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Phase II Study of Azacitidine in Myelofibrosis

Phase 2
18 Years
Not Enrolling

Thank you

Trial Information

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.

Inclusion Criteria:

- Diagnosis of MF requiring therapy, including those previously treated and relapsed or
refractory, or if newly diagnosed, with intermediate or high risk according to Lille
scoring system (adverse prognostic factors are: Hemoglobin (Hb) < 10 g/dl, White
Blood Cell (WBC) < 4 or > 30 x 10*9/L; risk group: 0 = low, 1 = intermediate, 2 =

- Performance 0-2 Eastern Cooperative Oncology Group (ECOG).

- Signed informed consent.

- Patients must have been off chemotherapy for 2 weeks prior to entering this study and
have recovered from the toxic effects (grade 0-1) of that therapy. Patients are
allowed to be on anagrelide and hydroxyurea to control high platelet and WBC counts
for their safety.

- Serum bilirubin levels laboratory Upper Limit of of Normal(ULN). Higher levels are acceptable if these can
be attributed to active hemolysis or ineffective erythropoiesis.

- Serum glutamic-oxaloacetic transaminase (SGOT) (aspartate aminotransferase [AST]) or
serum glutamic-pyruvic transaminase (SGPT) (alanine aminotransferase [ALT]) levels

- Serum creatinine levels treating physicians.

- Women of childbearing potential must have a negative serum pregnancy test prior to
azacitidine treatment and should be advised to avoid becoming pregnant. Men must be
advised to not father a child while receiving treatment with azacitidine. Both women
of childbearing potential and men must practice effective methods of contraception
(those generally accepted as standard of care measures).

- Age >/= 18.

Exclusion Criteria:

- Nursing and pregnant females. Should a woman become pregnant or suspect she is
pregnant while participating in this study, she should inform her treating physician

- Uncontrolled intercurrent illness including, but not limited to, uncontrolled active
infection, symptomatic congestive heart failure, unstable angina pectoris, or
psychiatric illness/social situations that would limit compliance with study

- Known or suspected hypersensitivity to azacitidine or mannitol.

Type of Study:


Study Design:

Allocation: Non-Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Outcome Measure:

Number of Participants With Objective Clinical Response

Outcome Description:

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.

Outcome Time Frame:

Every 2 courses of 4 week therapy = each 8 weeks

Safety Issue:


Principal Investigator

Srdan Verstovsek, M.D.

Investigator Role:

Principal Investigator

Investigator Affiliation:

M.D. Anderson Cancer Center


United States: Institutional Review Board

Study ID:




Start Date:

June 2005

Completion Date:

April 2008

Related Keywords:

  • Myelofibrosis
  • Myelofibrosis
  • MF
  • Leukemia
  • Azacitidine
  • Vidaza
  • Primary Myelofibrosis



UT MD Anderson Cancer Center Houston, Texas  77030