A Phase I Study of AG-120 or AG-221 with Induction and Consolidation Therapy for Patients Newly Diagnosed with Acute Myeloid Leukemia Who Have an IDH1 or IDH2 Mutation

Full Title

AG120-221-C-001: A Phase 1, Multicenter, Open-Label, Safety Study of AG-120 or AG-221 in Combination with Induction Therapy and Consolidation Therapy in Patients with Newly Diagnosed Acute Myeloid Leukemia with an IDH1 and/or IDH2 Mutation

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, followed by consolidation therapy (also delivered in the hospital over several cycles) to eliminate any remaining leukemia cells. In this study, researchers are studying the safety of the investigational drug AG-120 when given with induction and consolidation chemotherapy in patients newly diagnosed with AML who have IDH1 gene mutations. They also want to find the highest dose of the investigational drug AG-221 that can be given with chemotherapy in patients who have IDH2 mutations.

AG-120 blocks an abnormal form of a protein called IDH1, 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 the chemotherapy drugs used in this study are given intravenously.

If patients respond well to the study treatment during induction and consolidation therapy, they may be able to take AG-120 or AG-221 as maintenance therapy for up to one year.

Eligibility

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

  • Patients must have AML and a mutation in the IDH1 or IDH2 genes.
  • Patients may not have received prior treatment for AML.
  • 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

16-012

Phase

I

Investigator

Co-Investigators

Locations