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.
FGFR2 inhibitors
Drugs targeting FGFR2 fusions produce meaningful responses in intrahepatic cholangiocarcinoma patients with this alteration, representing one of the first precision oncology successes in bile duct cancer.
IDH1 inhibitors
Ivosidenib targets IDH1 mutations found in about 10–20% of intrahepatic cholangiocarcinomas, extending progression-free survival in this molecularly defined subset.
Checkpoint plus chemotherapy
Adding PD-L1 inhibitors to standard gemcitabine-cisplatin chemotherapy has improved overall survival for unresectable disease, and trials are testing additional immunotherapy combinations.
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.
Recently added Cholangiocarcinoma trials
Chidamide Combined With Chemotherapy and Immunotherapy as First-line Treatment for Advanced Intrahepatic Cholangiocarcinoma
This is a single-center, open-label, phase II clinical trial designed to evaluate the efficacy and safety of chidamide in combination with chemotherapy and immunotherapy as a first-line treatment for patients with advanced intrahepatic cholangiocarcinoma (ICC), a type of liver cancer. The study will enroll approximately 35 patients with histologically or pathologically confirmed unresectable or metastatic ICC who have not received prior systemic therapy. All participants will receive chidamide, an oral HDAC inhibitor, in combination with gemcitabine, cisplatin, and an immune checkpoint inhibitor. Treatment will be administered in 21-day cycles until disease progression, unacceptable toxicity, or other withdrawal criteria are met. The primary study endpoints are objective response rate (ORR), and safety, evaluated by the frequency and severity of adverse events. Secondary endpoints include progression-free survival (PFS), duration of response (DOR), overall survival (OS), and exploratory biomarker analyses. The study aims to assess whether the addition of chidamide to standard chemotherapy and immunotherapy can improve treatment outcomes in this patient population.
A Study of ASP546C in Adults With Gastroesophageal Cancer, Pancreatic Cancer or Other Solid Tumors
This study will help find the most suitable dose of ASP546C in people with gastric cancer, gastroesophageal junction (GEJ) cancer, pancreatic cancer, and other specific solid tumors. GEJ is where the food pipe (esophagus) joins the stomach. This study is in 2 parts. In both parts of the study, ASP546C will be given once in 3-week cycles. It will be given slowly through a tube into a vein. This is called an infusion. In Part 1, people with gastric cancer or GEJ cancer can take part. They will receive an infusion of either a higher dose or a lower dose of ASP546C. In Part 2, people with pancreatic cancer or who have one of the other solid tumors can take part. Part 2 doesn't include people with gastric cancer or GEJ cancer. All people in this part of the study will receive an infusion of the higher dose of ASP546C. People will visit the clinic on certain days to receive ASP546C and have health checks. The number of visits and checks done during the study will depend on the health of each person and whether they are still receiving infusions of ASP546C.
Find Cholangiocarcinoma trials matched specifically to you
Answer 3 quick questions and we'll show you trials that fit your situation.