Study ASTX030-01 is a multi-phase study comprising of Phases 1-3 Monotherapy arms, and Phase 1 and Phase 2 Combination Therapy arms. Phase 1 Monotherapy consists of an open-label Dose Escalation Stage (Stage A) using multiple cohorts at escalating dose levels of oral cedazuridine and azacitidine (only one study drug will be escalated at a time) followed by a Dose Expansion Stage (Stage B). Phase 2 Monotherapy is a randomized, open-label, crossover study to compare oral ASTX030 to subcutaneous (SC) azacitidine. Phase 3 Monotherapy is a randomized open-label crossover study comparing the final fixed dose of oral ASTX030 to SC azacitidine. Phase 1 Combination Therapy is an open-label, multicenter, randomized, exploratory study comparing ASTX030 and SC azacitidine in combination with venetoclax in participants with treatment-naïve AML. Phase 2 Combination Therapy is an open-label, single arm, study evaluating the efficacy, safety, pharmacokinetics (PK), and drug interactions of ASTX030 in combination with venetoclax in participants with treatment-naïve AML.
The duration of this multi-phase study is approximately 8 years.
Eastern Cooperative Oncology Group (ECOG) performance status of 0 to 1.
Participants with adequate organ function.
For participants with prior allogeneic stem cell transplant, no evidence of graft-versus-host disease (GVHD).
Participants with no major surgery within 3 weeks before first study treatment.
Has an active uncontrolled gastric or duodenal ulcer.
Has poor medical risk because of other conditions.
Has known human immunodeficiency virus (HIV) infection.
Is known to be positive for Hepatitis B or C infection.
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Treatment groupsSee each treatment group below.