Phase II Study of Azacytidine Followed by GM-CSF in Patients With Low- or Intermediate-1- Risk Myelodysplastic Syndrome (MDS)
Study Drug Administration:
If you are found to be eligible to take part in this study, on Days 1-4 of every cycle, you
will receive azacitidine by vein over 15-30 minutes.
You may receive drugs to help prevent nausea and vomiting before you receive your dose of
On Days 5-7 of every cycle, you will receive GM-CSF by vein over about 15 minutes or by
Each study cycle will be about 4-6 weeks, depending on the study doctor's decision.
One (1) time each week during every cycle, blood (about 2-3 teaspoons) will be drawn for
At any time, if your doctor thinks it is needed, you will have a bone marrow aspirate to
check the status of the disease.
Length of Study:
You may continue taking the study drugs for as long as the doctor thinks it is in your best
interest. You will no longer be able to take the study drugs if the disease gets worse, if
intolerable side effects occur, or if you are unable to follow study directions.
Your follow-up visits will be per standard of care for the disease.
This is an investigational study. Both azacitidine and GM-CSF are FDA approved and
commercially available for the treatment of MDS. The study drug combination to treat MDS is
Up to 40 patients will take part in this study. All will be enrolled at MD Anderson.
Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
Overall Response Rate
Overall response rate defined as number participants with overall response (OR), complete response (CR) + partial response (PR) within two treatment cycles. Response based on the modified International Working Group (IWG) criteria.
Zeev Estrov, MD
UT MD Anderson Cancer Center
United States: Institutional Review Board
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