Phase II Study of Azacitidine in Myelofibrosis
Azacitidine is a drug that is designed to block certain genes in cancer cells whose job is
to stop the function of the tumor-fighting genes. By blocking the "bad" genes, the
tumor-fighting genes may be able to work better.
If you are found to be eligible to take part in this study, you will be able to begin
treatment with azacitidine. You will receive azacitidine as an injection under the skin
once a day for 7 days in a row. This will be repeated every 4 weeks (4 weeks equals 1
cycle). The first cycle of azacitidine will be given at M. D. Anderson, in an outpatient
setting. Later cycles of treatment courses may be given at M. D. Anderson or by a cancer
doctor in your community.
You may receive up to 12 cycles of treatment if you are responding well to treatment. You
will be taken off study if your disease gets worse or intolerable side effects occur. Once
you go off study, you will receive follow-up as is standard of care for your disease.
This is an investigational study. Azacitidine is FDA approved for the treatment of
myelodysplastic syndrome. Its use in this study is experimental. A total of up to 34
patients will take part in this study. All will be enrolled at M. D. Anderson.
Allocation: Non-Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
Number of Participants With Objective Clinical Response
Objective Clinical Response includes Participants with Complete Response, Partial Response or Hematologic Improvement and No Response. Bone marrow aspiration and biopsy with cytogenetics every 2 to 4 courses.
Every 2 courses of 4 week therapy = each 8 weeks
Srdan Verstovsek, M.D.
M.D. Anderson Cancer Center
United States: Institutional Review Board
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