Futibatinib in Patients With Specific FGFR Aberrations

NCT04189445 | Terminated Phase 2 Results FDA Drug

• 2 other identifiers: TAS-120-2022019-004084-49
• Study Type: interventional
• Target: 115
• Locations: 15 countries
• Sites: 61

Brief Summary

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.

Conditions

Advanced or Metastatic Solid Tumor; Advanced or Metastatic Gastric or Gastroesophageal Cancer; Myeloid or Lymphoid Neoplasms (MLN)

Keywords

Futibatinib; Gastric cancer; Gastro-esophageal junction cancer; Solid tumor; Myeloid neoplasm; Lymphoid neoplasm; FGFR; Amplification; Rearrangement; TAS-120

Outcome Measures

Primary Outcomes (2)

• 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 Outcomes (16)

• ORR Based on Investigator Assessment in Cohorts A and B

  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

  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

  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

  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

  At the end of every 2 cycles until disease progression (Up to 31 months)

Study Arms (3)

Futibatinib (Cohort A)

EXPERIMENTAL

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 (Cohort B)

EXPERIMENTAL

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 (Cohort C)

EXPERIMENTAL

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

Interventions

Futibatinib DRUG

Futibatinib tablets were dosed orally every day on a continuous 28-day cycle

Also known as: TAS-120

Futibatinib (Cohort A); Futibatinib (Cohort B); Futibatinib (Cohort C)

Eligibility Criteria

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

You may qualify if:

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

You may not qualify if:

• 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.
• Prior treatment with an FGFR inhibitor
• Brain metastases that are untreated or clinically or radiologically unstable (that is, have been stable for \<1 month)

Sponsors & Collaborators

• Taiho Oncology, Inc.: lead

Study Sites (61)

Banner MD Anderson Cancer Center

Gilbert, Arizona, 85234-2165, United States

Location

UCLA Medical Center

Los Angeles, California, 90404, United States

Location

Georgetown University - Lombardi Comprehensive Cancer Center

Washington D.C., District of Columbia, 20007, United States

Location

University of Chicago Comprehensive Cancer Center

Chicago, Illinois, 60637, United States

Location

University of Maryland

Baltimore, Maryland, 21201, United States

Location

Beth Israel Deaconess Medical Center

Boston, Massachusetts, 02215-5400, United States

Location

Henry Ford Hospital

Woodhaven, Michigan, 48183, United States

Location

Mercy Clinic Oncology and Hematology - Coletta

Oklahoma City, Oklahoma, 73120, United States

Location

Fox Chase Cancer Center

Philadelphia, Pennsylvania, 53705, United States

Location

Houston Methodist Cancer Center

Houston, Texas, 77030, United States

Location

The University of Texas M. D. Anderson Cancer Center

Houston, Texas, 77030, United States

Location

Institut Jules Bordet

Brussels, 1000, Belgium

Location

Centre Antoine Lacassagne

Nice, Alpes Maritimes, 06200, France

Location

Centre Paul Strauss

Strasbourg, Bas Rhin, 67000, France

Location

Centre Georges François Leclerc

Dijon, Côte-d'Or, 21079, France

Location

Institut Bergonié

Bordeaux, Gironde, 33076, France

Location

Hôpital Saint-Louis

Paris, Paris, 75475, France

Location

Centre Léon Bérard

Lyon, Rhone, 69008, France

Location

Centre Hospitalier Lyon Sud

Pierre-Bénite, Rhone, 69495, France

Location

Institut Gustave Roussy

Villejuif, Val De Marne, 94805, France

Location

Universitaetsklinikum Freiburg

Freiburg im Breisgau, Baden-Wurttemberg, 79106, Germany

Location

Universitaetsklinikum Heidelberg

Heidelberg, Baden-Wurttemberg, 69120, Germany

Location

Universitaetsklinikum Koeln

Cologne, North Rhine-Westphalia, 50924, Germany

Location

The University of Hong Kong

Hong Kong, Hong Kong

Location

Hong Kong United Oncology Centre

Jordon, 0000, Hong Kong

Location

Istituto Scientifico Romagnolo Per Lo Studio e La Cura Dei Tumori

Meldola, Forli - Cesena, 47014, Italy

Location

Ospedale Sacro Cuore Don Calabria

Negrar, Verona, 37024, Italy

Location

Azienda Ospedaliera Universitaria Careggi

Florence, 50134, Italy

Location

IEO Istituto Europeo di Oncologia

Milan, 20141, Italy

Location

Azienda Ospedaliera Universitaria Integrata Verona (Ospedale Borgo Trento)

Verona, 37124, Italy

Location

Aichi Cancer Center Hospital

Nagoya, Aichi-ken, 464-8681, Japan

Location

National Cancer Center Hospital East

Kashiwa-shi, Chiba, 277-8577, Japan

Location

NHO Shikoku Cancer Center

Matsuyama, Ehime, 791-0280, Japan

Location

Hokkaido University Hospital

Sapporo, Hokkaido, 060-8648, Japan

Location

Osaka University Hospital

Suita-shi, Osaka, 565-0871, Japan

Location

National Cancer Center Hospital

Chūōku, Tokyo-To, 104-0045, Japan

Location

Antoni van Leeuwenhoek

Amsterdam, 1066 CX, Netherlands

Location

Erasmus Medisch Centrum

Rotterdam, 3015 AA, Netherlands

Location

Centro Hospitalar de Lisboa Central, E.P.E. - Hospital de Santo António dos Capuchos

Lisbon, 1169-050, Portugal

Location

Fundação Champalimaud

Lisbon, 4099-001, Portugal

Location

Centro Hospitalar do Porto, E.P.E - Hospital de Santo Antonio

Porto, 4099-001, Portugal

Location

National University Cancer Institute

Singapore, 119074, Singapore

Location

Seoul National University Bundang Hospital

Seongnam-si, Gyeonggi-do, 13620, South Korea

Location

Seoul National University Hospital

Seoul, 03080, South Korea

Location

Severance Hospital, Yonsei University Health System

Seoul, 03722, South Korea

Location

Asan Medical Center

Seoul, 05505, South Korea

Location

Samsung Medical Center

Seoul, 06351, South Korea

Location

Hospital Universitari Vall d'Hebron

Barcelona, 08035, Spain

Location

Hospital Universitario Ramon y Cajal

Madrid, 28034, Spain

Location

Hospital Universitario HM Madrid Sanchinarro

Madrid, 28050, Spain

Location

Clinica Universidad de Navarra

Pamplona, 31008, Spain

Location

Hospital Universitario Virgen Macarena

Seville, 41009, Spain

Location

Instituto Valenciano de Oncologia IVO

Valencia, 46009, Spain

Location

Hospital Clinico Universitario de Valencia

Valencia, 46010, Spain

Location

Hospital Universitari i Politecnic La Fe

Valencia, 46026, Spain

Location

Karolinska universitetssjukhuset - Solna

Solna, 171 64, Sweden

Location

Akademiska Sjukhuset

Uppsala, 75185, Sweden

Location

Acibadem Adana Hospital

Adana, 01130, Turkey (Türkiye)

Location

Acibadem Maslak Hospital

Istanbul, 34457, Turkey (Türkiye)

Location

Namik Kemal University

Tekirdağ, 59100, Turkey (Türkiye)

Location

Sarah Cannon Research Institute UK

London, Greater London, W1G 6AD, United Kingdom

Location

MeSH Terms

Conditions

Neoplasm Metastasis; Stomach Neoplasms

Interventions

futibatinib

Condition Hierarchy (Ancestors)

Neoplastic Processes; Neoplasms; Pathologic Processes; Pathological Conditions, Signs and Symptoms; Gastrointestinal Neoplasms; Digestive System Neoplasms; Neoplasms by Site; Digestive System Diseases; Gastrointestinal Diseases; Stomach Diseases

Limitations and Caveats

The Sponsor made a strategic decision to terminate the study considering enrollment challenges for some of the cohorts in the trial.

Results Point of Contact

• Title: Taiho Oncology, Inc
• Organization: Taiho Oncology, Inc

clinicaltrialinfo@taihooncology.com

609-250-7336

Publication Agreements

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

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: The study consists of independent 3 cohorts (Cohorts A, B and C) and there is no difference in the treatment regimen between the cohorts

Study Record Dates

• First Submitted: December 3, 2019
• First Posted: December 6, 2019
• Study Start: August 5, 2020
• Primary Completion: November 11, 2024
• Study Completion: November 11, 2024
• Last Updated: October 8, 2025
• Results First Posted: October 8, 2025

Record last verified: 2025-09

Data Sharing

• IPD Sharing: Will not share

Locations

US (11); IT (5); HK (2); SG (1); KR (5); SE (2); ES (8); FR (8); GB (1); BE (1); JP (6); NL (2); TU (3); DE (3); PT (3)