A Multicenter, Randomized, Open-Label, Parallel-Group, Phase 3 Trial of Subcutaneous Azacitidine Plus Best Supportive Care Versus Conventional Care Regimens Plus Best Supportive Care for the Treatment of Myelodysplastic Syndromes (MDS)
- Conditions
- 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: 6.1Level: PTClassification code 10028533
- Registration Number
- EUCTR2004-001507-35-CZ
- Lead Sponsor
- Pharmion Corporation
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ot Recruiting
- Sex
- All
- Target Recruitment
- 32
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 <13,000 X109/L ;
2. Be =18 years of age;
3. Have a life expectancy =3 months;
4. Be unlikely to proceed to bone marrow or stem cell transplantation therapy following remission;
5. Be capable of giving informed consent and have signed the informed consent form (ICF);
6. Have an Eastern Cooperative Oncology Group (ECOG) Performance Status Grade of 0-2;
7. Have serum bilirubin levels =1.5 x the upper limit of the normal range for the laboratory (ULN). Higher levels are acceptable if these can be attributed to active hemolysis (as indicated by positive direct Coombs’ testing, decreased haptoglobin level, elevated indirect bilirubin and/or lactate dehydrogenase [LDH]), or ineffective erythropoiesis (as indicated by bone marrow findings);
8. Have serum glutamic-oxaloacetic transaminase (SGOT) (aspartate aminotransferase [AST]) or serum glutamic-pyruvic transaminase (SGPT) (alanine aminotransferase [ALT]) levels =2 x ULN;
9. Have serum creatinine levels =1.5 x ULN;
10. Women of childbearing potential may participate, providing they meet the following conditions:
a. Must agree to use at least 2 effective contraceptive methods throughout the
study and for 3 months following the date of the last dose of study medication.
b. Must have a negative serum pregnancy test obtained within 24 hours prior to Day 1.
11. Males with female partner of childbearing potential must agree to use at least 2 effective contraceptive methods throughout the study and should avoid fathering a child for 6 months following the date of the last dose of study medication.
Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range
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, including thalidomide and arsenic trioxide, 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.
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method
- Secondary Outcome Measures
Name Time Method