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Trial details imported from ClinicalTrials.gov

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




Registration number
NCT02052778
Ethics application status
Date submitted
23/01/2014
Date registered
3/02/2014
Date last updated
31/03/2020

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
Brain Tumor 0 0
Urothelial Cancer 0 0
Other Tumor Types With FGFR2 Gene Fusions 0 0
Activating Mutations 0 0
FGFR2 Amplification 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
Description of intervention(s) / exposure
Treatment: Drugs - TAS-120

Experimental: TAS-120 - TAS-120 tablets, oral; 21-day cycle
Dose escalation portion of the study was completed.
Dose expansion- patients with tumors harboring specific FGFR aberrations, specifically in CCA, Brain Tumor , Urotherial carcinoma and any other tumors with FGFR fusion, activating mutation and amplification.
Phase 2- intra-hepatic CCA patients with tumors harboring FGFR2 gene fusions


Treatment: Drugs: TAS-120


Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Phase 1 - Overall Response Rate (ORR) - Response assessments will be made based on RECIST guidelines (version 1.1, 2009) for solid tumors
Timepoint [1] 0 0
12 months
Primary outcome [2] 0 0
Phase 1 - Early Progression Rate (EPR) for GBM or grade III glioma - Response assessments will be made based RANO for brain tumors
Timepoint [2] 0 0
12 months
Primary outcome [3] 0 0
Phase 2 - Overall Response Rate (ORR) - Response assessments will be made based on RECIST guidelines (version 1.1, 2009) for solid tumors
Timepoint [3] 0 0
12 months
Secondary outcome [1] 0 0
Duration of Response (DOR) - DOR is defined as time from first documentation of response to the first documentation of objective tumor progression or death due to any cause, whichever occurs first
Timepoint [1] 0 0
12 months
Secondary outcome [2] 0 0
Disease Control Rate (DCR) - DCR is defined as proportion of patients with objective evidence of complete response, partial response or stable disease
Timepoint [2] 0 0
12 months
Secondary outcome [3] 0 0
Progression free survival (PFS) - PFS is defined as time from the day of first dose to the date of first objectively documented disease progression or death
Timepoint [3] 0 0
12 months
Secondary outcome [4] 0 0
Patient Reported Outcome (PRO) - PCR is define as the analysis of EQ-5D and EORTC QLQ-C30 from baseline through end of treatment
Timepoint [4] 0 0
12 months
Secondary outcome [5] 0 0
Overall Survival (OS) - OS is defined as date of first dose to death date in the safety population of Phase 1 and safety and efficacy population for phase 2
Timepoint [5] 0 0
12 months

Eligibility
Key inclusion criteria
Has histologically or cytologically confirmed, locally advanced, metastatic cancer meeting
the following criteria:

Phase 1 Expansion

1. Patient has failed all standard therapies or standard therapy does not exist or is not
tolerated.

2. Patient has specific FGF/FGFR aberrations

- Intrahepatic or extrahepatic cholangiocarcinoma with FGFR2 gene fusions or other
FGFR2 abnormalities, i.e., gene mutations (see Appendix A), rearrangements or
amplifications

- Glioblastoma or grade III glioma (i.e., anaplastic astrocytoma or anaplastic
oligodendroglioma) with FGFR gene fusions or activating mutations.

- Advanced urothelial carcinoma with FGFR3 fusions or FGFR3 activating mutations

- All other tumor types harboring FGF9, FGF19 or FGFR2 amplifications (= 10
copies), FGFR gene fusions, or FGFR activating mutations

Phase 2

1. Patient has histologically or cytologically confirmed, locally advanced, metastatic,
unresectable iCCA harboring FGFR2 gene fusions based on results from a NGS assay by
the Sponsor's designated central laboratory

2. Patient has been treated with and failed at least one prior systemic gemcitabine and
platinum-based chemotherapy for the advanced disease

3. Must have documentation of radiographic progression of disease on prior systemic
therapy

4. Patient has measurable disease as defined by Response Evaluation Criteria in Solid
Tumors (RECIST) guidelines (version 1.1, 2009) for advanced solid tumors or RANO
criteria (2010) for brain tumors.

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

6. Adequate organ function
Minimum age
18 Years
Maximum age
No limit
Gender
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
A patient will be excluded from this study if any of the following criteria are met:

1. History and/or current evidence of 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 confirmed
by retinal examination.

4. A serious illness or medical condition(s)

Study design
Purpose of the study
Treatment
Allocation to intervention
N/A
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
Single group
Other design features
Phase
Phase 1/Phase 2
Type of endpoint(s)
Statistical methods / analysis

Recruitment
Recruitment status
Active, not recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
Sample size
Target
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
Recruitment hospital [1] 0 0
Royal Melbourne Hospital - Melbourne
Recruitment hospital [2] 0 0
Scientia Clinical Research University of New South Wales - Randwick
Recruitment postcode(s) [1] 0 0
- Melbourne
Recruitment postcode(s) [2] 0 0
2031 - Randwick
Recruitment postcode(s) [3] 0 0
NSW 2031 - Randwick
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
Florida
Country [4] 0 0
United States of America
State/province [4] 0 0
Georgia
Country [5] 0 0
United States of America
State/province [5] 0 0
Illinois
Country [6] 0 0
United States of America
State/province [6] 0 0
Kansas
Country [7] 0 0
United States of America
State/province [7] 0 0
Massachusetts
Country [8] 0 0
United States of America
State/province [8] 0 0
Michigan
Country [9] 0 0
United States of America
State/province [9] 0 0
Minnesota
Country [10] 0 0
United States of America
State/province [10] 0 0
New Mexico
Country [11] 0 0
United States of America
State/province [11] 0 0
New York
Country [12] 0 0
United States of America
State/province [12] 0 0
Pennsylvania
Country [13] 0 0
United States of America
State/province [13] 0 0
South Carolina
Country [14] 0 0
United States of America
State/province [14] 0 0
Texas
Country [15] 0 0
United States of America
State/province [15] 0 0
Washington
Country [16] 0 0
United States of America
State/province [16] 0 0
Wisconsin
Country [17] 0 0
France
State/province [17] 0 0
Bordeaux
Country [18] 0 0
France
State/province [18] 0 0
Bron
Country [19] 0 0
France
State/province [19] 0 0
Lyon
Country [20] 0 0
France
State/province [20] 0 0
Paris
Country [21] 0 0
France
State/province [21] 0 0
Rennes cedex
Country [22] 0 0
Hong Kong
State/province [22] 0 0
Sha Tin
Country [23] 0 0
Hong Kong
State/province [23] 0 0
Shatin
Country [24] 0 0
Japan
State/province [24] 0 0
Nagoya
Country [25] 0 0
Japan
State/province [25] 0 0
Osaka
Country [26] 0 0
Korea, Republic of
State/province [26] 0 0
Seoul
Country [27] 0 0
Netherlands
State/province [27] 0 0
Amsterdam
Country [28] 0 0
Spain
State/province [28] 0 0
Barcelona
Country [29] 0 0
Spain
State/province [29] 0 0
Madrid
Country [30] 0 0
Taiwan
State/province [30] 0 0
Taipei
Country [31] 0 0
United Kingdom
State/province [31] 0 0
London

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

Ethics approval
Ethics application status

Summary
Brief summary
This is an open-label, nonrandomized, Phase 1 dose-escalation, dose-expansion, and Phase 2
study targeting tumors with FGF/FGFR aberrations. The purpose of the study is to evaluate the
safety, tolerability, PK, pharmacodynamic, and anti-tumor activity of TAS-120 in patients
with advanced solid tumors with and without FGF/FGFR-related abnormalities.

The study will be conducted in 3 parts, (1) Dose escalation to determine the MTD and/ or RP2D
of TAS-120 in which this part of the study has been completed; (2) Phase 1 expansion to
further evaluate the safety and efficacy of RP2D of TAS-120 in patients with tumors harboring
specific FGFR aberrations, specifically in patients with cholangiocarcinoma, gliomas ,
urothelial carcinomas and any other tumors with FGFR fusion or activating mutation or
amplification. Up to approximately 185 patients will be enrolled in the phase 1 expansion;
and (3) Phase 2 study to confirm ORR of TAS-120 in intra-hepatic CCA patients with tumors
harboring FGFR2 gene fusions. Approx. 100 patients will be enrolled in phase 2.
Trial website
https://clinicaltrials.gov/show/NCT02052778
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Karim Benhadji, MD
Address 0 0
Taiho Oncology, Inc.
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

Summary results
Other publications