Cholangiocarcinoma (CCA) Clinical Trials and Treatments

Cholangiocarcinoma (CCA) is a heterogeneous group of cancers arising from the epithelial cells lining the bile ducts. It is classified by anatomic location: intrahepatic (within the liver), perihilar (at the liver hilum, also called Klatskin tumors), and distal (in the common bile duct). Each subtype has distinct biology, treatment approaches, and prognosis.

The most common symptoms of Cholangiocarcinoma include Jaundice (yellowing of skin and eyes), Intense itching (pruritus) without rash, Light-colored or clay-colored stools, Dark urine, Unintentional weight loss and loss of appetite, among others. About 8,000 new cases per year in the U.S.; rising incidence worldwide. Symptoms can range from mild to severe and may vary between individuals.

Trial Friend lists all active clinical trials for Cholangiocarcinoma from ClinicalTrials.gov, with eligibility criteria translated into plain English. Patients can filter by trial phase and recruitment status, search by location, and use the Match Me tool to score trials against their profile. Comprehensive genomic profiling is strongly recommended for all cholangiocarcinoma patients. About 10-20% of intrahepatic CCA has FGFR2 fusions (treatable with pemigatinib, futibatinib, or lirafugratinib, approved as Lyrfigtu in September 2026), and about 10-15% has IDH1 mutations (treatable with ivosidenib).

Yes. As of September 2026, Trial Friend lists 7 FDA-approved drugs used to treat Cholangiocarcinoma: Tibsovo (ivosidenib), first approved in 2018; Pemazyre (pemigatinib), first approved in 2020; Imfinzi (durvalumab), first approved in 2022; Lytgobi (futibatinib), first approved in 2022; Keytruda (pembrolizumab), first approved in 2023; Ziihera (zanidatamab), first approved in 2024; and Lyrfigtu (lirafugratinib), first approved in 2026.

Each has its own page with what the label covers, side effects and how it is given, and the Treatments section of this page adds any further approved drugs found in FDA labeling data.

Cholangiocarcinoma arises from acquired (somatic) genetic changes in the affected cells, commonly involving FGFR2 fusion, IDH1 and ERBB2 amplification, rather than from an inherited variant. Tumor testing guides targeted therapy: FGFR2 fusions in about 15% of intrahepatic tumors and IDH1 variants in up to 15% of bile duct cancers; there is no inherited gene test for the disease itself. About 8,000 new cases per year in the U.S.; rising incidence worldwide.

For Cholangiocarcinoma the genetic testing that matters is usually of the tumor or affected blood cells rather than inherited DNA, looking for FGFR2 fusion, IDH1 and ERBB2 amplification. Tumor testing guides targeted therapy: FGFR2 fusions in about 15% of intrahepatic tumors and IDH1 variants in up to 15% of bile duct cancers; there is no inherited gene test for the disease itself.

Those results guide treatment choice and are often the entry requirement for targeted-therapy trials, so ask whether the biopsy was sent for molecular testing and request the report. The Genetic Testing section of this page lists laboratories in the NIH Genetic Testing Registry.

Eligibility for Cholangiocarcinoma clinical trials depends on factors like age, diagnosis, treatment history, and overall health. Each trial sets its own inclusion and exclusion criteria. Trial Friend translates these requirements from medical language into plain English so patients can review them more easily. The Match Me tool can score your profile against active trials to help identify studies that may be a fit.

Participating in a Cholangiocarcinoma clinical trial typically involves scheduled visits, medical tests, and close monitoring by a research team. Depending on the study phase, you may receive an investigational treatment, a placebo, or the current standard of care. Trials may last from a few weeks to several years. Study participation is voluntary, and you can withdraw at any time.

Often, yes, and usually because of cancer treatment you have already had. In a Trial Friend review of 216 cholangiocarcinoma treatment trials that were recruiting or about to open on ClinicalTrials.gov on September 28, 2026, 20% had exclusion criteria based on PD-1 or PD-L1 immunotherapy such as Imfinzi (durvalumab) or Keytruda (pembrolizumab), 33% on steroids, 9% on blood thinners and 3% on FGFR inhibitors such as Pemazyre, Lytgobi or Lyrfigtu. Trials that set a break after your last cancer treatment usually ask for 1 to 4 weeks. Trial Friend's medication conflict checker scans each open trial's exclusion criteria for your medicines, including chemotherapy (gemcitabine, cisplatin, capecitabine and FOLFOX), targeted drugs (Tibsovo, Ziihera, Tafinlar and Mekinist), steroids and blood thinners, and shows the trial's exact wording with a confidence level so you can review it with your oncologist.