Pharmacokinetic Guided Dose Escalation and Dose Confirmation With Oral Decitabine and Oral Cytidine Deaminase Inhibitor (CDAi) in Patients With Myelodysplastic Syndromes (MDS)

• Status: Completed
• Trial end date: 2019-12-04
• Target enrollment: 0
• Participant gender: All

Summary

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.

• Phase: Phase 1/Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Astex Pharmaceuticals; Astex Pharmaceuticals, Inc.
• Treatments: Azacitidine; Decitabine

Criteria

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