A Clinical Trial of Decitabine in Patients With Myelodysplastic Syndrome

Phase I/II Trial of Subcutaneous Decitabine Maximizing Genomic Demethylation in Patients With Myelodysplastic Syndrome.

The purpose of this study was to determine which of the doses of decitabine maximizes genomic demethylation in patients with Myelodysplastic Syndrome (MDS).

Phase I: The purpose of this study was to determine which of the subcutaneous doses of decitabine administered twice daily for 5 days maximizes genomic demethylation in patients with MDS

Phase II: To evaluate hematological responses at the dose selected in the Phase I portion of the study.

Note: This study was planned as Phase 1/2 study but only the Phase 1 part was conducted due to a change in product development strategy

Inclusion Criteria:

1. Eastern Cooperative Oncology Group (ECOG) performance status score of 0-2.
2. Confirmed diagnosis of MDS or non-proliferative chronic myelomonocytic leukemia (CMML).

Exclusion Criteria:

1. Prior therapy with decitabine or azacytidine (Vidaza).
2. Experimental or standard drugs for the treatment of MDS within 28 days of the first day of study drug treatment.
3. Clinically significant anemia.
4. Prior history of malignancy other than MDS.
5. Any active infection.
6. Radiotherapy within 14 days prior to study enrollment.