FGFR) inhibitor futibatinib (TAS-120). The purpose of the study is to evaluate the safety, tolerability, pharmacokinetic, pharmacodynamic, and anti-tumor activity of futibatinib in patients with advanced solid tumors with and without genomic FGF/FGFR abnormalities. The study will be conducted in 3 parts:

  1. Dose escalation portion to determine the -Maximum Tolerated Dose and/ or Recommended Phase 2 Dose of futibatinib.
  2. Phase 1 expansion portion to further evaluate the safety and efficacy of futibatinib in patients with tumors harboring FGF/FGFR aberrations, including patients with cholangiocarcinoma (CCA), primary central nervous system tumors, urothelial carcinoma, breast cancer, gastric cancer.
  3. Phase 2 study portion to confirm objective response rate of futibatinib in intrahepatic CCA patients with tumors harboring FGFR2 gene rearrangements (incl fusions).
Being tested
Futibatinib
Keywords
FGF, FGFR, Futibatinib, TAS-120
Medical subject headings
Cholangiocarcinoma, Breast Neoplasms, Stomach Neoplasms

What participants receive

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

  • FutibatinibDrug

    Also called TAS-120

    oral once daily dosing, 21-day cycle

Study groups in NCT02052778
Study groupRoleWhat they receive
Phase 1: Dose Escalation: QOD Dosing: 8 mgExperimentalParticipants with or without fibroblast growth factor [FGF]/fibroblast growth factor receptor [FGFR] gene abnormalities received orally TAS-120 8 milligrams (mg) orally every other day (QOD; Monday, Wednesday and Friday of each week) in a 21-day treatment cycle until disease progression, unacceptable toxicity, withdrawal of consent or death.
Receives: Drug: Futibatinib
Phase 1: Dose Escalation: QOD Dosing: 16 mgExperimentalParticipants with or without FGF/FGFR gene abnormalities received orally TAS-120 16 mg orally QOD (Monday, Wednesday and Friday of each week) in a 21-day treatment cycle until disease progression, unacceptable toxicity, withdrawal of consent or death.
Receives: Drug: Futibatinib
Phase 1: Dose Escalation: QOD Dosing: 24 mgExperimentalParticipants with or without FGF/FGFR gene abnormalities received orally TAS-120 24 mg orally QOD (Monday, Wednesday and Friday of each week) in a 21-day treatment cycle until disease progression, unacceptable toxicity, withdrawal of consent or death.
Receives: Drug: Futibatinib
Phase 1: Dose Escalation: QOD Dosing: 36 mgExperimentalParticipants with or without FGF/FGFR gene abnormalities received orally TAS-120 36 mg orally QOD (Monday, Wednesday and Friday of each week) in a 21-day treatment cycle until disease progression, unacceptable toxicity, withdrawal of consent or death.
Receives: Drug: Futibatinib
Phase 1: Dose Escalation: QOD Dosing: 56 mgExperimentalParticipants with FGF/FGFR gene abnormalities received orally TAS-120 56 mg orally QOD (Monday, Wednesday and Friday of each week) in a 21-day treatment cycle until disease progression, unacceptable toxicity, withdrawal of consent or death.
Receives: Drug: Futibatinib
Phase 1: Dose Escalation: QOD Dosing: 80 mgExperimentalParticipants with FGF/FGFR gene abnormalities received orally TAS-120 80 mg orally QOD (Monday, Wednesday and Friday of each week) in a 21-day treatment cycle until disease progression, unacceptable toxicity, withdrawal of consent or death.
Receives: Drug: Futibatinib
Phase 1: Dose Escalation: QOD Dosing: 120 mgExperimentalParticipants with FGF/FGFR gene abnormalities received orally TAS-120 120 mg orally QOD (Monday, Wednesday and Friday of each week) in a 21-day treatment cycle until disease progression, unacceptable toxicity, withdrawal of consent or death.
Receives: Drug: Futibatinib
Phase 1: Dose Escalation: QOD Dosing: 160 mgExperimentalParticipants with FGF/FGFR gene abnormalities received orally TAS-120 160 mg orally QOD (Monday, Wednesday and Friday of each week) in a 21-day treatment cycle until disease progression, unacceptable toxicity, withdrawal of consent or death.
Receives: Drug: Futibatinib
Phase 1: Dose Escalation: QOD Dosing: 200 mgExperimentalParticipants with FGF/FGFR gene abnormalities received orally TAS-120 200 mg orally QOD (Monday, Wednesday and Friday of each week) in a 21-day treatment cycle until disease progression, unacceptable toxicity, withdrawal of consent or death.
Receives: Drug: Futibatinib
Phase 1: Dose Escalation: QD Dosing: 4 mgExperimentalParticipants with or without FGF/FGFR gene abnormalities received a dose between 4 mg orally once daily (QD) in a 21-day treatment cycle until disease progression, unacceptable toxicity, withdrawal of consent or death.
Receives: Drug: Futibatinib
Phase 1: Dose Escalation: QD Dosing: 8 mgExperimentalParticipants with or without FGF/FGFR gene abnormalities received a dose between 8 mg orally QD in a 21-day treatment cycle until disease progression, unacceptable toxicity, withdrawal of consent or death.
Receives: Drug: Futibatinib
Phase 1: Dose Escalation: QD Dosing: 16 mgExperimentalParticipants with or without FGF/FGFR gene abnormalities received a dose between 16 mg orally QD in a 21-day treatment cycle until disease progression, unacceptable toxicity, withdrawal of consent or death.
Receives: Drug: Futibatinib
Phase 1: Dose Escalation: QD Dosing: 20 mgExperimentalParticipants with or without FGF/FGFR gene abnormalities received a dose between 20 mg orally QD in a 21-day treatment cycle until disease progression, unacceptable toxicity, withdrawal of consent or death.
Receives: Drug: Futibatinib
Phase 1: Dose Escalation: QD Dosing: 24 mgExperimentalParticipants with or without FGF/FGFR gene abnormalities received a dose between 24 mg orally QD in a 21-day treatment cycle until disease progression, unacceptable toxicity, withdrawal of consent or death.
Receives: Drug: Futibatinib
Phase 1: Dose Expansion Cohort 1ExperimentalParticipants with intra-hepatic or extrahepatic cholangiocarcinoma (iCCA or eCCA) harboring FGFR2 gene fusions or rearrangements and who were treated or not treated with prior FGFR inhibitors received TAS-120 20 mg tablets orally QD in each of 21-day treatment cycle until disease progression, unacceptable toxicity, withdrawal of consent or death.
Receives: Drug: Futibatinib
Phase 1: Dose Expansion: Cohort 2ExperimentalParticipants with primary central nervous system (CNS) tumors harboring FGFR gene fusions or FGFR1 activating mutations received TAS-120 20 mg tablets orally QD in each of 21-day treatment cycle until disease progression, unacceptable toxicity, withdrawal of consent or death.
Receives: Drug: Futibatinib
Phase 1: Dose Expansion: Cohort 3ExperimentalParticipants with advanced urothelial carcinoma harboring FGFR3 gene fusions or FGFR3 activating mutations received TAS-120 20 mg tablets orally QD in each of 21-day treatment cycle until disease progression, unacceptable toxicity, withdrawal of consent or death.
Receives: Drug: Futibatinib
Phase 1: Dose Expansion: Cohort 4ExperimentalParticipants with breast or gastric cancer with harboring FGFR2 amplification received TAS-120 20 mg tablets orally QD in each of 21-day treatment cycle until disease progression, unacceptable toxicity, withdrawal of consent or death.
Receives: Drug: Futibatinib
Phase 1:Dose Expansion: Cohort 5ExperimentalParticipants with tumor types harboring FGFR gene fusions or activating mutations received TAS-120 20 mg tablets orally QD in each of 21-day treatment cycle until disease progression, unacceptable toxicity, withdrawal of consent or death.
Receives: Drug: Futibatinib
Phase 1: Dose Expansion: Cohort 6ExperimentalParticipants who were not included in Cohorts 1 to 5 received TAS-120 20 mg tablets orally QD in each of 21-day treatment cycle until disease progression, unacceptable toxicity, withdrawal of consent or death.
Receives: Drug: Futibatinib
Phase 1: Dose Expansion: Sub-cohort 1ExperimentalParticipants with iCCA who were enrolled prior to the confirmation of the recommended Phase 2 dose (RP2D) received TAS-120 16 mg tablets orally QD in each of 21-day treatment cycle until disease progression, unacceptable toxicity, withdrawal of consent or death.
Receives: Drug: Futibatinib
Phase 1: Dose Expansion: Sub-cohort 2ExperimentalParticipants with other tumor types who were enrolled prior to the confirmation of the RP2D received TAS-120 16 mg tablets orally QD in each of 21-day treatment cycle until disease progression, unacceptable toxicity, withdrawal of consent or death.
Receives: Drug: Futibatinib
Phase 2ExperimentalParticipants with iCCA with tumors harboring FGFR2 gene rearrangements received TAS-120 20 mg tablets orally QD in each of 21-day treatment cycle until disease progression, unacceptable toxicity, withdrawal of consent or death.
Receives: Drug: Futibatinib

What is being measured

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

  1. Phase 1: Dose Escalation-Maximum Tolerated Dose (MTD)

    MTD:Highest dose level at which <33% of participants experience dose-limiting toxicity (DLT) during Cycle1. DLT: >=Grade(G)3: - nonhematologic toxicity, - nausea/vomiting lasting >48hrs(uncontrolled by aggressive antiemetic), - diarrhea lasting >48hrs (unresponsive to antidiarrheal drug); G4 neutropenia lasting >7days; Febrile neutropenia (ANC<1000/mm^3 with body temperature=>38.3°C/sustained temperature >=38°C for >1hr; Thrombocytopenia G4/G3 with bleeding, required blood transfusion; Corneal disorder worsened by 1 grade or more; Increased phosphorus: >=9mg/dL or >=7mg/dL lasting for >=7days or phosphate lowering therapy[PLT] for 7days); Creatinine increase (>1.5×upper limit of normal [ULN]) lasting for >=7 days associated with serum phosphorus >5.5 mg/dL(PLT=7days)/calcium×phosphorus >55 mg/dL(PLT=7days); Hypercalcemia G2 for >7days or G3; Ectopic de novo calcification in soft tissues; >G2 DLT: prevented Cycle 1 completion, inability to start Cycle 2 within 2 weeks of schedule.

    Measured: Cycle 1 (21-day cycle)

  2. Phase 1: Dose Escalation-Recommended Phase 2 Dose (RP2D) of TAS-120

    RP2D was MTD or less. MTD: Highest dose level at which <33% of participants experience DLT) during Cycle1. DLT: >=Grade(G)3: - nonhematologic toxicity, - nausea/vomiting lasting >48hrs(uncontrolled by aggressive antiemetic), - diarrhea lasting >48hrs (unresponsive to antidiarrheal drug); G4 neutropenia lasting >7days; Febrile neutropenia (ANC<1000/mm^3 with body temperature=>38.3°C/sustained temperature >=38°C for >1hr; Thrombocytopenia G4/G3 with bleeding, required blood transfusion; Corneal disorder worsened by 1 grade or more; Increased phosphorus: >=9mg/dL or >=7mg/dL lasting for >=7days or phosphate lowering therapy[PLT] for 7days); Creatinine increase (>1.5×upper limit of normal [ULN]) lasting for >=7 days associated with serum phosphorus >5.5 mg/dL(PLT=7days)/calcium×phosphorus >55 mg/dL(PLT=7days); Hypercalcemia G2 for >7days or G3; Ectopic de novo calcification in soft tissues; >G2 DLT: prevented Cycle 1 completion, inability to start Cycle 2 within 2 weeks of schedule.

    Measured: Cycle 1 (21-day cycle)

  3. Phase 1: Dose Expansion: Percentage of Participants With Objective Response

    Objective response was defined as proportion of participants who had achieved best overall response of partial response (PR) or complete response (CR) per Response Evaluation Criteria in Solid Tumors (RECIST) v1.1. The CR was defined as a disappearance of all target lesions. Any pathological lymph nodes (target or non-target) must had reduction in short axis to <10 millimeters (mm) and PR was defined as at least a 30 percent (%) decrease in the sum of diameters of target lesions. For Cohorts 1 to 6: Objective response was based on Independent Review Committee (IRC) and for pooled Sub-cohort: Objective response was based on investigator review.

    Measured: Up to approximately 50.5 months (through cut-off date 29-May-2021) for Cohorts 1 to 6; up to approximately 27.5 months (through cut-off date 30-Jun-2019) for pooled sub-cohorts

  4. Phase 2: Percentage of Participants With Objective Response

    Objective response was defined as proportion of participants who had achieved best overall response of PR or CR per RECIST v1.1. CR was defined as a disappearance of all target lesions. Any pathological lymph nodes (target or non-target) must had reduction in short axis to <10 mm and PR was defined as at least a 30% decrease in the sum of diameters of target lesions. The Phase 2 evaluation of objective response was based on central independent CT/MRI image assessment.

    Measured: Up to approximately 37.5 months (through cut-off date 29-May-2021)

Secondary outcomes (17)
  1. Phase 1: Dose Expansion: Duration of Response (DOR)

    A DOR was defined as the time (in months) from the first documentation of response (CR or PR) to the first documentation of objective progressive disease (PD) or death due to any cause, whichever occurred first. Participants who started subsequent anticancer therapy without a prior reported progression were censored at the last tumor assessments prior to initiation of the subsequent anticancer therapy. For Cohorts 1 to 6: DOR was based on IRC and for Group 3, 4, 5, 6 and for pooled Sub-cohort: DOR was based on investigator review.

    Measured: Up to approximately 50.5 months (through cut-off date 29-May-2021) for Cohorts 1 to 6; up to approximately 27.5 months (through cut-off date 30-Jun-2019) for pooled sub-cohorts

  2. Phase 2: Duration of Response (DOR)

    A DOR was defined as the time (in months) from the first documentation of response (CR or PR) to the first documentation of objective tumor progression or death due to any cause, whichever occurred first. Participants who started subsequent anticancer therapy without a prior reported progression were censored at the last tumor assessments prior to initiation of the subsequent anticancer therapy. Kaplan-Meier method was used for the analysis.

    Measured: Up to approximately 37.5 months (through cut-off date 29-May-2021)

  3. Phase 1: Dose Expansion: Disease Control Rate (DCR)

    A DCR was defined as the proportion of participants with objective evidence of CR, PR, or stable disease (SD), except that there was no requirement for a confirmation of an SD, if it is maintained for at least 6 weeks post treatment initiation. CR was defined as the disappearance of all target lesions. Any pathological lymph nodes might had reduction in short axis to <10 mm. PR was defined as at least a 30% decrease in the sum of diameters of the target lesions, taking as a reference the Baseline sum diameters. SD was defined as neither sufficient shrinkage to qualify for PR nor sufficient increase to qualify for PD, taken as a reference the smallest sum diameters while on study. For Cohorts 1 to 6: DCR was based on IRC and for pooled Sub-cohort: DCR was based on investigator review.

    Measured: Up to approximately 50.5 months (through cut-off date 29-May-2021) for Cohorts 1 to 6; up to approximately 27.5 months (through cut-off date 30-Jun-2019) for pooled sub-cohort

  4. Phase 2: Disease Control Rate (DCR)

    A DCR was defined as the proportion of participants with objective evidence of CR, PR, or SD, except that there was no requirement for a confirmation of an SD response, if it is maintained for at least 6 weeks post treatment initiation. CR was defined as the disappearance of all target lesions. Any pathological lymph nodes might had reduction in short axis to <10 mm. PR was defined as at least a 30% decrease in the sum of diameters of the target lesions, taking as a reference the Baseline sum diameters. SD was defined as neither sufficient shrinkage to qualify for PR nor sufficient increase to qualify for PD, taken as a reference the smallest sum diameters while on study. DCR was based on IRC.

    Measured: Up to approximately 37.5 months (through cut-off date 29-May-2021)

  5. Phase 1: Dose Expansion: Progression-free Survival (PFS)

    A PFS was defined as the time (in months) from the day of the first dose to the date of first documented disease progression or death (due to any cause), whichever occurred first. Participants who died without a reported disease progression were considered to have progressed on the date of their death, participants who did not progress or die were censored on the date of their last tumor assessment, participants who had no on-study assessments and did not die were censored on the first dosing date, and participants who started any subsequent anti-cancer therapy without a prior reported progression were censored at the last tumor assessment prior to initiation of the subsequent anti-cancer therapy. PFS assessment per protocol was by IRC assessment for Cohorts 1 to 6 and for pooled Sub-cohort: PFS was based on investigator review.

    Measured: up to approximately 27.5 months (through cut-off date 30-Jun-2019)

  6. Phase 2: Progression-free Survival (PFS)

    A PFS was defined as the time (in months) from the day of the first dose to the date of first objectively documented disease progression or death (any cause), whichever occurred first. Participants who had died without a reported disease progression were considered to have progressed on the date of their death, participants who did not progress or die were censored on the date of their last tumor assessment, participants who had no on-study assessments and did not die were censored on the first dosing date, and participants who started any subsequent anti-cancer therapy without a prior reported progression were censored at the last tumor assessment prior to initiation of the subsequent anti-cancer therapy. PFS was analyzed as using Kaplan-Meier estimate.

    Measured: Up to approximately 37.5 months (through cut-off date 29-May-2021)

  7. Phase 1: Dose Expansion: Overall Survival (OS)

    An OS was defined as the time (in months) from the date of the first dose to the death date. In the absence of death confirmation or for participants alive as of the OS cut-off date, survival time was censored at the date of last study follow-up, or the cut-off date, whichever was earlier.

    Measured: up to approximately 27.5 months (through cut-off date 30-Jun-2019)

  8. Phase 2: Overall Survival (OS)

    An OS was defined as the time (in months) from the date of the first dose to the death date. In the absence of death confirmation or for participants alive as of the OS cut-off date, survival time was censored at the date of last study follow-up, or the cut-off date, whichever was earlier.

    Measured: Up to approximately 37.5 months (through cut-off date 29-May-2021)

  9. Phase 2: European Quality of Life-5 Dimensions-3 Level (EQ-5D-3L) Questionnaire: Mobility Scores at Specified Visits

    EQ-5D-3L was a self-administered standardized questionnaire to assess health outcome. It comprised 5 dimensions: mobility, self-care, usual activities, pain/discomfort and anxiety/depression. Each dimension was rated on 3 levels of function: no problem, some problem and extreme problem. In this outcome measure, data were reported categorically as the number of participants who chose each category.

    Measured: Baseline, Cycle 2, 4, 7, 10, 13, 16, 19, 22, 25, 28, 31, 34, 37, 40 and end of treatment (i.e., Cycle 43 [30 months])

  10. Phase 2: European Quality of Life-5 Dimensions-3 Level (EQ-5D-3L) Questionnaire: Self-care Scores at Specified Visits

    EQ-5D-3L was a self-administered standardized questionnaire to assess health outcome. It comprised 5 dimensions: mobility, self-care, usual activities, pain/discomfort and anxiety/depression. Each dimension was rated on 3 levels of function: no problem, some problem and extreme problem. In this outcome measure, data were reported categorically as the number of participants who chose each category.

    Measured: Baseline, Cycle 2, 4, 7, 10, 13, 16, 19, 22, 25, 28, 31, 34, 37, 40 and end of treatment (i.e., Cycle 43 [30 months])

  11. Phase 2: European Quality of Life-5 Dimensions-3 Level (EQ-5D-3L) Questionnaire: Usual Activities Scores at Specified Visits

    EQ-5D-3L was a self-administered standardized questionnaire to assess health outcome. It comprised 5 dimensions: mobility, self-care, usual activities, pain/discomfort and anxiety/depression. Each dimension was rated on 3 levels of function: no problem, some problem and extreme problem. In this outcome measure, data were reported categorically as the number of participants who chose each category.

    Measured: Baseline, Cycle 2, 4, 7, 10, 13, 16, 19, 22, 25, 28, 31, 34, 37, 40 and end of treatment (i.e., Cycle 43 [30 months])

  12. Phase 2: European Quality of Life-5 Dimensions-3 Level (EQ-5D-3L) Questionnaire: Pain/Discomfort Scores at Specified Visits

    EQ-5D-3L was a self-administered standardized questionnaire to assess health outcome. It comprised 5 dimensions: mobility, self-care, usual activities, pain/discomfort and anxiety/depression. Each dimension was rated on 3 levels of function: no problem, some problem and extreme problem. In this outcome measure, data were reported categorically as the number of participants who chose each category.

    Measured: Baseline, Cycle 2, 4, 7, 10, 13, 16, 19, 22, 25, 28, 31, 34, 37, 40 and end of treatment (i.e., Cycle 43 [30 months])

  13. Phase 2: European Quality of Life-5 Dimensions-3 Level (EQ-5D-3L) Questionnaire: Anxiety/Depression Scores at Specified Visits

    EQ-5D-3L was a self-administered standardized questionnaire to assess health outcome. It comprised 5 dimensions: mobility, self-care, usual activities, pain/discomfort and anxiety/depression. Each dimension was rated on 3 levels of function: no problem, some problem and extreme problem. In this outcome measure, data were reported categorically as the number of participants who chose each category.

    Measured: Baseline, Cycle 2, 4, 7, 10, 13, 16, 19, 22, 25, 28, 31, 34, 37, 40 and end of treatment (i.e., Cycle 43 [30 months])

  14. Phase 2: Change From Baseline in EQ-5D-3L Visual Analogue Scale (VAS) at Specified Visits

    EQ-5D-3L was a self-administered standardized questionnaire to assess health outcome. It comprised 5 dimensions: mobility, self-care, usual activities, pain/discomfort and anxiety/depression. A vertical VAS allows the participants to indicate their health state that day, and ranged from 0 (worst imaginable) to 100 (best imaginable), with higher scores indicating better health state.

    Measured: Baseline, Cycle 2, 4, 7, 10, 13, 16, 19, 22, 25, 28, 31, 34, 37, 40 and end of treatment (i.e., Cycle 43 [30 months])

  15. Phase 2:Change From Baseline in European Organization for Research and Treatment of Cancer Quality of Life Questionnaire-Core 30 (EORTC QLQ-C30) Global Health Status Score at Specified Timepoints

    EORTC QLQ-C30 is a cancer-specific instrument that contains 30 questions for evaluation of new chemotherapy \& assessment of participant reported outcome. EORTC QLQ-C30 included global health status/quality of life (GHS/QOL), functional scales (physical, role, cognitive, emotional, social), symptom scales (fatigue, pain, nausea/vomiting), and 6 single items (dyspnea, appetite loss, insomnia, constipation, diarrhea, financial difficulties). Most questions from QLQ-C30 are 4-point scale (1/Not at All to 4/Very Much), except Items 29-30, which comprise GHS scale \& are 7-point scale (1/Very Poor to 7/Excellent). GHS total score is calculated as ([{Q29+Q30}/2]-1)/6*100. Answers are converted into grading scale, with values between 0 (worse outcome) to100 (best outcome). High score represents a favorable outcome with best quality of life for participant.

    Measured: Baseline, Cycle 2, 4, 7, 10, 13, 16, 19, 22, 25, 28, 31, 34, 37, 40 and end of treatment (i.e., Cycle 43 [30 months])

  16. Phase 1: Dose Expansion: Number of Participants With Any Adverse Events (AEs) and Any Serious AEs (SAEs)

    An AE was defined as any untoward medical condition that occurred in a participant from the time the informed consent form (ICF) was signed and does not necessarily had a causal relationship with the use of the product. An SAE was an AE that falls into one or more of the following categories: a. resulted in death, b. was life threatening, c. required inpatient hospitalization or prolongation of existing hospitalization, d. resulted in persistent or significant disability or incapacity, e. was a congenital anomaly/birth defect, f. other important medical event.

    Measured: From the first dose up to approximately 50.5 months (through cut-off date 29-May-2021)

  17. Phase 2: Number of Participants With Any Adverse Events (AEs) and Any Serious Adverse Events (SAEs)

    An AE was defined as any untoward medical condition that occurs in a participant from the time the informed consent form (ICF) was signed and does not necessarily had a causal relationship with the use of the product. An SAE was an AE that falls into one or more of the following categories: a. resulted in death, b. was life threatening, c. required inpatient hospitalization or prolongation of existing hospitalization, d. resulted in persistent or significant disability or incapacity, e. was a congenital anomaly/birth defect, f. other important medical event.

    Measured: From the first dose up to approximately 37.5 months (through cut-off date 29-May-2021)

Timeline

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

  1. First posted3 Feb 2014 (estimated)
  2. Study start21 Jul 2014
  3. Primary completion1 Oct 2020
  4. Study completion22 Oct 2024

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 1/2
Primary purpose
Treatment
Allocation
Non-randomised
Intervention model
Sequential
Masking
None (open label)
Enrolment
407 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT02052778
Sponsor's protocol ID
TPU-TAS-120-101
Other IDs
2013-004810-16
Status last verified
Nov 2024
First submitted
23 Jan 2014
Last updated
27 Mar 2025
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
No
Expanded access
Available

Publications

  • Goyal L, Meric-Bernstam F, Hollebecque A, Valle JW, Morizane C, Karasic TB, Abrams TA, Furuse J, Kelley RK, Cassier PA, Klumpen HJ, Chang HM, Chen LT, Tabernero J, Oh DY, Mahipal A, Moehler M, Komatsu Y, Ahn DH, Epstein RS, Halim AB, Wacheck V, He Y, Liu M, Benhadji KA, Bridgewater JA; FOENIX-CCA2 Study Investigators. Plain language summary of the FOENIX-CCA2 study: futibatinib for people with advanced bile duct cancer. Future Oncol. 2024;20(36):2811-2822. doi: 10.1080/14796694.2024.2364504. Epub 2024 Jun 17.

    PubMed 38884254
  • DiPeri TP, Zhao M, Evans KW, Varadarajan K, Moss T, Scott S, Kahle MP, Byrnes CC, Chen H, Lee SS, Halim AB, Hirai H, Wacheck V, Kwong LN, Rodon J, Javle M, Meric-Bernstam F. Convergent MAPK pathway alterations mediate acquired resistance to FGFR inhibitors in FGFR2 fusion-positive cholangiocarcinoma. J Hepatol. 2024 Feb;80(2):322-334. doi: 10.1016/j.jhep.2023.10.041. Epub 2023 Nov 14.

    PubMed 37972659
  • Goyal L, Meric-Bernstam F, Hollebecque A, Valle JW, Morizane C, Karasic TB, Abrams TA, Furuse J, Kelley RK, Cassier PA, Klumpen HJ, Chang HM, Chen LT, Tabernero J, Oh DY, Mahipal A, Moehler M, Mitchell EP, Komatsu Y, Masuda K, Ahn D, Epstein RS, Halim AB, Fu Y, Salimi T, Wacheck V, He Y, Liu M, Benhadji KA, Bridgewater JA; FOENIX-CCA2 Study Investigators. Futibatinib for FGFR2-Rearranged Intrahepatic Cholangiocarcinoma. N Engl J Med. 2023 Jan 19;388(3):228-239. doi: 10.1056/NEJMoa2206834.

    PubMed 36652354
  • Bahleda R, Meric-Bernstam F, Goyal L, Tran B, He Y, Yamamiya I, Benhadji KA, Matos I, Arkenau HT. Phase I, first-in-human study of futibatinib, a highly selective, irreversible FGFR1-4 inhibitor in patients with advanced solid tumors. Ann Oncol. 2020 Oct;31(10):1405-1412. doi: 10.1016/j.annonc.2020.06.018. Epub 2020 Jul 2.

    PubMed 32622884

Questions about this trial

What is this clinical trial studying?

A Phase 1/2 study testing Futibatinib in people with Cholangiocarcinoma and Urothelial Cancer, run by Taiho Oncology, Inc. at 58 sites in 13 countries.

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 26 inclusion and 5 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 27 Mar 2025. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 58 sites in United States, France, Japan, South Korea, Spain and 8 more countries. 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 combined phase mean?

Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.

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 20 Mar 2024). 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?

Phase 1: Dose Escalation-Maximum Tolerated Dose (MTD) (time frame: Cycle 1 (21-day cycle)). The study lists 4 primary outcomes in all. 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 NCT02052778, which also lists 4 linked publications.

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 27 Mar 2025.

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