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Futibatinib treatment for advanced cholangiocarcinoma with FGFR2 gene changes

Phase 2 Study of Futibatinib 20 mg and 16 mg in Patients With Advanced Cholangiocarcinoma With FGFR2 Fusions or Rearrangements

Phase 2 Interventional Taiho Oncology, Inc. · NCT05727176

This study is testing if two different doses of a new drug called futibatinib can help people with advanced bile duct cancer that has specific gene changes feel better after other treatments.

Quick facts

PhasePhase 2
Study typeInterventional
Enrollment120 (estimated)
Ages18 Years and up
SexAll
SponsorTaiho Oncology, Inc. Industry-sponsored
Drugs / interventionsfutibatinib, immunotherapy
Locations65 sites (La Jolla, California and 64 other locations)
Trial IDNCT05727176 on ClinicalTrials.gov

What this trial studies

This open-label, multinational Phase 2 study evaluates the safety and efficacy of two different doses of futibatinib in patients with advanced cholangiocarcinoma that have FGFR2 gene fusions or rearrangements. Participants will be randomized to receive either 16 mg or 20 mg of futibatinib daily for 21-day cycles. The study aims to confirm the clinical benefit of the drug in patients who have previously been treated for their condition. Patients will continue treatment until disease progression or other withdrawal criteria are met.

Who should consider this trial

Good fit: Ideal candidates include patients with advanced, unresectable cholangiocarcinoma who have FGFR2 gene fusions and have previously received systemic therapy.

Not a fit: Patients with significant retinal disorders or those who have not progressed after prior therapies may not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment could provide a new therapeutic option for patients with advanced cholangiocarcinoma that have specific genetic alterations.

How similar studies have performed: Other studies targeting FGFR2 alterations in cholangiocarcinoma have shown promising results, indicating potential for success in this approach.

Eligibility criteria

Show full inclusion / exclusion criteria 
Inclusion Criteria:

1. Histologically or cytologically confirmed, locally advanced, metastatic, or unresectable intrahepatic of extrahepatic Cholangiocarcinoma.
2. Documented evidence of FGFR2 gene fusions or other FGFR2 rearrangement
3. Received at least one prior systemic gemcitabine and platinum-based regimen for CCA
4. Documentation of radiographic disease progression on the most recent prior therapy
5. Measurable disease
6. performance status 0 or 1
7. Adequate organ function

Exclusion Criteria:

1. History or current evidence of calcium and phosphate homeostasis disorder
2. Current evidence of clinically significant retinal disorder
3. Treatment with any of the following within the specified time frame prior to the first dose of futibatinib:

   1. 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
   2. Patients with locoregional therapy, eg, transarterial chemoembolization (TACE), selective internal radiotherapy (SIRT) or ablation within 4 weeks
   3. Any non investigational anticancer therapy within 3 weeks or have not recovered from side effects of such therapy prior to futibatinib. Endocrine therapy is allowed for patients with breast or prostate cancer
   4. Targeted therapy or immunotherapy within 3 weeks or within 5 half lives Any investigational agent received within 5 half-lives of the drug or 4 weeks, whichever is shorter.
   5. Patients with prior FGFR-directed therapy
4. A serious illness or medical condition(s) including (but not limited to) the following:

   1. Known brain metastasis (not including primary brain tumors) unless patient is clinically stable for ≥1 month
   2. Known acute systemic infection
   3. Myocardial infarction, severe/unstable angina, symptomatic congestive heart failure (New York Heart Association \[NYHA\] Class III or IV New York Heart Association \[NYHA\] Classification) 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
   4. Significant gastrointestinal disorder(s) that could interfere with the absorption of futibatinib.
   5. 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. 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. Pregnant or lactating female.
7. Known hypersensitivity or severe reaction to futibatinib or its excipients.

Where this trial is running

La Jolla, California and 64 other locations

+15 more sites — see ClinicalTrials.gov for the full list.

Study contacts

How to participate

  1. Review the eligibility criteria above with your treating physician.
  2. Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
  3. Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.
View on ClinicalTrials.gov → Find more trials like this →
Conditions Advanced CholangiocarcinomaFGFR2 FusionsGene RearrangementFutibatinibAdvanced cholangiocarcinomacholangiocarcinomaFGFR2Fusion
Last reviewed 2026-06-13 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.