What side effects are associated with this type of intervention?

Common treatments for cholangiocarcinoma, such as gemcitabine-cisplatin, are associated with side effects including neutropenia, thrombocytopenia, anemia, fatigue, nausea, vomiting, and increased risk of infection. Futibatinib, a selective FGFR inhibitor, may cause hyperphosphatemia, dry mouth, fatigue, and gastrointestinal disturbances. Both treatment types can lead to significant toxicities that require careful management and monitoring.

What prior FDA approvals exist?

Futibatinib is a selective FGFR inhibitor being studied for the treatment of cholangiocarcinoma with FGFR2 gene rearrangements. Prior FDA approvals for cholangiocarcinoma include pemigatinib (Pemazyre), another selective FGFR inhibitor, approved for patients with previously treated, unresectable locally advanced or metastatic cholangiocarcinoma with an FGFR2 fusion or rearrangement. Additionally, ivosidenib (Tibsovo) has been approved for patients with IDH1-mutant cholangiocarcinoma. These targeted therapies represent significant advancements in the personalized treatment of cholangiocarcinoma.

What other types of research exist?

Promising alternatives to Futibatinib for cholangiocarcinoma patients include the combination of anlotinib (an antiangiogenic tyrosine kinase inhibitor) and TQB2450 (a PD-L1 inhibitor). This combination has shown an objective response rate of 21.21%, a disease control rate of 72.73%, and a median progression-free survival of 6.24 months in pretreated advanced biliary tract cancers. Additionally, patients with high tumor mutational burden had better clinical benefit rates and longer overall survival.

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Alkylating agents/Anti-metabolites

Futibatinib for Cholangiocarcinoma (FOENIX-CCA3 Trial)

Phase 3

Waitlist Available

Research Sponsored by Taiho Oncology, Inc.

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Patients who have received treatment for locally advanced disease (for example, trans-arterial chemoembolization, selective internal radiation therapy, external beam radiation) must have evidence of radiographic progression with measurable disease outside the previously-treated lesions

Is ≥18 years of age (or meets the country's regulatory definition for legal adult age)

Must not have

Major surgery within the previous 4 weeks (the surgical incision should be fully healed prior to the first dose of study therapy)

Congenital long QT syndrome, or any known history of torsade de pointes, or family history of unexplained sudden death

Timeline

Screening 3 weeks

Treatment Varies

Follow Up up to12 months

Awards & highlights

No Placebo-Only Group

Pivotal Trial

Summary

This trial is testing futibatinib, an oral medication, to treat patients with advanced liver cancer that has a specific genetic change. The drug works by blocking a protein that helps cancer cells grow. Futibatinib has shown promise in early studies.

Who is the study for?

This trial is for adults with advanced, metastatic, or recurrent inoperable bile duct cancer (cholangiocarcinoma) that has a specific genetic change (FGFR2 gene rearrangements). Participants must have measurable disease, adequate organ function, and be able to take oral medication. They should not have had previous systemic cancer treatment or certain heart conditions. Women of childbearing potential must test negative for pregnancy and agree to use birth control.

What is being tested?

The study compares the effectiveness and safety of futibatinib versus gemcitabine-cisplatin chemotherapy as first-line treatments. It's an open-label Phase 3 trial where patients are randomly assigned to one of two groups: one receiving futibatinib and the other receiving standard chemotherapy.

What are the potential side effects?

Potential side effects may include issues related to liver function abnormalities due to underlying liver metastasis, retinal disorders if present before joining the study, uncontrolled ventricular arrhythmias or QT interval prolongation on ECGs indicating heart rhythm problems.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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My cancer has grown or spread after local treatment like radiation.

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I am 18 years old or older.

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My cancer is advanced or has returned, can't be surgically removed, and has a specific genetic change.

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I am fully active or can carry out light work.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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I have not had major surgery in the last 4 weeks or my surgical wound has fully healed.

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I have a family history of sudden unexplained death or specific heart rhythm abnormalities.

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I recently had radiotherapy and am still feeling its effects.

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I currently have a serious infection.

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I have another cancer that is serious and might spread or needs treatment now.

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I have had treatment for cancer that affects my whole body.

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I have a type of liver cancer that is mixed hepatocellular and intrahepatic cholangiocarcinoma.

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I have or had heart rhythm problems that are not under control.

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I have had a liver transplant.

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I have not had a heart attack or severe heart issues in the last 6 months.

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I am currently pregnant or breastfeeding.

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I cannot take medicine by mouth.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ up to12 months

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~up to12 months

This trial's timeline: 3 weeks for screening, Varies for treatment, and up to12 months for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

PFS: defined as the time from date of randomization to the date of documentation of disease progression by ICR per RECIST (version 1.1, 2009) or date of death, whichever comes first.

Secondary study objectives

DCR

ORR

+2 more

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Pivotal Trial

The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.

Trial Design

2Treatment groups

Experimental Treatment

Active Control

Group I: TAS-120Experimental Treatment1 Intervention

TAS-120 tablets, oral; 21-day cycle

Group II: Cisplatin/GemcitabineActive Control1 Intervention

• On Days 1 and 8 of a 21-day cycle, patients will receive: * Cisplatin 25 mg/m2 in 1000 mL 0.9% saline by intravenous (I.V.) infusion over 1 hour, followed by 500 mL 0.9% saline over 30 minutes; and * Gemcitabine 1000 mg/m2 in 250-500 mL 0.9% saline by I.V. infusion over 30 minutes, beginning after completion of the cisplatin and saline infusions.

0 / 16Eligibility criteria met

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

Cholangiocarcinoma treatments often target specific genetic mutations to inhibit cancer growth. Futibatinib, a selective FGFR inhibitor, works by blocking the fibroblast growth factor receptor (FGFR) pathways, which are involved in cell growth and survival. This is particularly important for patients with FGFR2 gene rearrangements, as it can directly target the underlying genetic driver of their cancer. Other treatments, such as dabrafenib and trametinib, target BRAF mutations by inhibiting the MAPK/ERK signaling pathway, which is crucial for cell division and proliferation. These targeted therapies offer a more personalized approach, potentially leading to better outcomes and fewer side effects compared to traditional chemotherapy.

Role of adjuvant and neoadjuvant therapy for resectable biliary tract cancer.Dramatic response to dabrafenib and trametinib combination in a BRAF V600E-mutated cholangiocarcinoma: implementation of a molecular tumour board and next-generation sequencing for personalized medicine.Biliary tract cancers: understudied and poorly understood.