Safety and Pharmacokinetics Study of SyB C-1101 in Patients With Recurrent/Relapsed or Refractory Myelodysplastic Syndrome (MDS)

Inclusion Criteria

Patients must satisfy the following conditions listed below:

• Patients who have been histologically documented or cytologically confirmed with myelodysplastic syndrome (MDS), and who have been found to meet any of the following criteria on the basis of the World Health Organization (WHO) classification or the French-American-British (FAB) classification.
• Refractory Anemia (RA)* ( = 15% ringed sideroblasts)
• RA with Excess of Blasts-1(RAEB-1)(5% to 9% myeloblasts)
• RAEB-2 (10% to 19% myeloblasts)
• RAEB-t (20% to 29% myeloblasts or = 1,000/mm^3 peripheral monocytes, < 13,000/mm^3 leukocytes) * RA and RARS patients should have 2 or more units of erythrocyte transfusion within 8 weeks.
• Patients with a low value in at least one blood cell lineage (having at least one of the following cytopenias).
• Neutrophils : < 1,800/mm^3
• Platelets : < 100,000/mm^3
• Hemoglobin : < 10 g/dL
• Patients with a previous history of chemotherapy (including immunosuppressive therapy, anabolic steroid and lenalidomide) for the target disease who meet any of the following criteria.
• Patients who have not achieved complete remission, partial remission, or hematologic improvement.*
• Patients with recurrence/relapse after complete remission, partial remission, or hematologic improvement.*
• Patients with intolerability that has led to discontinuation of treatment because of the development of liver dysfunction, kidney dysfunction, etc., after the start of treatment. *Proximate therapeutic efficacy judged under International Working Group (IWG) 2006 criteria.
• For the patients who have received chemotherapy such as immunosuppressive therapy, anabolic steroid and lenalidomide, the patients should have not been treated for four weeks or longer after the end of the previous therapy and be judged to have no residual effects (antitumor effects) from the previous therapy.
• Patients who can be expected to survive at least three months or longer.
• Patients at least 20 years old (when informed consent is obtained).
• Patients who have score of 0 to 2 in The Eastern Cooperative Oncology Group (ECOG) Performance Status (PS).
• Patients with adequate function in major organs (heart, lungs, liver, kidneys, etc.).
• Aspartate aminotransferase (AST) : no greater than 3.0 times the upper boundary of the reference range at each institution
• Alanine aminotransferase (ALT) : no greater than 3.0 times the upper boundary of the reference range at each institution
• Total bilirubin: no more than 1.5 times the upper boundary of the reference range at each institution
• Serum creatinine: no more than 1.5 times the upper boundary of the reference range at each institution
• ECG: no abnormal findings requiring treatment
• Echocardiography: no abnormal findings requiring treatment
• Patients who personally signed an informed consent document for participation in this study.

Exclusion Criteria

Patients who satisfy any of the following conditions will not be enrolled in the study.

• Patients with anemia caused by factors other than MDS (hemolytic anemia, gastrointestinal (GI) bleeding, etc.)
• Patients with hypoplasia MDS (< 10% osteocyte density)
• Patients who have undergone treatment for an active malignant tumor within the past year (except basal cell or squamous cell skin cancer, or primary squamous cell carcinoma of the cervix or noninvasive breast cancer).
• Patients who have been administered a cytokine preparation such as G-CSF (granulocyte-colony stimulating factor), erythropoietin, etc. within 14 days of tests for enrollment of the study.
• Patients with obvious infectious diseases (including viral infections).
• Patients with serious complications (liver failure, renal failure, etc.).
• Patients with a complication or previous history of serious heart disease (myocardial infarction, ischemic heart disease, etc.) within the past two years before enrollment, and with cardiac arrhythmia requiring treatment.
• Patien