The myelodysplastic syndromes (MDS) are a group of potentially acute myeloid leukemic disorders. The disorders of myeloid origin include acute myeloid leukemia (AML), MDS and myeloproliferative disorders such as chronic myeloid leukemia. MedDRA version: 6.1 Level: PT Classification code 10028533
Conditions
Interventions
Trade Name: Vidaza
Product Name: Azactidine
Pharmaceutical Form: Powder and solvent for solution for injection
INN or Proposed INN: Azacitidine
CAS Number: 320-67-2
Concentration unit: mg/m2 milligram
Sponsors
Pharmion Corporation
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1. Have an IPSS score of INT-2 or High and a diagnosis of RAEB or RAEBT per FAB classification criteria or a diagnosis of Myelodysplastic CMMoL per modified FAB criteria meeting the following: a. Monocytosis in peripheral blood >1x109/L, b. Dysplasia in one or more myeloid cell lines, c. 10 to 29% blasts in the BM d. WBC =65 years) yes F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: 1. Secondary MDS, i.e., MDS that is known to have arisen as the result of chemical injury or treatment with chemotherapy and/or radiation for other diseases; 2. Any prior treatment with azacitidine; 3. Any prior history of AML; 4. Any diagnosis of malignant disease within the previous 12 months (excluding basal cell carcinoma with no complications); 5. Any diagnosis of metastatic disease; 6. Have hepatic tumors; 7. Radiation therapy, chemotherapy, or cytotoxic therapy, given to treat conditions other than MDS and administered within the previous 12 months prior to the first day of treatment (Day 1); 8. Known or suspected hypersensitivity to azacitidine or mannitol; 9. Prior or active disease that, in the opinion of the Investigator, may interfere with the procedures or evaluations to be conducted in the study; 10. Prior transplantation or cytotoxic therapy, including azacitidine and chemotherapy, administered to treat MDS; 11. Serious medical illness likely to limit survival to =12 months after screening or likely to prevent granting of informed consent (e.g., history of severe congestive heart failure, clinically unstable cardiac disease, or pulmonary disease); 12. Psychiatric illness that would prevent granting of informed consent; 13. Treatment with erythropoietin or myeloid growth factors (granulocyte colony-stimulating factor [G-CSF] or granulocyte-macrophage colony-stimulating factor [GM-CSF]) during the previous 21 days prior to Day 1; 14. Treatment with androgenic hormones during the previous 14 days prior to Day 1; 15. Active viral infection with known human immunodeficiency virus (HIV) or viral hepatitis type B or C; 16. Treatment with other investigational drugs within the previous 30 days prior to Day 1, or ongoing adverse events from previous treatment with investigational drugs, regardless of the time period; 17. Within the 28-day screening period, documented red cell folate deficiency, as evidenced by red blood cell folate (not serum folate) or vitamin B12 deficiency.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To determine the effect of azactidine plus Best Supportive Care, as compared with Conventional Care Regimens plus Best Supportive Care, on survival in MDS patients.;Secondary Objective: To determine the • effect of azacitidine plus Best Supportive Care (BSC), relative to Conventional Care Regimens (CCR) plus BSC, on hematologic status and episodes of infections requiring IV antibiotics ; • time to Relapse after complete remission (CR) or partial remission (PR), or Disease Progression , censored at death, in MDS patients treated with azacitidine plus BSC, compared to patients receiving CCR plus BSC; • time to transformation to AML, censored at death, in MDS patients treated with azacitidine plus BSC compared to patients receiving CCR plus BSC; • effect of azacitidine plus BSC, relative to that of CCR plus BSC on time to AML transformation or death from any cause; • safety and toxicity of azacitidine plus BSC, relative to CCR plus BSC in MDS patients; • to examine pharmacoeconomic differences in MDS patients treated with azacitidine plus BSC, compared to patients receiving CCR plus BSC.;Primary end point(s): Time to death from any cause. | — |
Countries
Czech Republic, Hungary
Outcome results
None listed