CAR-T cell therapy for CEA-positive biliary tract cancers.
Personalized / biomarker-guided — NCT05615818 Personalized Medicine for Advanced Biliary Cancer (Phase 3, biomarker-stratified).
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 — FGFR2 fusion+ → futibatinib or pemigatinib; IDH1-mutated → ivosidenib; HER2+ → zanidatamab; MSI-H → pembrolizumab; otherwise 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. The FGFR2 fusion / IDH1 mutation / HER2 status results determine which trial-eligibility tier you fall into:
FGFR2 fusion / rearrangement — present in ~15% of intrahepatic CCA. FDA-approved targeted therapy: futibatinib (Lytgobi), pemigatinib (Pemazyre). Multiple combination trials are recruiting: pemigatinib + durvalumab (NCT06728410), pemigatinib + atezolizumab + bevacizumab (NCT06439485), tinengotinib vs physician's choice (NCT05948475 Phase 3), ICP-192 (NCT05678270). For patients with FGFR2 fusion documented on tumor next-generation sequencing or liquid biopsy, these trials are typically the highest-priority match.
IDH1 mutation — present in ~15% of intrahepatic CCA. FDA-approved: ivosidenib (Tibsovo). New trials test combinations with immunotherapy + chemotherapy (NCT06501625 1L ivosidenib + durvalumab + GemCis) and adjuvant use (NCT07260175).
HER2 overexpression / amplification — present in ~5-15% (higher in extrahepatic). Zanidatamab (HER2 × HER2 bispecific) Phase 3 (NCT06282575). Trastuzumab deruxtecan (T-DXd) + rilvegostomig Phase 3 in biliary (NCT06467357) — the registrational T-DXd biliary trial. Trastuzumab + chemo vs chemo alone 1L HER2+ BTC Phase 3 (NCT07062263).
MSI-H / dMMR — rare (~2-3%) but eligible for pembrolizumab (FDA-approved for all MSI-H cancers).
NTRK fusion — very rare but druggable with larotrectinib or entrectinib (FDA-approved).
Comprehensive genomic profiling (e.g., Foundation Medicine, Tempus, Guardant360) is recommended for ALL advanced cholangiocarcinoma patients — at least 30-40% of intrahepatic CCA have an actionable biomarker.
Know your FGFR2, IDH1, or HER2 status? Get matched to bile duct cancer trials in minutes.
If your tumor has an FGFR2 fusion or rearrangement (present in ~15% of intrahepatic CCA), the trials below are typically the best match. Both pemigatinib (Pemazyre) and futibatinib (Lytgobi) are FDA-approved; combination trials test how to extend or sequence them with immunotherapy:
NCT06728410 - Pemigatinib + Durvalumab in advanced intrahepatic cholangiocarcinoma with FGFR2 rearrangement / fusion, after GemCis + IO (Phase 1b/2)
NCT06439485 - Pemigatinib + Atezolizumab + Bevacizumab in advanced cholangiocarcinoma with FGFR2 fusion (Phase 1/2)
NCT05727176 - Futibatinib (FDA-approved FGFR inhibitor) in advanced cholangiocarcinoma with FGFR2 fusions / gene rearrangement
NCT05948475 - Tinengotinib vs Physician's Choice in FGFR-altered cholangiocarcinoma (Phase 3, the registrational tinengotinib trial)
NCT05678270 - ICP-192 in FGFR2-rearranged unresectable or metastatic cholangiocarcinoma
NCT06467357 - Trastuzumab Deruxtecan (T-DXd) + Rilvegostomig vs Standard of Care in advanced HER2-expressing biliary tract cancer (Phase 3 — the registrational T-DXd biliary trial)
NCT06282575 - Zanidatamab (HER2 × HER2 bispecific) + SOC vs SOC in 1L HER2+ biliary tract cancer (Phase 3)
NCT07062263 - Trastuzumab + chemotherapy vs chemotherapy alone in first-line HER2-positive advanced BTC (Phase 3)
First-Line (Treatment-Naive, No Targetable Mutation)
Standard first-line is GemCis + durvalumab (TOPAZ-1). Trials test additions and alternatives:
NCT07221253 - Rilvegostomig (PD-1 × TIGIT bispecific) or Durvalumab + GemCis in 1L advanced BTC (Phase 3)
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 243 recruiting interventional trials for cholangiocarcinoma / bile duct / biliary tract cancer in May 2026, including FGFR2 fusion-targeted (pemigatinib + durvalumab NCT06728410, pemigatinib + atezo + bev NCT06439485, futibatinib NCT05727176, tinengotinib vs physician's choice NCT05948475 Phase 3, ICP-192 NCT05678270), IDH1-mutated (ivosidenib + durvalumab + GemCis 1L NCT06501625, ivosidenib adjuvant NCT07260175), HER2-expressing (T-DXd + rilvegostomig Phase 3 NCT06467357 — registrational T-DXd biliary trial, zanidatamab Phase 3 NCT06282575, trastuzumab + chemo Phase 3 NCT07062263), 1L immunotherapy combinations (rilvegostomig NCT07221253, SHR-8068 + adebrelimab NCT07229625), bispecific ADCs (BL-B01D1, ivonescimab), 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
Enter your subtype, biomarkers, and treatment history to get AI-matched trial results in minutes.
This page is for information only and is not medical advice. ClinTrialFinder helps you find clinical trials that may match your situation, but enrollment decisions and treatment choices should always be made with your oncologist or healthcare team. Trial eligibility, recruitment status, and treatment details can change — verify directly with the trial sponsor or on ClinicalTrials.gov before acting on any information here.
