A Phase I Study of Oral AG-120 to Treat Advanced Acute Myeloid Leukemia or Myelodysplastic Syndromes with an IDH1 Mutation


Full Title

A Phase 1, Multicenter, Open-Label, Dose-Escalation and Expansion, Safety, Pharmacokinetic, Pharmacodynamic, and Clinical Activity Study of Orally Administered AG-120 in Subjects With Advanced Hematologic Malignancies With an IDH1 Mutation, With a Substudy in Subjects With Relapsed or Refractory Myelodysplastic Syndrome and a Substudy in Subjects With Advanced Hematologic Malignancies With Organ Impairment


The purpose of this study is to find the highest dose of the drug AG-120 that can be given safely in patients with acute myeloid leukemia (AML) or myelodysplastic syndromes (MDS) that have returned despite prior therapy or are not responding to standard therapy. Researchers will also observe the preliminary effectiveness of AG-120 against AML and MDS. AG-120 is taken orally (by mouth).

AG-120 blocks an abnormal form of a protein called IDH1. Abnormal IDH1 causes too much of a substance called 2-HG to be produced. Scientists believe that too much 2-HG fuels leukemia growth.


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

  • Patients must have AML or MDS that has either returned despite prior therapy or is not responding to standard therapy.
  • Patients with AML or MDS who are age 60 or older and who are not candidates for standard therapy (because they may not be able to tolerate the side effects) may also be eligible.
  • All patients must have mutated IDH1 .
  • At least 2 weeks must pass since completion of prior therapy and entry into the study.
  • 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.