Full TitleA Phase 1, Multicenter, Open-Label, Dose-Escalation, Safety, Pharmacokinetic, Pharmacodynamic, and Clinical Activity Study of Orally Administered AG-120 in Subjects with Advanced Hematologic Malignancies with an IDH1 Mutation
The purpose of this study is to find the highest dose of an investigational drug called 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 be 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.