A Phase III Study of Ivosidenib (AG-120) versus Placebo in Patients with Previously Treated Inoperable or Metastatic Cholangiocarcinoma

Full Title

AG120-C-005: A Phase 3, Multicenter, Randomized, Double-Blind, Placebo-controlled Study of AG-120 in Previously-treated Subjects with Nonresectable or Metastatic Cholangiocarcinoma with an IDH1 Mutation


Cholangiocarcinoma is cancer of the bile duct, and is very challenging to treat. Doctors are seeking better therapies for patients with cholangiocarcinoma that continues to grow despite treatment. In this study, researchers are comparing the drug ivosidenib (AG-120) with a placebo in patients with inoperable or metastatic cholangiocarcinoma that continues to grow after prior therapy.

Ivosidenib blocks an abnormal form of a protein called IDH1, which is involved in cancer cell metabolism. Abnormal IDH1 causes too much of a substance called 2-HG to be produced. Scientists believe that too much 2-HG fuels cancer growth. Patients in this study must have cholangiocarcinoma that contains a mutated form of the IDH1 gene. They will be randomly assigned to receive either ivosidenib or a placebo.

Ivosidenib is taken orally (by mouth). It is approved for treating leukemia; its use in this study is considered investigational.


To be eligible for this study, patients must meet several criteria, including but not limited to the following:

  • Patients must have inoperable or metastatic cholangiocarcinoma that continues to grow despite therapy.
  • Patients’ tumors must contain mutated IDH1.
  • Patients must recover from the serious side effects of previous treatments before entering the study.
  • Patients must be physically well enough that they are fully ambulatory, capable of all self care, and are capable of all but physically strenuous activities. As an example, patients must be well enough that they would be able to carry out office work or light housework.
  • This study is for patients age 18 and older.

For more information and to inquire about eligibility for this study, please contact Dr. Ghassan Abou-Alfa at 646-888-4184.