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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT02052778




Registration number
NCT02052778
Ethics application status
Date submitted
23/01/2014
Date registered
3/02/2014

Titles & IDs
Public title
A Study of TAS-120 in Patients With Advanced Solid Tumors
Scientific title
Phase 1/2 Study of TAS-120 in Patients With Advanced Solid Tumors Harboring FGF/FGFR Aberrations
Secondary ID [1] 0 0
2013-004810-16
Secondary ID [2] 0 0
TPU-TAS-120-101
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Cholangiocarcinoma 0 0
Urothelial Cancer 0 0
Advanced and Metastatic Cancer Patients With Tumors Harboring FGF/FGFR Tumors 0 0
Primary CNS Tumors 0 0
Breast Cancer 0 0
Gastric Cancer 0 0
Condition category
Condition code
Cancer 0 0 0 0
Biliary tree (gall bladder and bile duct)
Oral and Gastrointestinal 0 0 0 0
Other diseases of the mouth, teeth, oesophagus, digestive system including liver and colon

Intervention/exposure
Study type
Interventional(has expanded access)
Description of intervention(s) / exposure
Treatment: Drugs - Futibatinib

Experimental: Phase 1: Dose Escalation: QOD Dosing: 8 mg - Participants 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.

Experimental: Phase 1: Dose Escalation: QOD Dosing: 16 mg - Participants 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.

Experimental: Phase 1: Dose Escalation: QOD Dosing: 24 mg - Participants 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.

Experimental: Phase 1: Dose Escalation: QOD Dosing: 36 mg - Participants 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.

Experimental: Phase 1: Dose Escalation: QOD Dosing: 56 mg - Participants 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.

Experimental: Phase 1: Dose Escalation: QOD Dosing: 80 mg - Participants 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.

Experimental: Phase 1: Dose Escalation: QOD Dosing: 120 mg - Participants 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.

Experimental: Phase 1: Dose Escalation: QOD Dosing: 160 mg - Participants 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.

Experimental: Phase 1: Dose Escalation: QOD Dosing: 200 mg - Participants 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.

Experimental: Phase 1: Dose Escalation: QD Dosing: 4 mg - Participants 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.

Experimental: Phase 1: Dose Escalation: QD Dosing: 8 mg - Participants 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.

Experimental: Phase 1: Dose Escalation: QD Dosing: 16 mg - Participants 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.

Experimental: Phase 1: Dose Escalation: QD Dosing: 20 mg - Participants 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.

Experimental: Phase 1: Dose Escalation: QD Dosing: 24 mg - Participants 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.

Experimental: Phase 1: Dose Expansion Cohort 1 - Participants 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.

Experimental: Phase 1: Dose Expansion: Cohort 2 - Participants 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.

Experimental: Phase 1: Dose Expansion: Cohort 3 - Participants 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.

Experimental: Phase 1: Dose Expansion: Cohort 4 - Participants 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.

Experimental: Phase 1:Dose Expansion: Cohort 5 - Participants 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.

Experimental: Phase 1: Dose Expansion: Cohort 6 - Participants 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.

Experimental: Phase 1: Dose Expansion: Sub-cohort 1 - Participants 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.

Experimental: Phase 1: Dose Expansion: Sub-cohort 2 - Participants 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.

Experimental: Phase 2 - Participants 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.


Treatment: Drugs: Futibatinib
oral once daily dosing, 21-day cycle
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Phase 1: Dose Escalation-Maximum Tolerated Dose (MTD)
Timepoint [1] 0 0
Cycle 1 (21-day cycle)
Primary outcome [2] 0 0
Phase 1: Dose Escalation-Recommended Phase 2 Dose (RP2D) of TAS-120
Timepoint [2] 0 0
Cycle 1 (21-day cycle)
Primary outcome [3] 0 0
Phase 1: Dose Expansion: Percentage of Participants With Objective Response
Timepoint [3] 0 0
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
Primary outcome [4] 0 0
Phase 2: Percentage of Participants With Objective Response
Timepoint [4] 0 0
Up to approximately 37.5 months (through cut-off date 29-May-2021)
Secondary outcome [1] 0 0
Phase 1: Dose Expansion: Duration of Response (DOR)
Timepoint [1] 0 0
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
Secondary outcome [2] 0 0
Phase 2: Duration of Response (DOR)
Timepoint [2] 0 0
Up to approximately 37.5 months (through cut-off date 29-May-2021)
Secondary outcome [3] 0 0
Phase 1: Dose Expansion: Disease Control Rate (DCR)
Timepoint [3] 0 0
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
Secondary outcome [4] 0 0
Phase 2: Disease Control Rate (DCR)
Timepoint [4] 0 0
Up to approximately 37.5 months (through cut-off date 29-May-2021)
Secondary outcome [5] 0 0
Phase 1: Dose Expansion: Progression-free Survival (PFS)
Timepoint [5] 0 0
up to approximately 27.5 months (through cut-off date 30-Jun-2019)
Secondary outcome [6] 0 0
Phase 2: Progression-free Survival (PFS)
Timepoint [6] 0 0
Up to approximately 37.5 months (through cut-off date 29-May-2021)
Secondary outcome [7] 0 0
Phase 1: Dose Expansion: Overall Survival (OS)
Timepoint [7] 0 0
up to approximately 27.5 months (through cut-off date 30-Jun-2019)
Secondary outcome [8] 0 0
Phase 2: Overall Survival (OS)
Timepoint [8] 0 0
Up to approximately 37.5 months (through cut-off date 29-May-2021)
Secondary outcome [9] 0 0
Phase 2: European Quality of Life-5 Dimensions-3 Level (EQ-5D-3L) Questionnaire: Mobility Scores at Specified Visits
Timepoint [9] 0 0
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])
Secondary outcome [10] 0 0
Phase 2: European Quality of Life-5 Dimensions-3 Level (EQ-5D-3L) Questionnaire: Self-care Scores at Specified Visits
Timepoint [10] 0 0
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])
Secondary outcome [11] 0 0
Phase 2: European Quality of Life-5 Dimensions-3 Level (EQ-5D-3L) Questionnaire: Usual Activities Scores at Specified Visits
Timepoint [11] 0 0
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])
Secondary outcome [12] 0 0
Phase 2: European Quality of Life-5 Dimensions-3 Level (EQ-5D-3L) Questionnaire: Pain/Discomfort Scores at Specified Visits
Timepoint [12] 0 0
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])
Secondary outcome [13] 0 0
Phase 2: European Quality of Life-5 Dimensions-3 Level (EQ-5D-3L) Questionnaire: Anxiety/Depression Scores at Specified Visits
Timepoint [13] 0 0
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])
Secondary outcome [14] 0 0
Phase 2: Change From Baseline in EQ-5D-3L Visual Analogue Scale (VAS) at Specified Visits
Timepoint [14] 0 0
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])
Secondary outcome [15] 0 0
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
Timepoint [15] 0 0
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])
Secondary outcome [16] 0 0
Phase 1: Dose Expansion: Number of Participants With Any Adverse Events (AEs) and Any Serious AEs (SAEs)
Timepoint [16] 0 0
From the first dose up to approximately 50.5 months (through cut-off date 29-May-2021)
Secondary outcome [17] 0 0
Phase 2: Number of Participants With Any Adverse Events (AEs) and Any Serious Adverse Events (SAEs)
Timepoint [17] 0 0
From the first dose up to approximately 37.5 months (through cut-off date 29-May-2021)

Eligibility
Key inclusion criteria
1. Provide written informed consent
2. Age = 18 years of age
3. Has histologically or cytologically confirmed, locally advanced or metastatic cancer
4. The following specific criteria for each study portion

Phase 1 (Dose Escalation):
* Patients with any type of solid tumor
* 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)
5. Eastern Cooperative Oncology Group (ECOG) performance status 0 or 1
6. Adequate organ function.
Minimum age
18 Years
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
1. History and/or current evidence of clinically significant non-tumor related alteration of calcium-phosphorus homeostasis.
2. History and/or current evidence of clinically significant ectopic mineralization/calcification.
3. History and/or current evidence of clinically significant retinal disorder
4. A serious illness or medical condition(s)
5. Pregnant or breast-feeding female

Study design
Purpose of the study
Treatment
Allocation to intervention
Non-randomised trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Other
Other design features
Phase
Phase 1
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Completed
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
21/07/2014
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
29/05/2021
Sample size
Target
Accrual to date
Final
407
Recruitment in Australia
Recruitment state(s)
Recruitment hospital [1] 0 0
Royal Melbourne Hospital - Melbourne
Recruitment postcode(s) [1] 0 0
- Melbourne
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
Arizona
Country [2] 0 0
United States of America
State/province [2] 0 0
California
Country [3] 0 0
United States of America
State/province [3] 0 0
Georgia
Country [4] 0 0
United States of America
State/province [4] 0 0
Illinois
Country [5] 0 0
United States of America
State/province [5] 0 0
Kansas
Country [6] 0 0
United States of America
State/province [6] 0 0
Massachusetts
Country [7] 0 0
United States of America
State/province [7] 0 0
Michigan
Country [8] 0 0
United States of America
State/province [8] 0 0
Minnesota
Country [9] 0 0
United States of America
State/province [9] 0 0
New Mexico
Country [10] 0 0
United States of America
State/province [10] 0 0
New York
Country [11] 0 0
United States of America
State/province [11] 0 0
Pennsylvania
Country [12] 0 0
United States of America
State/province [12] 0 0
South Carolina
Country [13] 0 0
United States of America
State/province [13] 0 0
Texas
Country [14] 0 0
United States of America
State/province [14] 0 0
Utah
Country [15] 0 0
United States of America
State/province [15] 0 0
Virginia
Country [16] 0 0
United States of America
State/province [16] 0 0
Washington
Country [17] 0 0
United States of America
State/province [17] 0 0
Wisconsin
Country [18] 0 0
Canada
State/province [18] 0 0
Toronto
Country [19] 0 0
France
State/province [19] 0 0
Cedex
Country [20] 0 0
France
State/province [20] 0 0
Bordeaux
Country [21] 0 0
France
State/province [21] 0 0
Bron
Country [22] 0 0
France
State/province [22] 0 0
Paris
Country [23] 0 0
France
State/province [23] 0 0
Rennes cedex
Country [24] 0 0
France
State/province [24] 0 0
Villejuif
Country [25] 0 0
Germany
State/province [25] 0 0
Essen
Country [26] 0 0
Hong Kong
State/province [26] 0 0
Sha Tin
Country [27] 0 0
Italy
State/province [27] 0 0
Milano
Country [28] 0 0
Italy
State/province [28] 0 0
Padova
Country [29] 0 0
Japan
State/province [29] 0 0
Hokkaido
Country [30] 0 0
Japan
State/province [30] 0 0
Kyoto
Country [31] 0 0
Japan
State/province [31] 0 0
Miyagi
Country [32] 0 0
Japan
State/province [32] 0 0
Osaka
Country [33] 0 0
Japan
State/province [33] 0 0
Tokyo
Country [34] 0 0
Korea, Republic of
State/province [34] 0 0
Seoul
Country [35] 0 0
Netherlands
State/province [35] 0 0
Jenna
Country [36] 0 0
Spain
State/province [36] 0 0
Barcelona
Country [37] 0 0
Spain
State/province [37] 0 0
Madrid
Country [38] 0 0
Taiwan
State/province [38] 0 0
Tainan
Country [39] 0 0
Taiwan
State/province [39] 0 0
Taipei
Country [40] 0 0
United Kingdom
State/province [40] 0 0
London
Country [41] 0 0
United Kingdom
State/province [41] 0 0
Manchester

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Taiho Oncology, Inc.
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries

Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
No
No/undecided IPD sharing reason/comment


What supporting documents are/will be available?




Results publications and other study-related documents

No documents have been uploaded by study researchers.


Results not provided in https://clinicaltrials.gov/study/NCT02052778