A Phase II Study of Azacitidine Alone or with AG-120 or AG-221 in Patients Newly Diagnosed with Acute Myeloid Leukemia

Full Title

AG-221-AML-005: A Phase 1B/2 Open-Label, Randomized Study of 2 Combinations of Isocitrate Dehydrogenase (IDH) Mutant Targeted Therapies Plus Azacitidine: Oral AG-120 Plus Subcutaneous Azacitidine and Oral AG-221 Plus SC Azacitidine in Subjects with Newly Diagnosed Acute Myeloid Leukemia Harboring an IDH1 or an IDH2 Mutation, Respectively, Who are Not Candidates to Receive Intensive Induction Chemotherapy (Version date 25-OCT-2016)

Purpose

The standard treatment for acute myeloid leukemia (AML) may include induction chemotherapy (delivered daily, in the hospital) for several weeks to get rid of as many leukemia cells as possible. However, some people can’t receive induction therapy because of their age or other medical conditions. They may instead receive another treatment, such as the anticancer drug azacitidine. In this study, researchers are assessing the combination of azacitidine with the investigational drugs AG-120 or AG-221 in newly diagnosed patients with AML that contains a mutation in the IDH1 and/or IDH2 gene.

AG-120 blocks an abnormal form of the IDH1 protein, which is involved in cancer cell metabolism. AG-221 blocks a mutated form of the IDH2 protein. Abnormal IDH1 and IDH2 cause too much of a substance called 2-HG to be produced, which may make leukemia cells grow. AG-120 and AG-221 are taken orally (by mouth), while azacitidine is given as a subcutaneous (under the skin) injection.

Patients in this study will be randomly assigned to one of two groups: azacitidine alone, or azacitidine plus AG-120 (for patients with IDH1 mutations) or AG-221 (for those with IDH2 mutations).

Eligibility

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

  • Patients must have newly diagnosed AML that contains an IDH1 or IDH2 mutation and cannot be treated with standard induction therapy.
  • Patients must be able to walk and do routine activities for more than half of their normal waking hours.
  • This study is for patients age 18 and older.

For more information and to inquire about eligibility for this study, please contact Dr. Eytan Stein at 212-639-3314.

Protocol

16-1550

Phase

I/II

Investigator

Co-Investigators