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NCT04189445

Futibatinib in Patients With Specific FGFR Aberrations

Terminated Phase 2 Results posted Last updated 8 October 2025
What this trial tests

Phase 2 trial testing Futibatinib in Advanced or Metastatic Solid Tumor in 115 participants. Terminated before completion.

Timeline
5 August 2020
Primary endpoint
11 November 2024
11 November 2024

Quick facts

Lead sponsorTaiho Oncology, Inc.
PhasePhase 2
StatusTerminated
Study typeINTERVENTIONAL
Allocationnon randomized
Designparallel
Maskingnone
Primary purposetreatment
Enrollment115
Start date5 August 2020
Primary completion11 November 2024
Estimated completion11 November 2024
Sites61 locations across France, Hong Kong, Italy, Japan, Netherlands, Belgium, Sweden, United Kingdom

Drugs / interventions tested

Conditions studied

Sponsor

Taiho Oncology, Inc. — full company profile →

Who can join

18 and older, any sex, with Advanced or Metastatic Solid Tumor or Advanced or Metastatic Gastric or Gastroesophageal Cancer. Patients with the condition only — healthy volunteers not accepted.

Results — posted to ClinicalTrials.gov

Per-arm endpoint measurements with 95% confidence intervals where reported. Source: trial results section.

Objective Response Rate (ORR) Based on Independent Central Review (IRC) in Cohorts A and B Primary · At the end of every 2 cycles until disease progression (Up to 31 months)

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

GroupValue95% CI
Futibatinib (Cohort A)6.92.6 – 14.4
Futibatinib (Cohort B)17.96.1 – 36.9
ORR Based on Investigator Assessment in Cohorts A and B Secondary · At the end of every 2 cycles until disease progression (Up to 31 months)

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 wer

GroupValue95% CI
Futibatinib (Cohort A)9.24.1 – 17.3
Futibatinib (Cohort B)10.72.3 – 28.2
Duration of Response (DOR) Based on IRC in Cohorts A, B and C Secondary · At the end of every 2 cycles until disease progression (Up to 31 months)

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.

GroupValue95% CI
Futibatinib (Cohort A)5.63.5 – NA
Futibatinib (Cohort B)3.92.1 – NA
DOR Based on Investigator Assessment in Cohorts A, B and C Secondary · At the end of every 2 cycles until disease progression (Up to 31 months)

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.

GroupValue95% CI
Futibatinib (Cohort A)5.63.0 – NA
Futibatinib (Cohort B)5.62.0 – NA
Progression- Free Survival (PFS) Based on IRC in Cohorts A, B and C Secondary · At the end of every 2 cycles until disease progression (Up to 31 months)

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.

GroupValue95% CI
Futibatinib (Cohort A)1.91.8 – 3.5
Futibatinib (Cohort B)2.91.8 – 3.7
PFS Based on Investigator Review in Cohorts A, B and C Secondary · At the end of every 2 cycles until disease progression (Up to 31 months)

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.

GroupValue95% CI
Futibatinib (Cohort A)3.31.9 – 3.8
Futibatinib (Cohort B)3.32.1 – 3.7
Overall Survival (OS) in Cohorts A, B and C Secondary · Up to 31 months

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.

GroupValue95% CI
Futibatinib (Cohort A)11.17.3 – 15.6
Futibatinib (Cohort B)5.93.9 – 8.9
Disease Control Rate (DCR) Based on IRC in Cohort A and B Secondary · At the end of every 2 cycles until disease progression (Up to 31 months)

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 pat

GroupValue95% CI
Futibatinib (Cohort A)37.927.7 – 49.0
Futibatinib (Cohort B)50.030.6 – 69.4
DCR Based on Investigator Review in Cohort A and B Secondary · At the end of every 2 cycles until disease progression (Up to 31 months)

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 reductio

GroupValue95% CI
Futibatinib (Cohort A)52.941.9 – 63.7
Futibatinib (Cohort B)64.344.1 – 81.4
Number of Participants With Treatment Emergent Adverse Events (TEAEs) - Cohort A, B and C Secondary · From the first dose of study drug up to 30 days after the last dose (Up to 31 months)

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.

GroupValue95% CI
Futibatinib (Cohort A)87
Futibatinib (Cohort B)27

Adverse events — posted to ClinicalTrials.gov

Time frame: From the first dose of study drug up to 30 days after the last dose (Up to 31 months). Reporting threshold: 0%. Adverse-event reports describe events observed during the trial — not all are caused by the drug.

Futibatinib (Cohort A)
Serious: 23/87 (26%)
Deaths: 54/87
Futibatinib (Cohort B)
Serious: 6/28 (21%)
Deaths: 24/28

Serious adverse events (36 terms)

ReactionSystemFutibatinib (Cohort A)Futibatinib (Cohort B)
DyspnoeaRespiratory, thoracic and mediastinal disorders
General physical health deteriorationGeneral disorders
COVID-19Infections and infestations
PneumoniaInfections and infestations
Decreased appetiteMetabolism and nutrition disorders
HyponatraemiaMetabolism and nutrition disorders
VomitingGastrointestinal disorders
Eyelid ptosisEye disorders
AscitesGastrointestinal disorders
Gastrointestinal haemorrhageGastrointestinal disorders
Small intestinal obstructionGastrointestinal disorders
SubileusGastrointestinal disorders
AstheniaGeneral disorders
FatigueGeneral disorders
Hepatic failureHepatobiliary disorders
Device related infectionInfections and infestations
Escherichia sepsisInfections and infestations
Blood lactic acid increasedInvestigations
General physical condition abnormalInvestigations
DehydrationMetabolism and nutrition disorders
RhabdomyolysisMusculoskeletal and connective tissue disorders
Cerebrovascular accidentNervous system disorders
Ischaemic strokeNervous system disorders
Calculus urinaryRenal and urinary disorders
HaematuriaRenal and urinary disorders
Other adverse events (213 terms — click to expand)

ReactionSystemFutibatinib (Cohort A)Futibatinib (Cohort B)
HyperphosphataemiaMetabolism and nutrition disorders
DiarrhoeaGastrointestinal disorders
ConstipationGastrointestinal disorders
Dry mouthGastrointestinal disorders
Alanine aminotransferase increasedInvestigations
Decreased appetiteMetabolism and nutrition disorders
AnaemiaBlood and lymphatic system disorders
FatigueGeneral disorders
Aspartate aminotransferase increasedInvestigations
Abdominal painGastrointestinal disorders
AstheniaGeneral disorders
StomatitisGastrointestinal disorders
Blood creatinine increasedInvestigations
DysgeusiaNervous system disorders
Dry skinSkin and subcutaneous tissue disorders
NauseaGastrointestinal disorders
VomitingGastrointestinal disorders
MyalgiaMusculoskeletal and connective tissue disorders
ParonychiaInfections and infestations
Urinary tract infectionInfections and infestations
ArthralgiaMusculoskeletal and connective tissue disorders
OnychomadesisSkin and subcutaneous tissue disorders
PyrexiaGeneral disorders
Gamma-glutamyltransferase increasedInvestigations
Back painMusculoskeletal and connective tissue disorders
DyspnoeaRespiratory, thoracic and mediastinal disorders
Palmar-plantar erythrodysaesthesia syndromeSkin and subcutaneous tissue disorders
Gastrooesophageal reflux diseaseGastrointestinal disorders
Blood creatine phosphokinase increasedInvestigations
HeadacheNervous system disorders
CoughRespiratory, thoracic and mediastinal disorders
KeratitisEye disorders
Oedema peripheralGeneral disorders
ConjunctivitisInfections and infestations
Blood alkaline phosphatase increasedInvestigations
Blood phosphorus increasedInvestigations
HypercalcaemiaMetabolism and nutrition disorders
HyponatraemiaMetabolism and nutrition disorders
AlopeciaSkin and subcutaneous tissue disorders
Nail disorderSkin and subcutaneous tissue disorders

Most-reported serious reactions: Dyspnoea, General physical health deterioration, COVID-19, Pneumonia, Decreased appetite, Hyponatraemia, Vomiting, Eyelid ptosis.

Data from ClinicalTrials.gov NCT04189445 adverse events section.

Sponsor's own description

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.

Publications & conference data

8 peer-reviewed publications reference this trial (live from Europe PMC):

  1. Angiogenic signaling pathways and anti-angiogenic therapy for cancer.
    Liu ZL, Chen HH, Zheng LL, Sun LP, et al · · 2023 · cited 808× · PMID 37169756 · DOI 10.1038/s41392-023-01460-1
  2. Tyrosine kinase inhibitors for solid tumors in the past 20 years (2001-2020).
    Huang L, Jiang S, Shi Y. · · 2020 · cited 305× · PMID 33109256 · DOI 10.1186/s13045-020-00977-0
  3. FGFR Fusions in Cancer: From Diagnostic Approaches to Therapeutic Intervention.
    De Luca A, Esposito Abate R, Rachiglio AM, Maiello MR, et al · · 2020 · cited 87× · PMID 32962091 · DOI 10.3390/ijms21186856
  4. FGFR4: A promising therapeutic target for breast cancer and other solid tumors.
    Levine KM, Ding K, Chen L, Oesterreich S. · · 2020 · cited 68× · PMID 32492514 · DOI 10.1016/j.pharmthera.2020.107590
  5. Targeting FGFR for cancer therapy.
    Zhang P, Yue L, Leng Q, Chang C, et al · · 2024 · cited 64× · PMID 38831455 · DOI 10.1186/s13045-024-01558-1
  6. Cancer-associated fibroblasts as therapeutic targets for cancer: advances, challenges, and future prospects.
    Cao Z, Quazi S, Arora S, Osellame LD, et al · · 2025 · cited 57× · PMID 39780187 · DOI 10.1186/s12929-024-01099-2
  7. Fibroblast growth factor receptor fusions in cancer: opportunities and challenges.
    Chen L, Zhang Y, Yin L, Cai B, et al · · 2021 · cited 50× · PMID 34732230 · DOI 10.1186/s13046-021-02156-6
  8. Phase I study of the irreversible fibroblast growth factor receptor 1-4 inhibitor futibatinib in Japanese patients with advanced solid tumors.
    Doi T, Shitara K, Kojima T, Kuboki Y, et al · · 2023 · cited 28× · PMID 35838190 · DOI 10.1111/cas.15486

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