study of azacitidine to evaluate safety and effectiveness for chinese patients with higher risk myelodysplastic syndrome

Conditions: Myelodysplastic Syndrome (MDS)

Summary: the purpose of the study is to determine whether azacitidine is safe and effective in the treatment of chinese patients with higher risk myelodysplastic syndromes (mds).


Eligibility criteria:
inclusion criteria: subjects must satisfy the following criteria to be enrolled in the study: * chinese males and females of asian descent ≥ 18 years of age at the time of signing the informed consent document; * must have a documented diagnosis of refractory anemia with excess blasts (raeb) or refractory anemia with excess blasts in transformation (raeb-t) according to french-american-british (fab) classification for myelodysplastic syndrome (mds) and with an international prognostic scoring system (ipss) score of intermediate-2 or high risk or a diagnosis of myelodysplastic chronic myelomonocytic leukemia (cmml) per modified fab criteria meeting the following: * monocytosis in peripheral blood \> 1 x 10\^9/l; * dysplasia in one or more myeloid cell lines; * 10% to 29% blasts in the bone marrow; and white blood cell (wbc) count \< 13 x 10\^9/l * eastern cooperative oncology group (ecog) performance status of 0, 1, or 2 ; * adequate organ function, defined as: * serum bilirubin ≤ 1.5 times the upper limit of normal (uln); * serum aspartate aminotransferase (ast) and alanine aminotransferase (alt) ≤ 2.0 times the uln; * serum creatinine ≤ 1.5 times the uln; * females of childbearing potential (fcbp) must: * agree to the use of a physician-approved contraceptive method (oral, injectable, or implantable hormonal contraceptive; tubal ligation; intra-uterine device; barrier contraceptive with spermicide; or vasectomized partner) while on azacitidine; and * for 3 months following the last dose of azacitidine; and have a negative serum pregnancy test within 72 hours prior to starting investigational product (ip). * male subjects with a female partner of childbearing potential must agree to the use of a physician-approved contraceptive method throughout the course of the study and avoid fathering a child during the course of the study and for 3 months following the last dose of azacitidine; * understand and voluntarily sign an informed consent document prior to any study related assessments/procedures are conducted; * able to adhere to the study visit schedule and other protocol requirements. exclusion criteria: the presence of any of the following will exclude a subject from enrollment: * previous treatment with azacitidine or decitabine; * diagnosis of malignant disease within the previous 12 months (excluding basal cell carcinoma of the skin without complications, "in-situ" carcinoma of the cervix or breast, or other local malignancy excised or irradiated with a high probability of cure); * uncorrected red cell folate deficiency or vitamin b12 deficiency; * diagnosis of metastatic disease; * malignant hepatic tumors; * known or suspected hypersensitivity to azacitidine or mannitol; * candidate to proceed to bone marrow or stem cell transplant during the study; * prior transplantation or cytotoxic therapy, including azacitidine and chemotherapy, administered to treat mds; * treatment with erythropoietin or myeloid growth factors (granulocyte colony-stimulating factor \[g-csf\] or granulocyte-macrophage colony-stimulating factor \[gm-csf\]) during the 21 days prior to day 1 of cycle 1; * treatment with androgenic hormones during the 14 days prior to day 1 of cycle 1; * active viral infection with known human immunodeficiency virus (hiv) or viral hepatitis type b or c; * treatment with other investigational drugs, including thalidomide and arsenic trioxide, within the previous 30 days prior to day 1 of cycle 1, or ongoing adverse events from previous treatment with investigational drugs, regardless of the time period; and * pregnant or lactating females; * any significant medical condition, laboratory abnormality, or psychiatric illness that would prevent the subject from participating in the study; * any condition including the presence of laboratory abnormalities, which places the subject at unacceptable risk if he/she were to participate in the study; * any condition that confounds the ability to interpret data from the study.