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

Status: Recruiting
Phase: Phase 1/Phase 2
Diagnosis: Leukemia/MDS
NCT ID: NCT02103478 (View complete trial on
DFCI Protocol ID: 14-195


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 and duration.


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

Overall PI:
David Steensma, MD, Dana-Farber Cancer Institute

Site-responsible Investigators:
Philip Amrein, MD, Massachusetts General Hospital

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

Eligibility Criteria

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
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