NCT02052778

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

Completed Phase 1, PHASE2 Results posted Last updated 27 March 2025

What this trial tests

Phase 1, PHASE2 trial testing Futibatinib in Cholangiocarcinoma in 407 participants. Completed in 22 October 2024.

Timeline

21 July 2014

Primary endpoint
1 October 2020

22 October 2024

Quick facts

Lead sponsor	Taiho Oncology, Inc.
Phase	Phase 1, PHASE2
Status	Completed
Study type	INTERVENTIONAL
Allocation	non randomized
Design	sequential
Masking	none
Primary purpose	treatment
Enrollment	407
Start date	21 July 2014
Primary completion	1 October 2020
Estimated completion	22 October 2024
Sites	58 locations across France, Hong Kong, Italy, Japan, Netherlands, Taiwan, United Kingdom, Germany

Drugs / interventions tested

• Futibatinib (futibatinib) — full drug profile →

Conditions studied

• Cholangiocarcinoma — all drugs for Cholangiocarcinoma →
• Urothelial Cancer — all drugs for Urothelial Cancer →
• Advanced and Metastatic Cancer Patients With Tumors Harboring FGF/FGFR Tumors — all drugs for Advanced and Metastatic Cancer Patients With Tumors Harboring FGF/FGFR Tumors →
• Primary CNS Tumors — all drugs for Primary CNS Tumors →

Sponsor

Taiho Oncology, Inc. — full company profile →

Who can join

18 and older, any sex, with Cholangiocarcinoma or Urothelial 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.

Phase 1: Dose Escalation-Maximum Tolerated Dose (MTD) Primary · Cycle 1 (21-day cycle)

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

Group	Value	95% CI
Phase 1: Dose Escalation: QOD Dosing	NA
Phase 1: Dose Escalation: QD Dosing	20

Phase 1: Dose Escalation-Recommended Phase 2 Dose (RP2D) of TAS-120 Primary · Cycle 1 (21-day cycle)

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 f

Group	Value	95% CI
Phase 1: Dose Escalation: QOD Dosing	NA
Phase 1: Dose Escalation: QD Dosing	20

Phase 1: Dose Expansion: Percentage of Participants With Objective Response Primary · 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

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:

Group	Value	95% CI
Phase 1: Dose Expansion Cohort 1	15.8	7.5 – 27.9
Phase 1: Dose Expansion: Cohort 2	8.8	1.9 – 23.7
Phase 1: Dose Expansion: Cohort 3	13.3	1.7 – 40.5
Phase 1: Dose Expansion: Cohort 4	0	0.0 – 24.7
Phase 1: Dose Expansion: Cohort 5	12.5	2.7 – 32.4
Phase 1: Dose Expansion: Cohort 6	0	0.0 – 12.8
Phase 1: Dose Expansion: Pooled Sub-cohort	33.3	16.5 – 54.0

Phase 2: Percentage of Participants With Objective Response Primary · Up to approximately 37.5 months (through cut-off date 29-May-2021)

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.

Group	Value	95% CI
Phase 2	41.7	32.1 – 51.9

Phase 1: Dose Expansion: Duration of Response (DOR) Secondary · 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

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.

Group	Value	95% CI
Phase 1: Dose Expansion Cohort 1	6.18	2.8 – 10.6
Phase 1: Dose Expansion: Cohort 2	3.52	2.8 – 4.3
Phase 1: Dose Expansion: Cohort 3	2.45	1.4 – 3.4
Phase 1: Dose Expansion: Cohort 5	2.79	2.0 – 4.1
Phase 1: Dose Expansion: Pooled Sub-cohort	8.77	3.5 – 20.8

Phase 2: Duration of Response (DOR) Secondary · Up to approximately 37.5 months (through cut-off date 29-May-2021)

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.

Group	Value	95% CI
Phase 2	8.31	2.1 – 24.8

Phase 1: Dose Expansion: Disease Control Rate (DCR) Secondary · 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

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 in

Group	Value	95% CI
Phase 1: Dose Expansion Cohort 1	66.7	52.9 – 78.6
Phase 1: Dose Expansion: Cohort 2	23.5	10.7 – 41.2
Phase 1: Dose Expansion: Cohort 3	33.3	11.8 – 61.6
Phase 1: Dose Expansion: Cohort 4	38.5	13.9 – 68.4
Phase 1: Dose Expansion: Cohort 5	54.2	32.8 – 74.4
Phase 1: Dose Expansion: Cohort 6	22.2	8.6 – 42.3
Phase 1: Dose Expansion: Pooled Sub-cohort	66.7	46.0 – 83.5

Phase 2: Disease Control Rate (DCR) Secondary · Up to approximately 37.5 months (through cut-off date 29-May-2021)

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 t

Group	Value	95% CI
Phase 2	82.5	73.8 – 89.3

Phase 1: Dose Expansion: Progression-free Survival (PFS) Secondary · up to approximately 27.5 months (through cut-off date 30-Jun-2019)

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 progres

Group	Value	95% CI
Phase 1: Dose Expansion Cohort 1	4.1	2.7 – 6.9
Phase 1: Dose Expansion: Cohort 2	1.9	1.3 – 4.9
Phase 1: Dose Expansion: Cohort 3	1.8	1.3 – 4.7
Phase 1: Dose Expansion: Cohort 4	1.3	0.9 – 4.1
Phase 1: Dose Expansion: Cohort 5	3.5	1.7 – 6.8
Phase 1: Dose Expansion: Cohort 6	2.7	1.4 – 2.7
Phase 1: Dose Expansion: Pooled Sub-cohort	6.9	4.6 – 10.8

Phase 2: Progression-free Survival (PFS) Secondary · Up to approximately 37.5 months (through cut-off date 29-May-2021)

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 reporte

Group	Value	95% CI
Phase 2	8.9	6.7 – 11.0

Phase 1: Dose Expansion: Overall Survival (OS) Secondary · up to approximately 27.5 months (through cut-off date 30-Jun-2019)

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.

Group	Value	95% CI
Phase 1: Dose Expansion Cohort 1	11.4	8.1 – 15.1
Phase 1: Dose Expansion: Cohort 2	11.8	5.5 – NA
Phase 1: Dose Expansion: Cohort 3	8.6	2.1 – NA
Phase 1: Dose Expansion: Cohort 4	5.8	4.6 – NA
Phase 1: Dose Expansion: Cohort 5	10.5	6.7 – NA
Phase 1: Dose Expansion: Cohort 6	7.2	5.6 – NA
Phase 1: Dose Expansion: Pooled Sub-cohort	NA	12.2 – NA

Phase 2: Overall Survival (OS) Secondary · Up to approximately 37.5 months (through cut-off date 29-May-2021)

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.

Group	Value	95% CI
Phase 2	20.0	16.4 – 24.6

Adverse events — posted to ClinicalTrials.gov

Time frame: From the first dose administration up to approximately up to 59.5 months for Phase 1 Dose escalation; up to 50.5 months for Phase 1 Dose expansion Cohorts and up to 37.5 months for Phase 2. Reporting threshold: 5%. Adverse-event reports describe events observed during the trial — not all are caused by the drug.

Phase 1: Dose Escalation: QOD Dosing: 8 mg

Serious: 1/6 (17%)

Deaths: 0/6

Phase 1: Dose Escalation: QOD Dosing: 16 mg

Serious: 0/3 (0%)

Deaths: 0/3

Phase 1: Dose Escalation: QOD Dosing: 24 mg

Serious: 1/3 (33%)

Deaths: 0/3

Phase 1: Dose Escalation: QOD Dosing: 36 mg

Serious: 1/3 (33%)

Deaths: 0/3

Phase 1: Dose Escalation: QOD Dosing: 56 mg

Serious: 1/3 (33%)

Deaths: 0/3

Phase 1: Dose Escalation: QOD Dosing: 80 mg

Serious: 2/5 (40%)

Deaths: 0/5

Phase 1: Dose Escalation: QOD Dosing: 120 mg

Serious: 4/4 (100%)

Deaths: 1/4

Phase 1: Dose Escalation: QOD Dosing: 160 mg

Serious: 7/8 (88%)

Deaths: 0/8

Phase 1: Dose Escalation: QOD Dosing: 200 mg

Serious: 4/7 (57%)

Deaths: 2/7

Phase 1: Dose Escalation: QD Dosing: 4 mg

Serious: 0/4 (0%)

Deaths: 0/4

Phase 1: Dose Escalation: QD Dosing: 8 mg

Serious: 1/5 (20%)

Deaths: 0/5

Phase 1: Dose Escalation: QD Dosing: 16 mg

Serious: 9/14 (64%)

Deaths: 2/14

Phase 1: Dose Escalation: QD Dosing: 20 mg

Serious: 3/7 (43%)

Deaths: 1/7

Phase 1: Dose Escalation: QD Dosing: 24 mg

Serious: 6/14 (43%)

Deaths: 1/14

Phase 1: Dose Expansion Cohort 1

Serious: 27/57 (47%)

Deaths: 40/57

Phase 1: Dose Expansion: Cohort 2

Serious: 13/34 (38%)

Deaths: 22/34

Phase 1: Dose Expansion: Cohort 3

Serious: 10/15 (67%)

Deaths: 12/15

Phase 1: Dose Expansion: Cohort 4

Serious: 9/13 (69%)

Deaths: 11/13

Phase 1: Dose Expansion: Cohort 5

Serious: 13/24 (54%)

Deaths: 15/24

Phase 1: Dose Expansion: Cohort 6

Serious: 14/27 (52%)

Deaths: 17/27

Phase 1: Dose Expansion: Sub-cohort 2

Serious: 0/8 (0%)

Deaths: 1/8

Phase 1: Dose Expansion: Sub-cohort 1

Serious: 0/19 (0%)

Deaths: 9/19

Phase 2

Serious: 42/103 (41%)

Deaths: 58/103

Serious adverse events (157 terms)

Reaction	System	Phase 1: Dose Escalation: …	Phase 1: Dose Escalation: …	Phase 1: Dose Escalation: …	Phase 1: Dose Escalation: …	Phase 1: Dose Escalation: …	Phase 1: Dose Escalation: …	Phase 1: Dose Escalation: …	Phase 1: Dose Escalation: …	Phase 1: Dose Escalation: …	Phase 1: Dose Escalation: …	Phase 1: Dose Escalation: …	Phase 1: Dose Escalation: …	Phase 1: Dose Escalation: …	Phase 1: Dose Escalation: …	Phase 1: Dose Expansion Co…	Phase 1: Dose Expansion: C…	Phase 1: Dose Expansion: C…	Phase 1: Dose Expansion: C…	Phase 1: Dose Expansion: C…	Phase 1: Dose Expansion: C…	Phase 1: Dose Expansion: S…	Phase 1: Dose Expansion: S…	Phase 2
Disease progression	General disorders	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—
Ascites	Gastrointestinal disorders	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—
Pyrexia	General disorders	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—
Abdominal pain	Gastrointestinal disorders	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—
Upper gastrointestinal haemorrhage	Gastrointestinal disorders	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—
Bile duct obstruction	Hepatobiliary disorders	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—
Sepsis	Infections and infestations	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—
Colitis	Gastrointestinal disorders	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—
Intestinal obstruction	Gastrointestinal disorders	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—
Hepatic failure	Hepatobiliary disorders	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—
Biliary tract infection	Infections and infestations	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—
Urinary tract infection	Infections and infestations	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—
Fall	Injury, poisoning and procedural complications	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—
Overdose	Injury, poisoning and procedural complications	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—
Blood bilirubin increased	Investigations	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—
Decreased appetite	Metabolism and nutrition disorders	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—
Dehydration	Metabolism and nutrition disorders	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—
Tumour pain	Neoplasms benign, malignant and unspecified (incl cysts and polyps)	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—
Headache	Nervous system disorders	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—
Hemiparesis	Nervous system disorders	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—
Migraine	Nervous system disorders	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—
Transient ischaemic attack	Nervous system disorders	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—
Acute kidney injury	Renal and urinary disorders	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—
Dyspnoea	Respiratory, thoracic and mediastinal disorders	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—
Anaemia	Blood and lymphatic system disorders	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—

Other adverse events (336 terms — click to expand)

Reaction	System	Phase 1: Dose Escalation: …	Phase 1: Dose Escalation: …	Phase 1: Dose Escalation: …	Phase 1: Dose Escalation: …	Phase 1: Dose Escalation: …	Phase 1: Dose Escalation: …	Phase 1: Dose Escalation: …	Phase 1: Dose Escalation: …	Phase 1: Dose Escalation: …	Phase 1: Dose Escalation: …	Phase 1: Dose Escalation: …	Phase 1: Dose Escalation: …	Phase 1: Dose Escalation: …	Phase 1: Dose Escalation: …	Phase 1: Dose Expansion Co…	Phase 1: Dose Expansion: C…	Phase 1: Dose Expansion: C…	Phase 1: Dose Expansion: C…	Phase 1: Dose Expansion: C…	Phase 1: Dose Expansion: C…	Phase 1: Dose Expansion: S…	Phase 1: Dose Expansion: S…	Phase 2
Hyperphosphataemia	Metabolism and nutrition disorders	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—
Constipation	Gastrointestinal disorders	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—
Diarrhoea	Gastrointestinal disorders	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—
Dry mouth	Gastrointestinal disorders	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—
Fatigue	General disorders	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—
Alopecia	Skin and subcutaneous tissue disorders	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—
Dry skin	Skin and subcutaneous tissue disorders	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—
Aspartate aminotransferase increased	Investigations	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—
Nausea	Gastrointestinal disorders	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—
Stomatitis	Gastrointestinal disorders	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—
Dry eye	Eye disorders	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—
Decreased appetite	Metabolism and nutrition disorders	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—
Arthralgia	Musculoskeletal and connective tissue disorders	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—
Palmar-plantar erythrodysaesthesia syndrome	Skin and subcutaneous tissue disorders	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—
Abdominal pain	Gastrointestinal disorders	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—
Dysgeusia	Nervous system disorders	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—
Vomiting	Gastrointestinal disorders	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—
Urinary tract infection	Infections and infestations	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—
Weight decreased	Investigations	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—
Alanine aminotransferase increased	Investigations	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—
Anaemia	Blood and lymphatic system disorders	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—
Hyponatraemia	Metabolism and nutrition disorders	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—
Back pain	Musculoskeletal and connective tissue disorders	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—
Hypercalcaemia	Metabolism and nutrition disorders	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—
Nail disorder	Skin and subcutaneous tissue disorders	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—
Onycholysis	Skin and subcutaneous tissue disorders	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—
Oedema peripheral	General disorders	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—
Blood creatinine increased	Investigations	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—
Muscle spasms	Musculoskeletal and connective tissue disorders	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—
Myalgia	Musculoskeletal and connective tissue disorders	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—
Onychomadesis	Skin and subcutaneous tissue disorders	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—
Hypophosphataemia	Metabolism and nutrition disorders	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—
Nail discolouration	Skin and subcutaneous tissue disorders	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—
Blood alkaline phosphatase increased	Investigations	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—
Thrombocytopenia	Blood and lymphatic system disorders	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—
Pyrexia	General disorders	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—
Blood creatine phosphokinase increased	Investigations	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—
Dizziness	Nervous system disorders	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—
Peripheral sensory neuropathy	Nervous system disorders	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—
Oropharyngeal pain	Respiratory, thoracic and mediastinal disorders	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—	—

Most-reported serious reactions: Disease progression, Ascites, Pyrexia, Abdominal pain, Upper gastrointestinal haemorrhage, Bile duct obstruction, Sepsis, Colitis.

Data from ClinicalTrials.gov NCT02052778 adverse events section.

Sponsor's own description

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).

Publications & conference data

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

1. Cholangiocarcinoma - evolving concepts and therapeutic strategies.
   Ilyas SI, Khan SA, Hallemeier CL, Kelley RK, et al · · 2018 · cited 1192× · PMID 28994423 · DOI 10.1038/nrclinonc.2017.157
2. New Horizons for Precision Medicine in Biliary Tract Cancers.
   Valle JW, Lamarca A, Goyal L, Barriuso J, et al · · 2017 · cited 475× · PMID 28818953 · DOI 10.1158/2159-8290.cd-17-0245
3. Polyclonal Secondary <i>FGFR2</i> Mutations Drive Acquired Resistance to FGFR Inhibition in Patients with FGFR2 Fusion-Positive Cholangiocarcinoma.
   Goyal L, Saha SK, Liu LY, Siravegna G, et al · · 2017 · cited 439× · PMID 28034880 · DOI 10.1158/2159-8290.cd-16-1000
4. Futibatinib for <i>FGFR2</i>-Rearranged Intrahepatic Cholangiocarcinoma.
   Goyal L, Meric-Bernstam F, Hollebecque A, Valle JW, et al · · 2023 · cited 391× · PMID 36652354 · DOI 10.1056/nejmoa2206834
5. Futibatinib, an Irreversible FGFR1-4 Inhibitor, in Patients with Advanced Solid Tumors Harboring <i>FGF</i>/<i>FGFR</i> Aberrations: A Phase I Dose-Expansion Study.
   Meric-Bernstam F, Bahleda R, Hierro C, Sanson M, et al · · 2022 · cited 208× · PMID 34551969 · DOI 10.1158/2159-8290.cd-21-0697
6. Fibroblast Growth Factor Receptors (FGFRs): Structures and Small Molecule Inhibitors.
   Dai S, Zhou Z, Chen Z, Xu G, et al · · 2019 · cited 206× · PMID 31216761 · DOI 10.3390/cells8060614
7. Molecular and clinical significance of fibroblast growth factor 2 (FGF2 /bFGF) in malignancies of solid and hematological cancers for personalized therapies.
   Akl MR, Nagpal P, Ayoub NM, Tai B, et al · · 2016 · cited 148× · PMID 27007053 · DOI 10.18632/oncotarget.8203
8. Emerging molecular therapeutic targets for cholangiocarcinoma.
   Ilyas SI, Gores GJ. · · 2017 · cited 140× · PMID 28389139 · DOI 10.1016/j.jhep.2017.03.026