Cholangiocarcinoma is a rare cancer that starts in the bile ducts, the tubes carrying digestive fluid from the liver. About 8,000 people are diagnosed in the US each year. Surgery offers the best chance of cure when the cancer is caught early, but most cases are found at advanced stages where treatment focuses on slowing growth and managing symptoms.
What's actually going on in research
Trials are testing targeted drugs that match specific genetic mutations found in bile duct cancers, including FGFR2 inhibitors and IDH1 inhibitors already approved for some patients. Researchers are also studying immunotherapy combinations, newer chemotherapy regimens, radiation techniques, and ways to deliver treatment directly into bile ducts. Because each tumor has different genetic drivers, molecular testing is becoming standard.
FGFR2 inhibitors
About 15% of bile duct cancers have FGFR2 fusions that make tumors depend on this growth signal. FDA-approved drugs like pemigatinib and futibatinib target this pathway, and trials are testing newer versions with fewer side effects.
IDH1 mutations
Ivosidenib was approved in 2021 for bile duct cancers with IDH1 mutations, found in about 13% of these tumors. Studies are testing it earlier in treatment and in combination with other drugs.
Immunotherapy combinations
While checkpoint inhibitors alone rarely work for bile duct cancer, trials are testing them with chemotherapy, targeted drugs, or other immune treatments. Early results suggest combinations may help more patients than single agents.
What to know before you search
Eligibility typically depends on tumor location (inside or outside the liver), whether cancer has spread, liver function, prior treatments, and specific genetic mutations in your tumor.
What types of trials are currently open
- Targeted therapy trials — Testing drugs matched to specific genetic changes in your tumor, such as FGFR2 fusions, IDH mutations, HER2 amplification, or BRAF mutations. Requires molecular testing of tumor tissue.
- Immunotherapy trials — Testing checkpoint inhibitors and other immune treatments, often combined with chemotherapy or targeted drugs. May focus on tumors with high microsatellite instability.
- Chemotherapy trials — Testing new combinations or sequences of chemotherapy drugs, including drugs not yet approved for bile duct cancer.
- Radiation trials — Testing newer radiation techniques like stereotactic body radiation or radiation given during surgery. Some trials combine radiation with drugs that make cancer cells more sensitive.
- Regional treatment trials — Testing ways to deliver chemotherapy or radiation directly to the liver through its blood vessels, which may allow higher doses with fewer side effects.
Recently added Cholangiocarcinoma trials
Modified Single-Incision Plus One-Port Versus Conventional Five-Port Laparoscopic Pancreaticoduodenectomy for Nonpancreatic Periampullary Adenocarcinomas
This multicenter, randomized, controlled, non-inferiority trial will compare modified single-incision plus one-port laparoscopic pancreaticoduodenectomy (mSILPD+1) with conventional five-port laparoscopic pancreaticoduodenectomy (CLPD) in adults with histologically confirmed or highly suspected non-pancreatic periampullary carcinoma who are candidates for pancreaticoduodenectomy. A total of 232 participants will be randomly assigned in a 1:1 ratio. Outcome assessors who adjudicate complications and interpret imaging will be masked to treatment assignment. The primary outcomes are all-cause mortality within 90 days after surgery and overall survival at 1 year after surgery. Secondary outcomes will assess postoperative complications, surgical performance, postoperative recovery, disease-free survival, quality of life, and health-care costs.
Study on Sequential Cryoablation, Relaforp Alpha, and Chemotherapy for Bile Duct Tumors
This study is a prospective, multicenter, single-arm, open-label Phase II clinical trial, planned to enroll 30 patients with unresectable locally advanced or metastatic iCCA/GBC. Patients will first receive cryoablation treatment, and two weeks after ablation, they will receive 8 cycles of immunotherapy combined with chemotherapy (Relatlimab α plus GC chemotherapy, every 3 weeks). During the maintenance phase, patients will continue with Relatlimab α alone, every 3 weeks, for up to one year. During the combination treatment phase, efficacy will be evaluated every 6 weeks, and during the maintenance phase, every 6 to 9 weeks. From the date of randomization/enrollment, survival follow-ups will be conducted every 3 months (±14 days) for the first 2 years; afterwards, follow-ups will occur every 6 months (±28 days) until the participant dies, is lost to follow-up, or the study ends, whichever comes first.
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