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This small pilot study is being done to examine the effects of the drug Decitabine. Decitabine is a new formulation of a drug (5-azacytidine). Decitabine was shown to increase hemoglobin F (fetal hemoglobin) levels and therefore, decrease anemia, in patients with sickle cell anemia. Decitabine has been found to be very effective in increasing overall hemoglobin and it is easier to tolerate than similar previous drugs.
This study will enroll 8 non-transfused patients with thalassemia intermedia that are over 18 years old. Most of the patients will be enrolled in Toronto, Canada. If the results from the first 8 patients are positive, the study will be expanded to include patients in the United States.
Patients who participate must be willing to come into the clinical research center two times per week on consecutive days. Each visit would last about 15-20 minutes. The drug, decitabine is given in a subcutaneous shot by a nurse. The total length of this study is 12 weeks.
For more information, please contact Nancy Sweeters, RN, PNP at Children's Hospital & Research Center at Oakland, 510-428-3885, ext. 4151 or ask your clinical care provider.
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