A Phase 1, Multicenter, Open-Label, Dose-Escalation, Safety, Pharmacokinetic, Pharmacodynamic, and Clinical Activity Study of Orally Administered AG-221 in Subjects with Advanced Hematologic Malignancies with an IDH2 Mutation

Study Status

Open to Enrollment

Study Description

This clinical trial is for men and women with advanced hematologic malignancies. The study is evaluating an experimental drug called AG-221.

IDH2 is a type of protein involved in providing the body's cells with energy or metabolism. In certain types of diseases, like AML and MDS, an abnormal form of IDH2 is present in the diseased cells. When this happens, it produces an excess amount of 2-HG, a substance normally present in cells in low levels. Excessive amounts of 2-HG may prevent cells from maturing into normally functioning cells and may cause them to become diseased.

 AG-221 may stop the abnormal IDH2 protein and may reduce 2-HG levels in diseased cells so that they can become normal cells. The purpose of this study is to identify and study the highest dose of AG-221 that can be given safely and to determine the effects the drug has on hematologic cancers. AG-221 is a tablet taken by mouth.

 The study has 2 parts (participants will only be enrolled in 1 part).

In Part 1, different groups of participants will receive increasing dose levels of AG-221 to find the dose that has the most effect while still being tolerated by participants. Part 2 will study how the maximum tolerated dose (determined in Part 1) affects the cancer.

Participants will take AG-221 twice daily on every day of 28 day cycles. Participants will continue on study treatment for as long as they are responding to therapy and not experiencing unacceptable side effects. 

Disease Status and/or Stage

Advanced hematologic malignancies


Agios Pharmaceuticals

Key Eligibility

  • Men and women age 18 and older
  • Advanced hematologic malignancy, including: Relapsed and/or primary refractory acute myeloid leukemia (AML); Untreated AML in men and women age 60 and older; Myelodysplastic syndrome (MDS)
  • IDH2 gene mutation (testing done at screening)
  • Detailed eligibility reviewed when you contact the study team

Healthy Volunteers


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Contact Us

For general inquiries, or if you need assistance finding a study, please contact:

Erica Bersin
Tel: (646) 962-8232
[email protected]

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