IDH1 inhibitors — ivosidenib (FDA-approved) with new combination trials
Bispecific ADCs and antibodies — BL-B01D1, ivonescimab (PD-1/VEGF)
CAR-T cell therapy for CEA-positive biliary tract cancers
Standard of care: First-line: gemcitabine + cisplatin + durvalumab (TOPAZ-1 regimen). For resectable disease: surgery followed by adjuvant capecitabine (BILCAP). Second-line options depend on biomarkers — FGFR inhibitors, IDH1 inhibitors, or FOLFOX.
Subtypes of Cholangiocarcinoma
Cholangiocarcinoma (bile duct cancer) is classified by location, which affects both treatment and trial eligibility:
Intrahepatic (iCCA) — arises within the liver. Most common subtype in trials. Higher rate of FGFR2 fusions (~15%) and IDH1 mutations (~15%). Often grouped with liver cancers.
Perihilar (pCCA / Klatskin tumor) — at the junction of the left and right hepatic ducts. Often locally advanced at diagnosis. Fewer targeted therapy options.
Distal (dCCA) — in the bile duct near the small intestine. May be grouped with pancreatic cancer trials. Surgically resectable more often.
Most trials enroll all subtypes under "biliary tract cancer" (BTC), which also includes gallbladder cancer and ampullary cancer. ClinTrialFinder searches across all these terms automatically.
Key Biomarkers for Trial Eligibility
Molecular profiling is essential for cholangiocarcinoma — several biomarkers have FDA-approved or trial-specific therapies:
FGFR2 fusion/rearrangement — present in ~15% of intrahepatic CCA. FDA-approved: futibatinib, pemigatinib. Multiple combination trials recruiting.
IDH1 mutation — present in ~15% of intrahepatic CCA. FDA-approved: ivosidenib. New trials test combinations with immunotherapy and chemotherapy.
HER2 overexpression/amplification — present in ~5-15% (higher in extrahepatic). Zanidatamab (bispecific) in Phase 3. ADCs like trastuzumab deruxtecan and DB-1303 in trials.
MSI-H / dMMR — rare (~2-3%) but eligible for pembrolizumab (FDA-approved for all MSI-H cancers).
How do I find cholangiocarcinoma clinical trials I'm eligible for?
Enter your cholangiocarcinoma details into ClinTrialFinder — including subtype (intrahepatic, perihilar, or distal), biomarkers (FGFR2, IDH1, HER2), and prior treatments. The AI matches you with trials based on your specific profile in minutes. No login required.
What cholangiocarcinoma trials are currently recruiting?
There are 155 recruiting interventional trials for cholangiocarcinoma including FGFR inhibitors (futibatinib, pemigatinib), IDH1 inhibitors (ivosidenib), HER2-targeted therapies (zanidatamab, ADCs), immunotherapy combinations, bispecific antibodies, and CAR-T cell therapy for intrahepatic, perihilar, and distal bile duct cancer.
What is the difference between intrahepatic and extrahepatic cholangiocarcinoma?
Intrahepatic cholangiocarcinoma (iCCA) arises within the liver and has the highest rate of targetable mutations (FGFR2 fusions ~15%, IDH1 mutations ~15%). Extrahepatic includes perihilar (Klatskin tumor) and distal cholangiocarcinoma. Most trials enroll all subtypes under "biliary tract cancer" (BTC), but some targeted therapies are primarily studied in intrahepatic CCA.
Should I get molecular profiling for cholangiocarcinoma?
Yes — comprehensive genomic profiling is strongly recommended. Actionable mutations like FGFR2 fusions, IDH1 mutations, HER2 amplification, MSI-H, BRAF V600E, and NTRK fusions each have FDA-approved therapies or active clinical trials. Without profiling, you may miss targeted treatment options.
Find Cholangiocarcinoma Trials Matched to Your Situation
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