Cholangiocarcinoma is a rare cancer of the bile ducts, the tubes that carry digestive fluid from the liver. It's often caught late because early symptoms are vague, and treatment has historically been limited to chemotherapy and surgery when the tumor can be removed. Recent years have brought targeted therapies for people whose tumors carry specific genetic changes.
What's actually going on in research
Trials are testing targeted drugs for FGFR2 fusions, IDH1 mutations, and other genetic drivers found in subsets of patients. Immunotherapy combinations are being studied after some patients responded to checkpoint inhibitors. Researchers are also testing chemotherapy combinations, radiation techniques, and treatments for advanced disease that has spread to the liver or beyond.
Targeted therapies
FDA-approved drugs now block FGFR2 fusions and IDH1 mutations found in some cholangiocarcinomas. Trials are testing newer versions of these drugs and looking for additional genetic targets.
Immunotherapy combinations
Checkpoint inhibitors alone help a small fraction of patients, but trials are pairing them with chemotherapy or other immune drugs. Tumors with mismatch repair defects respond better to immunotherapy.
Locoregional treatments
Studies are testing radiation beads, ablation, and other techniques to treat tumors directly in the liver. These approaches may control disease when surgery isn't possible.
What to know before you search
Eligibility often depends on tumor location, whether it can be surgically removed, genetic test results, prior treatments, and liver function.
What types of trials are currently open
- Targeted therapy trials — Testing drugs that block specific genetic changes in your tumor, often after molecular testing identifies mutations like FGFR2 fusions or IDH1.
- Immunotherapy trials — Testing checkpoint inhibitors alone or with chemotherapy to help the immune system recognize cancer cells.
- Chemotherapy trials — Testing new combinations of chemotherapy drugs or comparing them to current standard regimens.
- Locoregional treatment trials — Testing radiation beads, ablation, or other treatments delivered directly to tumors in the liver.
- Surgical trials — Testing whether surgery combined with chemotherapy or radiation improves outcomes for tumors that can be removed.
Recently added Cholangiocarcinoma trials
Photodynamic Therapy Combined With a Self-expandable Radioactive Particle-loaded Biliary Stent for Unresectable Extrahepatic Cholangiocarcinoma
This is a prospective, single-arm, interventional clinical study. The purpose of this study is to evaluate the clinical efficacy and safety of Photodynamic Therapy (PDT) combined with a self-expandable radioactive particle-loaded biliary stent in patients with unresectable extrahepatic cholangiocarcinoma. Patients diagnosed with extrahepatic cholangiocarcinoma who are unable to undergo surgical resection will be enrolled. After initial percutaneous transhepatic cholangial drainage (PTCD) to improve liver function, patients will receive an intravenous injection of a photosensitizer (Hematoporphyrin, 3 mg/kg). Approximately 48-72 hours later, PDT will be performed under DSA guidance (1-2 sessions). Subsequently, a self-expandable biliary stent loaded with Iodine-125 radioactive particles will be implanted into the stricture to maintain biliary patency and provide local brachytherapy. The primary outcome is tumor response or biliary patency evaluated by RECIST v1.1. Secondary outcomes include overall survival, stent patency duration, changes in liver function and tumor markers, and incidence of adverse events. This study aims to provide a new, effective treatment strategy for unresectable extrahepatic cholangiocarcinoma.
SumMET: Study of [225Ac]Ac-FL-261 in Participants With Solid Tumors Overexpressing c-MET
This is a first-in-human, multicenter, open-label Phase 1/2 study designed to evaluate the safety, efficacy, and tolerability of \[225Ac\]Ac-FL-261, a targeted radioligand therapy directed at c-MET, in participants with advanced or metastatic solid tumors. Phase 1 will identify the recommended dose through dose escalation, followed by a randomized comparison of two candidate dosing regimens (dose optimization). In Phase 2, the selected dose will be tested in independent cohorts by tumor type.
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