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)

Protocol # :
Myelodysplastic Syndrome
Disease Sites
Lymphoid Leukemia
Myeloid and Monocytic Leukemia
Leukemia, other
Principal Investigator
Steensma, David, Peter
Site Investigator
Amrein, Philip, C.
Attar, Eyal, C.
Site Research Nurses
Toomey-Mathews, Ellen

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