AG120-C-001 PH1 in IDH1 Mutation (AML, MDS, other Hematologic Malignancies) - Clinical Trial

What is the Purpose of this Study?


We are doing this study to find out if an experimental drug called AG-120 (the study drug) is an effective treatment for AML, MDS, or other hematologic malignancies (diseases of the blood or bone marrow).

What is the Condition Being Studied?

Acute Myeloid Leukemia (AML), Myelodysplastic Syndrome (MDS), and other Hematologic Malignancies

Who Can Participate in the Study?

Key Eligibility

Adults ages 18+ diagnosed with hematologic malignancy who:
- Are not getting better with standard therapies; OR
- Had their disease come back after previous therapy; OR
- Cannot be treated with standard therapies; OR
- Do not have normal function of their liver or kidneys

For more information about who can join this study, please contact the study team at 919-660-2077.

Age Group

What is Involved?


If you choose to join this study, you will:
- Take the study drug by mouth once per day
- Have physical exams
- Have blood draws
- Have heart scans (ECG and echocardiogram)
- Have bone marrow biopsies

You will take the study drug for as long as your study doctor thinks is appropriate. Your use of the study drug will end if any of the following events happen:
- You have severe side effects
- You receive a stem cell transplant
- You become pregnant
- Your cancer gets worse
- You choose to leave the study

Study Details

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
Principal Investigator
Hematologic Oncologist
Protocol Number
IRB: PRO00104299
NCT: NCT02074839
Phase I/II
Enrollment Status
Open for Enrollment