New Treatments & Clinical Trials for Cholangiocarcinoma

Cholangiocarcinoma is cancer of the bile ducts and is a rare but increasingly common cancer with historically poor outcomes. Most patients are diagnosed when surgery is no longer possible, but targeted therapies have begun to change survival trajectories.

What's actually going on in research

FGFR2 inhibitors (pemigatinib, futibatinib) are now in standard use for the 10–15% of intrahepatic cholangiocarcinomas with FGFR2 fusions, and IDH1 inhibitors add another target for IDH1-mutated tumors. Immune checkpoint inhibitors combined with chemotherapy have modestly improved outcomes for many patients, and combination immunotherapy trials are ongoing. Antibody-drug conjugates targeting HER2 and KRAS G12C inhibitors are expanding the targetable fraction of this disease.

What to know before you search

Eligibility depends on tumor location (intrahepatic vs. perihilar vs. distal), specific molecular alterations (FGFR2 fusion, IDH1/2 mutation), prior chemotherapy, and performance status.

What types of trials are currently open

• Targeted therapy trials — Testing FGFR2 inhibitors, IDH1 inhibitors, and KRAS inhibitors matched to specific mutations.
• Immunotherapy trials — Evaluating checkpoint inhibitor combinations with chemotherapy or other targeted agents.
• Surgical trials — Testing hepatic resection approaches, liver transplant criteria, and intraoperative techniques.
• Locoregional trials — Evaluating transarterial chemoembolization, radiation, and ablation for unresectable disease.
• Biomarker trials — Identifying genetic alterations in FGFR2, IDH1, BRAF, HER2, and KRAS to guide therapy matching.