A Clinical Trial of Decitabine in Patients With Myelodysplastic Syndrome

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.