Futibatinib in Patients With Specific FGFR Aberrations

A Phase 2 Study of Futibatinib in Patients With Specific FGFR Aberrations

The purpose of this study is to evaluate the efficacy and safety of futibatinib in patients with FGFR aberrations in 3 distinct cohorts. Patients will be enrolled into one of 3 cohorts: patients with advanced, metastatic or locally-advanced solid tumors harboring FGFR1-4 rearrangements (excluding primary brain tumors and intrahepatic cholangiocarcinoma [iCCA]); patients with gastric or gastro-esophageal junction (GEJ) cancer harboring FGFR2 amplification; and patients with myeloid or lymphoid neoplasms with FGFR1 rearrangements.

Study Overview

• Status: Terminated
• Conditions: Advanced or Metastatic Solid Tumor; Advanced or Metastatic Gastric or Gastroesophageal Cancer; Myeloid or Lymphoid Neoplasms (MLN)
• Intervention / Treatment: Drug: Futibatinib

Detailed Description

Study TAS-120-202 is an open-label, multinational, 3-arm Phase 2 study evaluating the efficacy, safety, tolerability, PK, and pharmacodynamics of futibatinib in patients with FGFR aberrations. Eligible patients will be assigned to 1 of 3 treatment cohorts based on diagnosis and FGFR gene aberration status.

Patients will receive futibatinib at an oral dose of 20 mg once a day on a continuous 28-day cycle.

The study will enroll approximately:

• Cohort A: 60 patients with locally advanced, advanced, or metastatic solid tumor harboring FGFR rearrangements other than primary brain tumor or iCCA;
• Cohort B: 35 patients with locally-advanced, advanced, or metastatic gastric cancer or gastro-esophageal junction (GEJ) with FGFR2 amplification;
• Cohort C: 20 patients with myeloid or lymphoid neoplasms (MLN) with FGFR1 rearrangements

Treatment in all cohorts will continue until disease progression, unacceptable toxicity, or any other of the criteria for treatment discontinuation is met. For patients who discontinue treatment for reasons other than disease progression, tumor assessments should be continued until radiologic disease progression is documented or until initiation of subsequent new anticancer therapy (whichever occurs first).

Patients will be followed for survival every 12 weeks (±2 weeks) until survival events (deaths) have been reported for 75% of enrolled patients or the study is terminated early by the Sponsor.

Additional cohorts may be added in the future in case of new emerging efficacy data.

• Study Type: Interventional
• Enrollment (Actual): 115
• Phase: Phase 2

Contacts and Locations

Study Locations

• Belgium
  • Brussels, Belgium, 1000
    • Institut Jules Bordet
• France
  • Alpes Maritimes
    • Nice, Alpes Maritimes, France, 06200
      • Centre Antoine Lacassagne
  • Bas Rhin
    • Strasbourg, Bas Rhin, France, 67000
      • Centre Paul Strauss
  • Côte-d'Or
    • Dijon, Côte-d'Or, France, 21079
      • Centre Georges François Leclerc
  • Gironde
    • Bordeaux, Gironde, France, 33076
      • Institut Bergonie
  • Paris
    • Paris, Paris, France, 75475
      • Hôpital Saint-Louis
  • Rhone
    • Lyon, Rhone, France, 69008
      • Centre Léon Bérard
    • Pierre-Bénite, Rhone, France, 69495
      • Centre Hospitalier LYON SUD
  • Val De Marne
    • Villejuif, Val De Marne, France, 94805
      • Institut Gustave Roussy
• Germany
  • Baden-Wurttemberg
    • Freiburg im Breisgau, Baden-Wurttemberg, Germany, 79106
      • Universitaetsklinikum Freiburg
    • Heidelberg, Baden-Wurttemberg, Germany, 69120
      • Universitaetsklinikum Heidelberg
  • North Rhine-Westphalia
    • Cologne, North Rhine-Westphalia, Germany, 50924
      • Universitaetsklinikum Koeln
• Hong Kong
  • Hong Kong, Hong Kong
    • The University of Hong Kong
  • Jordon, Hong Kong, 0000
    • Hong Kong United Oncology Centre
• Italy
  • Florence, Italy, 50134
    • Azienda Ospedaliera Universitaria Careggi
  • Milan, Italy, 20141
    • IEO Istituto Europeo di Oncologia
  • Verona, Italy, 37124
    • Azienda Ospedaliera Universitaria Integrata Verona (Ospedale Borgo Trento)
  • Forli - Cesena
    • Meldola, Forli - Cesena, Italy, 47014
      • Istituto Scientifico Romagnolo per lo Studio e la Cura dei Tumori
  • Verona
    • Negrar, Verona, Italy, 37024
      • Ospedale Sacro Cuore Don Calabria
• Japan
  • Aichi-ken
    • Nagoya, Aichi-ken, Japan, 464-8681
      • Aichi Cancer Center Hospital
  • Chiba
    • Kashiwa-shi, Chiba, Japan, 277-8577
      • National Cancer Center Hospital East
  • Ehime
    • Matsuyama, Ehime, Japan, 791-0280
      • NHO Shikoku Cancer Center
  • Hokkaido
    • Sapporo, Hokkaido, Japan, 060-8648
      • Hokkaido University Hospital
  • Osaka
    • Suita-shi, Osaka, Japan, 565-0871
      • Osaka University Hospital
  • Tokyo-To
    • Chūōku, Tokyo-To, Japan, 104-0045
      • National Cancer Center Hospital
• Netherlands
  • Amsterdam, Netherlands, 1066 CX
    • Antoni van Leeuwenhoek
  • Rotterdam, Netherlands, 3015 AA
    • Erasmus Medisch Centrum
• Portugal
  • Lisbon, Portugal, 1169-050
    • Centro Hospitalar de Lisboa Central, E.P.E. - Hospital de Santo Antonio dos Capuchos
  • Lisbon, Portugal, 4099-001
    • Fundacao Champalimaud
  • Porto, Portugal, 4099-001
    • Centro Hospitalar do Porto, E.P.E - Hospital de Santo Antonio
• Singapore
  • Singapore, Singapore, 119074
    • National University Cancer Institute
• South Korea
  • Seoul, South Korea, 03080
    • Seoul National University Hospital
  • Seoul, South Korea, 05505
    • Asan Medical Center
  • Seoul, South Korea, 06351
    • Samsung Medical Center
  • Seoul, South Korea, 03722
    • Severance Hospital, Yonsei University Health System
  • Gyeonggi-do
    • Seongnam-si, Gyeonggi-do, South Korea, 13620
      • Seoul National University Bundang Hospital
• Spain
  • Barcelona, Spain, 08035
    • Hospital Universitari Vall d'Hebron
  • Madrid, Spain, 28034
    • Hospital Universitario Ramon y Cajal
  • Madrid, Spain, 28050
    • Hospital Universitario Hm Madrid Sanchinarro
  • Pamplona, Spain, 31008
    • Clinica Universidad de Navarra
  • Seville, Spain, 41009
    • Hospital Universitario Virgen Macarena
  • Valencia, Spain, 46026
    • Hospital Universitari i Politecnic La Fe
  • Valencia, Spain, 46010
    • Hospital Clínico Universitario de Valencia
  • Valencia, Spain, 46009
    • Instituto Valenciano de Oncologia IVO
• Sweden
  • Solna, Sweden, 171 64
    • Karolinska Universitetssjukhuset - Solna
  • Uppsala, Sweden, 75185
    • Akademiska Sjukhuset
• Turkey (Türkiye)
  • Adana, Turkey (Türkiye), 01130
    • Acibadem Adana Hospital
  • Istanbul, Turkey (Türkiye), 34457
    • Acibadem Maslak Hospital
  • Tekirdağ, Turkey (Türkiye), 59100
    • Namik Kemal University
• United Kingdom
  • Greater London
    • London, Greater London, United Kingdom, W1G 6AD
      • Sarah Cannon Research Institute UK
• United States
  • Arizona
    • Gilbert, Arizona, United States, 85234-2165
      • Banner MD Anderson Cancer Center
  • California
    • Los Angeles, California, United States, 90404
      • UCLA Medical Center
  • District of Columbia
    • Washington D.C., District of Columbia, United States, 20007
      • Georgetown University - Lombardi Comprehensive Cancer Center
  • Illinois
    • Chicago, Illinois, United States, 60637
      • University of Chicago Comprehensive Cancer Center
  • Maryland
    • Baltimore, Maryland, United States, 21201
      • University of Maryland
  • Massachusetts
    • Boston, Massachusetts, United States, 02215-5400
      • Beth Israel Deaconess Medical Center
  • Michigan
    • Woodhaven, Michigan, United States, 48183
      • Henry Ford Hospital
  • Oklahoma
    • Oklahoma City, Oklahoma, United States, 73120
      • Mercy Clinic Oncology and Hematology - Coletta
  • Pennsylvania
    • Philadelphia, Pennsylvania, United States, 53705
      • Fox Chase Cancer Center
  • Texas
    • Houston, Texas, United States, 77030
      • The University of Texas M. D. Anderson Cancer Center
    • Houston, Texas, United States, 77030
      • Houston Methodist Cancer Center

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

1. Eastern Cooperative Oncology Group (ECOG) performance status 0 or 1

2. Known FGFR aberration status and tumor type that meet all of the criteria for 1 of the following cohorts:

   a. Cohort A

   i. Histologically-confirmed, locally-advanced, advanced, or metastatic solid tumors harboring a FGFR1-4

ii. Measurable disease per Response Evaluation Criteria in Solid Tumors (RECIST) Version 1.1

iii. Had disease progression/recurrence after standard treatment for their cancer

b. Cohort B

i. Histologically-confirmed, locally-advanced, advanced, or metastatic gastric or gastroesophageal junction cancer harboring a FGFR2 amplification.

ii. Measurable disease per RECIST 1.1

iii. Received at least 2 prior systemic regimens for advanced/metastatic disease

iv. Experienced disease progression/recurrence during or after the most recent prior systemic treatment for advanced/metastatic gastric cancer or GEJ cancer

c. Cohort C

i. Confirmed myeloid or lymphoid neoplasms as defined by WHO criteria with a FGFR1 rearrangement

ii. Not a candidate for hematological stem cell transplant (HSCT) or relapsed after HSCT and donor lymphocyte infusion, and progressed and not a candidate for other therapies

Exclusion Criteria:

1. History and/or current evidence of any of the following disorders:

   1. Non-tumor related alteration of the calcium-phosphorus homeostasis that is considered clinically significant in the opinion of the Investigator
   2. 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
   3. Retinal or corneal disorder confirmed by retinal/corneal examination and considered clinically significant in the opinion of the Investigator.
2. Prior treatment with an FGFR inhibitor
3. Brain metastases that are untreated or clinically or radiologically unstable (that is, have been stable for <1 month)

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Non-Randomized
• Interventional Model: Parallel Assignment
• Masking: None (Open Label)

Number of Arms

3

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Futibatinib (Cohort A); Participants with advanced or metastatic solid tumors harboring FGFR1-4 rearrangements received futibatinib 20 mg, oral tablets, once a day on a continuous 28-day cycle up to a maximum of 841 days.	Drug: Futibatinib; Futibatinib tablets were dosed orally every day on a continuous 28-day cycle; Other Names: TAS-120
Experimental: Futibatinib (Cohort B); Participants with advanced or metastatic solid gastric or GEJ cancer harboring FGFR2 amplification received futibatinib 20 mg, oral tablets, once a day on a continuous 28-day cycle up to a maximum of 297 days.	Drug: Futibatinib; Futibatinib tablets were dosed orally every day on a continuous 28-day cycle; Other Names: TAS-120
Experimental: Futibatinib (Cohort C); Participants with myeloid or lymphoid neoplasm harboring FGFR1 rearrangement were to receive futibatinib 20 mg, oral tablets, once a day on a continuous 28-day cycle until disease progression, unacceptable toxicity or other treatment discontinuation criteria are met.	Drug: Futibatinib; Futibatinib tablets were dosed orally every day on a continuous 28-day cycle; Other Names: TAS-120

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Objective Response Rate (ORR) Based on Independent Central Review (IRC) in Cohorts A and B	ORR was defined as the percentage of participants experiencing a best overall response of partial response (PR) or complete response (CR) (per Response Evaluation Criteria in Solid Tumors, RECIST version 1.1), based on IRC of radiological images. CR was defined as disappearance of all target lesions. Any pathological lymph node must have reduction in short axis to <10 millimeters (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. ORR was calculated based on the best overall response recorded from the start of treatment until progressive disease or start of subsequent new anticancer treatment. Percentages were rounded off to the nearest single decimal place.	At the end of every 2 cycles until disease progression (Up to 31 months)
Complete Response (CR) Rate in Cohort C	CR rate was defined as the percentage of participants who achieved a CR (per response criteria for myeloid or lymphoid neoplasm) based on investigator assessment of imaging, peripheral blood, and bone marrow. CR was defined as disappearance of all target lesions. Any pathological lymph node must have reduction in short axis to <10 mm.	At the end of every 2 cycles until disease progression (Up to 31 months)

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
ORR Based on Investigator Assessment in Cohorts A and B	ORR was defined as the percentage of participants experiencing a best overall response of PR or CR (per RECIST 1.1), based on investigator assessment. CR was defined as disappearance of all target lesions. Any pathological lymph node must have 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. ORR was calculated based on the best overall response recorded from the start of treatment until progressive disease or start of subsequent new anticancer treatment. Percentages were rounded off to the nearest single decimal place.	At the end of every 2 cycles until disease progression (Up to 31 months)
Duration of Response (DOR) Based on IRC in Cohorts A, B and C	DOR was defined as the time from the first documentation of response (CR or PR in based on IRC) to the first documentation of objective tumor progression or death due to any cause, whichever occurs first. CR was defined as disappearance of all target lesions. Any pathological lymph node must have 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. DOR was estimated using the Kaplan-Meier method.	At the end of every 2 cycles until disease progression (Up to 31 months)
DOR Based on Investigator Assessment in Cohorts A, B and C	DOR was defined as the time from the first documentation of response (CR or PR in based on Investigator Assessment) to the first documentation of objective tumor progression or death due to any cause, whichever occurs first. CR was defined as disappearance of all target lesions. Any pathological lymph node must have 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. DOR was estimated using the Kaplan-Meier method.	At the end of every 2 cycles until disease progression (Up to 31 months)
Progression- Free Survival (PFS) Based on IRC in Cohorts A, B and C	PFS was defined as the time from first dose of the study therapy to the date of death (any cause) or disease progression (based on IRC), whichever occurs first. The PFS was analyzed using a Kaplan-Meier method, with PFS time being censored on the date of the last disease assessment. The 95% CI for median PFS was provided using the Kaplan-Meier procedure.	At the end of every 2 cycles until disease progression (Up to 31 months)
PFS Based on Investigator Review in Cohorts A, B and C	PFS was defined as the time from first dose of the study therapy to the date of death (any cause) or disease progression (based on Investigator Review), whichever occurs first. The PFS was analyzed using a Kaplan-Meier method, with PFS time being censored on the date of the last disease assessment. The 95% CI for median PFS was provided using the Kaplan-Meier procedure.	At the end of every 2 cycles until disease progression (Up to 31 months)
Overall Survival (OS) in Cohorts A, B and C	OS was defined as the time from the date of first dose to the death date. Participants without a documented death date were censored on the last date they were known to be alive. The OS was presented using a Kaplan-Meier estimate. The 95% CI for median OS was provided using the Kaplan-Meier procedure.	Up to 31 months
Disease Control Rate (DCR) Based on IRC in Cohort A and B	DCR was defined as the percentage of participants experiencing a best overall response of stable disease (SD), PR, or CR (per RECIST 1.1), based on IRC. SD was defined as neither sufficient shrinkage to qualify for PR nor sufficient increase to qualify for progressive disease (PD), referencing the smallest sum diameters while on study. PD was defined as at least a 20% increase in the sum of target lesion diameters from the smallest on study (including baseline), with an absolute increase of ≥ 5 mm, or the appearance of new lesions. CR was defined as disappearance of all target lesions. Any pathological lymph node must have 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. Percentages were rounded off to the nearest single decimal place.	At the end of every 2 cycles until disease progression (Up to 31 months)
DCR Based on Investigator Review in Cohort A and B	DCR was defined as the percentage of participants experiencing a best overall response of SD, PR, or CR (per RECIST 1.1), based on IRC. SD was defined as neither sufficient shrinkage to qualify for PR nor sufficient increase to qualify for PD, referencing the smallest sum diameters while on study. PD was defined as at least a 20% increase in the sum of target lesion diameters from the smallest on study (including baseline), with an absolute increase of ≥ 5 mm, or the appearance of new lesions. CR was defined as disappearance of all target lesions. Any pathological lymph node must have 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. Percentages were rounded off to the nearest single decimal place.	At the end of every 2 cycles until disease progression (Up to 31 months)
CR+ Complete Response With Incomplete Hematological Recovery (CRi) Rate in Cohort C	CR+CRi rate was defined as the percentage of participants who achieved a CR or CRi.	Up to 31 months
Duration of CR in Cohort C	Duration of CR was defined as the time from the first documentation of CR to the first documentation of recurrence or death due to any cause, whichever occurs first.	Up to 31 months
Duration of CR+CRi in Cohort C	Duration of CR+CRi was defined as the time from the first documentation of CR or CRi to the first documentation of disease relapse or death due to any cause, whichever occurs first.	Up to 31 months
Complete Cytogenetic Response (CCyR) Rate in Cohort C	CCyR rate was defined as the percentage of participants who achieved a CCyR.	Up to 31 months
Partial Cytogenetic Response (PCyR) Rate in Cohort C	PCyR rate was defined as the percentage of participants who achieved a PCyR.	Up to 31 months
Relapse-free Survival (RFS) in Cohort C	RFS was defined as the time from first documentation of CR to the date of death (any cause) or disease relapse or death due to any cause, whichever occurs first.	Up to 31 months
Event-free Survival (EFS) in Cohort C	EFS was defined as the time from first dose of study therapy to treatment failure, disease relapse after CR, or participant death due to any cause, whichever occurs first.	Up to 31 months
Number of Participants With Treatment Emergent Adverse Events (TEAEs) - Cohort A, B and C	An adverse event (AE) is defined as any untoward medical occurrence in a clinical study participant and does not necessarily have a causal relationship with the study drug. A treatment-emergent AE (TEAE) is defined as an AE that is starting or worsening at the time of or after the first dose of study drug administration and within 30 days after the last dose of study drug, and does not necessarily have a causal relationship to the use of the study drug.	From the first dose of study drug up to 30 days after the last dose (Up to 31 months)

Collaborators and Investigators

• Sponsor: Taiho Oncology, Inc.

Study record dates

Study Major Dates

• Study Start (Actual): August 5, 2020
• Primary Completion (Actual): November 11, 2024
• Study Completion (Actual): November 11, 2024

Study Registration Dates

• First Submitted: December 3, 2019
• First Submitted That Met QC Criteria: December 5, 2019
• First Posted (Actual): December 6, 2019

Study Record Updates

• Last Update Posted (Estimated): October 8, 2025
• Last Update Submitted That Met QC Criteria: September 19, 2025
• Last Verified: September 1, 2025

More Information

Terms related to this study

• Keywords: Gastric cancer; Solid tumor; FGFR; Futibatinib; Rearrangement; TAS-120; Gastro-esophageal junction cancer; Myeloid neoplasm; Lymphoid neoplasm; Amplification
• Additional Relevant MeSH Terms: Pathologic Processes; Neoplasms by Site; Neoplasms; Gastrointestinal Neoplasms; Digestive System Neoplasms; Digestive System Diseases; Gastrointestinal Diseases; Stomach Diseases; Neoplastic Processes; Pathological Conditions, Signs and Symptoms; Stomach Neoplasms; Neoplasm Metastasis; Antineoplastic Agents; futibatinib
• Other Study ID Numbers: TAS-120-202; 2019-004084-49 (EudraCT Number)

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No