Alternative Dosing Regimens of Subcutaneous Azacitidine for Myelodysplastic Syndromes
Condition: Myelodysplastic Syndromes
MeSH terms: Preleukemia; Myelodysplastic Syndromes; Syndrome
Intervention: azacitidine
Intervention description: Azacitidine is administered subcutaneously
Total of 18 cycles on treatment or early discontinuation.
Intervention MeSH terms: Azacitidine
Phase: Phase 2
Study type: Interventional
Primary purpose: Treatment
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: None (Open Label)
Enrollment: 151.0
Sex: All
Minimum age: 18 Years
Healthy volunteers: No

Summary:

      The purpose of this study is to determine if azacitidine, combined with Best Supportive Care
      (BSC), is effective in treating myelodysplastic syndromes (MDS) when given according to a
      different doses and dosing schedules.
    

Eligibility criteria:

        Inclusion Criteria:

          -  Diagnosis of refractory anemia, refractory anemia with ringed sideroblasts and at
             least one of the following: a)Anemia with hemoglobin <110g/L and requires at least 1
             unit packed red blood cell transfusions every 28 days; b)Thrombocytopenia with
             platelet counts <100 x 10^9/L; or c)Neutropenia with absolute neutrophil count <1.5 x
             10^9/L.

          -  OR, Refractory anemia with excess blasts or refractory anemia with excess blast in
             transformation, according to the French-American-British classification system for
             MDS.

          -  At least 18 years of age.

          -  Have a life expectancy of >7 months.

          -  Unlikely to proceed to bone marrow or stem cell transplantation therapy following
             remission.

          -  Have serum bilirubin levels less than or equal to 1.5 times the upper limit of the
             normal (ULN) range for the laboratory.

          -  Have serum glutamic-oxaloacetic transaminase (aspartate aminotransferase) or serum
             glutamic-pyruvic transaminase (alanine aminotransferase) levels less than or equal to
             2 x ULN.

          -  Have serum creatinine levels less than or equal to 1.5 x ULN.

        Exclusion Criteria:

          -  Secondary MDS.

          -  Prior treatment with azacitidine.

          -  Any prior history of Acute Myeloid Leukemia (AML).

          -  Malignant or metastatic disease within the previous 12 months.

          -  Uncorrected red cell folate deficiency or vitamin B12 deficiency.

          -  Hepatic tumors.

          -  Radiation, chemotherapy, or cytotoxic therapy for non-MDS conditions in the previous
             12 months.

          -  Known or suspected hypersensitivity to azacitidine or mannitol.

          -  Prior transplantation or cytotoxic therapy to treat MDS. Prior use of Revlimid and
             Thalomid allowed after 30 day washout.

          -  Serious medical illness likely to limit survival to less than or equal to 7 months.

          -  Treatment with androgenic hormones during the previous 14 days

          -  Active viral infection with known human immunodeficiency virus or vial hepatitis Type
             B or C.

          -  Treatment with other investigational drugs with the previous 30 days.