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Pharmacokinetic Guided Dose Escalation and Dose Confirmation With Oral Decitabine and Oral CDAi in Patients With MDS


Trial ID:NCT02103478

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Protocol #:14-195


Phase:Phase 1/Phase 2

Principal Investigator:David Steensma, MD

Site Investigator(s):Philip Amrein, MD

Trial Description:
This 2-stage, open-label study will evaluate safety and pharmacokinetics of ASTX727, as well as determine the dose for the study's second stage. In the second stage the selected dose will be confirmed and evaluated for clinical activity, including response rate.

Ilene Galinsky, Dana-Farber Cancer Institute, 617-632-3902,
Cancer Trials Call Center, Massachusetts General Hospital, 877-789-6100

Conducting Institutions:
Dana-Farber Cancer Institute
Brigham and Women's Hospital
Massachusetts General Hospital

Eligibilty Requirements:
Inclusion Criteria:

- IPSS low, intermediate -1, intermediate-2, or high risk MDS (including CMML) in Dose Escalation and Dose Confirmation-Randomization; only intermediate-2, or high risk MDS in Dose Confirmation-Open Label

- 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 AML

- Active uncontrolled gastric or duodenal ulcer

- Known history of HIV or hepatitis C or B