FOENIX-CCA4

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Taiho Oncology Inc.

Participant type

Patients

Age range

18-64 years, 65+ years

Gender

Male and Female

Therapeutic area

Diseases [C] - Neoplasms [C04]

Trial duration

14 Nov 2023 → ongoing

Decision date (initial)

2023-10-25

Transition trial

No

Low-intervention

No

Rare-disease indication

Yes

Vulnerable population

Yes

Funding sources

Taiho Oncology Inc.

External identifiers

EU CT number

2023-503665-39-00

ClinicalTrials.gov

NCT05727176

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Safety, Others, Efficacy, Pharmacokinetic

To assess the efficacy of futibatinib administered at 20 mg and 16 mg QD to verify and describe the clinical benefit

Secondary objectives 3

1. To evaluate further efficacy parameters of futibatinib administered at 20 mg and 16 mg QD
2. To evaluate the safety and tolerability of futibatinib administered at 20 mg and 16 mg QD
3. To evaluate Patient Reported Outcomes (PROs)

Conditions and MedDRA coding

Advanced cholangiocarcinoma

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 6

1. A patient must meet all of the following inclusion criteria to be eligible for participation in this study: 1. Histologically or cytologically confirmed, locally advanced, metastatic, or unresectable intrahepatic of extrahepatic Cholangiocarcinoma.
2. 2. Documented evidence of FGFR2 gene fusions or other FGFR2 rearrangement Received at least one prior systemic gemcitabine and platinum-based regimen for CCA
3. 3. Documentation of radiographic disease progression on the most recent prior therapy Measurable disease performance status 0 or 1 Adequate organ function
4. 4. Documentation of radiographic disease progression on the most recent prior therapy
5. 5. Measurable disease as defined by Response Evaluation Criteria in Solid Tumors (RECIST) guidelines (version 1.1, 2009)
6. 6. Eastern Cooperative Oncology Group (ECOG) performance status 0 or 1

Exclusion criteria 7

1. A patient must not meet any of the following exclusion criteria to be eligible for participation in this study: 1. History or current evidence of calcium and phosphate homeostasis disorder
2. 2. Current evidence of clinically significant retinal disorder
3. 3. Treatment with any of the following within the specified time frame prior to the first dose of futibatinib: a. Major surgery within the previous 4 weeks (the surgical incision should be fully healed prior to the first dose of futibatinib) and radiotherapy for extended field within 4 weeks or limited field radiotherapy within 2 weeks b. Patients with locoregional therapy, eg, transarterial chemoembolization (TACE), selective internal radiotherapy (SIRT) or ablation within 4 weeks c. Any noninvestigational anticancer therapy within 3 weeks or have not recovered from side effects of such therapy prior to futibatinib administration. Endocrine therapy is allowed for patients with breast or prostate cancer d. Targeted therapy or immunotherapy within 3 weeks or within 5 half lives (whichever is shorter) e. Any investigational agent received within 5 half-lives of the drug or 4 weeks, whichever is shorter. g. Patients with prior FGFR-directed therapy
4. 4. A serious illness or medical condition(s) including (but not limited to) the following: a. Known brain metastasis (not including primary brain tumors) unless patient is clinically stable for ≥1 month b. Known acute systemic infection c. Myocardial infarction, severe/unstable angina, symptomatic congestive heart failure (New York Heart Association [NYHA] Class III or IV within the previous 2 months; if >2 months, cardiac function must be within normal limits and the patient must be free of cardiac-related symptoms d. Significant gastrointestinal disorder(s) that could interfere with the absorption of futibatinib e. Other severe acute or chronic medical or psychiatric condition or laboratory abnormality that in the judgment of the Investigator would make the patient inappropriate for entry into this study
5. 5. Known additional malignancy that is progressing or requires active treatment, with the exception of patients with a prior or concurrent malignancy whose natural history or treatment does not have the potential to interfere with the safety or antitumor assessment of the investigational regimen. Exceptions must be discussed with the Sponsor prior to patient enrollment.
6. 6. Pregnant or lactating female
7. 7. Known hypersensitivity or severe reaction to futibatinib or its excipients.

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. ORR by independent central review defined as the proportion of patients experiencing a best overall response ofpartial response (PR) or complete response (CR) (per RECIST 1.1), based on ICR"

Secondary endpoints 6

1. Duration of response (DoR) by BICR
2. Progression-free survival (PFS) by BICR
3. ORR, DoR, and PFS by investigator assessment
4. Overall survival (OS)
5. Safety based on AEs (in particular ≥Grade 3), SAEs, dose modifications, clinical laboratory parameters, ophthalmological exams, and vital signs
6. PROs measured by the European Organization for Research and Treatment of Cancer (EORTC) QLQ-C30 and the EuroQol-5D (EQ-5D)

Investigational products

Investigational medicinal products (IMPs), comparators, placebo, auxiliary

Test 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Taiho Oncology Inc.

Sponsor organisation

Taiho Oncology Inc.

Address

101 Carnegie Center Suite 300

City

Princeton

Postcode

08540-6231

Country

United States

Scientific contact point

Organisation

Taiho Oncology Inc.

Contact name

Hamdy Elsayed

Public contact point

Organisation

Taiho Oncology Inc.

Contact name

Hamdy Elsayed

Third parties 8

Locations

4 EU/EEA countries · 16 investigational sites

By country

Investigational sites

Country notifications

Trial-start, recruitment-start, end and early-termination notifications submitted per Member State

Results and documents

Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial

Documents 40 files

Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.

Application history

15 submissions — initial application plus substantial / non-substantial modifications