Study of Ivosidenib (AG-120) vs. Placebo with Azacitidine for Untreated Acute Myeloid Leukemia with IDH1 Mutation

Inclusion Criteria

• Be 18 years or older and unable to undergo intensive chemotherapy due to age (75+), health conditions, or other reasons.
• Have newly diagnosed acute myeloid leukemia (AML) with at least 20% leukemia cells in the bone marrow.
• Have an IDH1 gene mutation.
• Be in good general health with a performance status score of 0 to 2.
• Have good liver and kidney function.
• Agree to provide blood and bone marrow samples during the study.
• Be able to understand and sign a consent form.
• Be willing to complete quality of life assessments.
• Women of childbearing potential must have a negative pregnancy test and agree to use effective birth control.

Exclusion Criteria

• Eligible and willing to receive intensive chemotherapy for AML.
• Have received any prior treatment for AML, except hydroxyurea.
• Have used a hypomethylating agent for myelodysplastic syndrome (MDS).
• Have taken an experimental drug for MDS recently.
• Have used an IDH1 inhibitor before.
• Have allergies to Ivosidenib, placebo, or Azacitidine.
• Are pregnant or breastfeeding.
• Have an active, uncontrolled infection.
• Have had another cancer, unless in remission for over a year.
• Have had serious heart disease recently.
• Have conditions affecting heart rhythm or drug absorption.
• Have uncontrolled high blood pressure.
• Have symptoms of leukemia affecting the brain or spinal cord.
• Have severe complications from leukemia.
• Have any condition that might interfere with the study.
• Are taking medications that affect heart rhythm unless they can be changed or monitored.
• Have a history of a specific brain infection.