About cholangiocarcinoma

Cholangiocarcinoma is a cancer that begins in the bile duct—a small tube that leads from the liver and gallbladder, through the pancreas, and connects to the small intestine. The bile duct carries bile, a fluid made by the liver to help with digestion. Doctors classify cholangiocarcinoma according to the location of the initial tumor: either within the liver (intrahepatic) or, more commonly, outside the liver (extrahepatic).1

If caught early, surgery can remove the tumor.1 Radiation treatment can kill cancer cells by directing small doses of radiation to the tumor region.1 However, cholangiocarcinoma often spreads to other areas within and outside the liver.1,2 Once the disease reaches this advanced stage, doctors may prescribe chemotherapy to kill the cancer cells, but this may also kill healthy cells throughout the body.1,2

Cholangiocarcinoma begins in the bile duct, but each patient's cancer is fundamentally different. A unique combination of genetic changes (mutations) contributes to the cancer cells' out-of-control growth.1,2 A new wave of therapies called targeted therapies are being developed that specifically target only those mutations present in a patient's cancer.2

Diagram of patient anatomy, indicating location of liver, gallbladder, and pancreas

AG-120: Investigational medication for IDH1-mutated advanced cholangiocarcinoma

AG-120, developed by Agios, is a targeted therapy that targets cancer cells with a mutation in a gene called IDH1. Among patients with cholangiocarcinoma, 13%-15% may have an IDH1 mutation in their tumor.3,4

The ClarIDHy clinical trial is a Phase 3 trial testing the safety and efficacy of AG-120 in patients with IDH1-mutated advanced cholangiocarcinoma.

The safety and efficacy of AG-120 have not been established. There is no guarantee that AG-120 will receive health authority approval or become commercially available for the use being investigated.

IDH1 mutations appear in 13%-15% of patients with cholangiocarcinoma

Genomic testing in cholangiocarcinoma

Genomic testing can identify the unique combination of mutations present in a patient's cancer, including mutations in the IDH1 gene. This information can help you and your doctor determine if you are eligible for the ClarIDHy clinical trial.

Genomic testing identifies mutations. Diagrams of patient's DNA from normal cell and from cancerous cell

Is the ClarIDHy clinical trial right for you or your loved one?

To be eligible for the ClarIDHy clinical trial, patients must meet certain criteria, including5:

  • Be at least 18 years old
  • Have advanced cholangiocarcinoma that cannot be surgically removed
  • Have a mutation in IDH1 (genomic testing will be provided by the clinical trial)
  • Have been treated with cancer medication, but your disease has grown or spread

Talk to your doctor to determine your eligibility for the ClarIDHy clinical trial. For additional eligibility criteria, and to find a ClarIDHy clinical trial site near you, please visit ClinicalTrials.gov

Ask your doctor if you're eligible for the ClarIDHy clinical trial.

Clinical studies FAQs

  • What is the ClarIDHy clinical trial?

  • Why is this clinical trial being done?

  • Am I eligible for the ClarIDHy clinical trial?

  • How do I know if my tumor has an IDH1 mutation?

  • What if I don't have an IDH1 mutation?

  • If I want to join this trial, how do I find a participating site?


  1. Blechacz B. Cholangiocarcinoma: Current Knowledge and New Developments. Gut Liver. 2016;1-14. [Epub ahead of print]
  2. Chong DQ, Zhu AX. The landscape of targeted therapies for cholangiocarcinoma: current status and emerging targets. Oncotarget. 2016;7(29):46750‌-46767.
  3. Borger DR, Tanabe KK, Fan KC, et al. Frequent mutation of isocitrate dehydrogenase (IDH)1 and IDH2 in cholangiocarcinoma identified through broad-based tumor genotyping. Oncologist. 2012;17(1):72-79.
  4. Kipp BR, Voss JS, Kerr SE, et al. Isocitrate dehydrogenase 1 and 2 mutations in cholangiocarcinoma. Hum Pathol. 2012;43(10):1552-1558.
  5. Study of AG-120 in Previously Treated Advanced Cholangiocarcinoma With IDH1 Mutations (ClarIDHy). ClinicalTrials.gov. U.S. National Institutes of Health. https://www.clinicaltrials.gov/ct2/show/study/NCT02989857. Accessed December 14, 2016.