Phase II Study of Subcutaneous Azacytidine in Patients with Myelodysplastic Syndromes

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Basic Trial Information

PhaseTypeStatusAgeSponsorProtocol IDs
Phase IITreatmentCompletedover 15NCICLB-8921


I.  Determine which myelodysplastic syndromes respond optimally to 
subcutaneous azacytidine (AZA).

II.  Evaluate the toxicity of subcutaneous AZA in patients with certain 
myelodysplastic syndromes.

Entry Criteria

Disease Characteristics:

See General Eligibility Criteria

Patient Characteristics:

See General Eligibility Criteria

General Eligibility Criteria:

Patients over 15 years of age with an 
established diagnosis of myelodysplastic syndrome that is confirmed by 
erythroid, megakaryocytic, and granulocytic maturational abnormalities in 
peripheral blood and bone marrow conforming to one of the following 
categories:  refractory anemia with excess blasts (RAEB), defined as 5-20% 
myeloblasts in bone marrow and less than 5% blasts in peripheral blood; RAEB 
in transformation (RAEB-T), defined as 21-30% myeloblasts in bone marrow, more 
than 5% blasts in peripheral blood, or Auer rods in granulocytic precursors in 
marrow or blood and no more than 30% myeloblasts in marrow; or chronic 
myelomonocytic leukemia (CMML), defined as an absolute monocytosis of more 
than 1 x 10 to the ninth cells/liter with less than 5% blasts in peripheral 
blood and no more than 20% blasts in bone marrow.  (At least 200 cells are 
required for bone marrow differential determinations.)  Patients with more 
than 30% myeloblasts in the bone marrow are ineligible.  No prior cytotoxic 
therapy for this disease is allowed, nor is prior treatment with azacytidine.  
At least 6 months must have elapsed since any prior radiotherapy or 
chemotherapy for cancer, during which time the patient must have been free of 
any evidence of malignancy.  A performance status of 2 or better and a life 
expectancy of at least 2 months are required, as are the following laboratory 
values (obtained within 2 weeks of registration):  total bilirubin no more 
than 1.5 times normal (unless the patient has active hemolysis or the 
elevation is secondary to ineffective erythropoiesis) and SGOT/SGPT no more 
than twice normal; serum creatinine no more than 1.5 times normal; and serum 
CO2 greater than 19 mEq/liter.  The following conditions exclude:  
uncontrolled or severe congestive heart failure; any other serious medical or 
psychiatric illness that would limit survival to less than 2 years or prevent 
informed consent; and pregnancy.  Patients must be able to begin protocol 
therapy within 2 weeks of completing prestudy requirements.

Expected Enrollment

50 patients will be entered.  It is expected that the study will be completed 
in less than 1.5 years.


Nonrandomized study.

Single-Agent Chemotherapy.  Azacytidine, AZA, NSC-102816.

Related Publications

Silverman LR, McKenzie DR, Peterson BL, et al.: Further analysis of trials with azacitidine in patients with myelodysplastic syndrome: studies 8421, 8921, and 9221 by the Cancer and Leukemia Group B. J Clin Oncol 24 (24): 3895-903, 2006.[PUBMED Abstract]

Trial Contact Information

Trial Lead Organizations

Cancer and Leukemia Group B

Lewis Silverman, MD, Protocol chair
Ph: 212-241-5520

Note: The purpose of most clinical trials listed in this database is to test new cancer treatments, or new methods of diagnosing, screening, or preventing cancer. Because all potentially harmful side effects are not known before a trial is conducted, dose and schedule modifications may be required for participants if they develop side effects from the treatment or test. The therapy or test described in this clinical trial is intended for use by clinical oncologists in carefully structured settings, and may not prove to be more effective than standard treatment. A responsible investigator associated with this clinical trial should be consulted before using this protocol.