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