A Phase 1/2 Pharmacokinetic Guided Dose-Escalation and Dose-Confirmation Study of ASTX727, A Combination of the Oral Cytidine Deaminase Inhibitor (CDAi) E7727 with Oral Decitabine in Subjects with Myelodysplastic Syndromes (MDS)
Trial Description
This first-in-human, 3-stage, open-label study evaluated the safety and pharmacokinetics
of ASTX727, as well as determined the dose for later stages.
Eligibility Requirements
Inclusion Criteria:
- International Prognostic Scoring System (IPSS) low, intermediate -1, intermediate-2,
or high risk MDS (including chronic myelomonocytic leukemia; CMML) in Dose
Escalation and Dose Confirmation-Randomization; only intermediate-2, or high risk
MDS in Dose Confirmation-Open Label
- Eastern Cooperative Oncology Group (ECOG) 0 to 2
- No major surgery within 2 weeks of starting study treatment
- No cytotoxic chemotherapy within 2 weeks of starting study treatment
- Able to swallow pills
Exclusion Criteria:
- Previous treatment with 2 or more courses of decitabine (all stages) or azacitidine
(Dose Confirmation stage only)
- Treatment with investigational therapy within 2 weeks of study treatment
- Uncontrolled medical disease(s) or active, uncontrolled infection
- Diagnosed with acute myeloid leukemia (AML)
- Active uncontrolled gastric or duodenal ulcer
- Known history of HIV or hepatitis C or B