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Phase II Randomized Study of Three Different Schedules of Low-Dose Decitabine (5-AZA-2'-Deoxycytidine) in Myelodysplastic Syndrome (MDS)

Phase 2
Not Enrolling
Myelodysplastic Syndrome, Chronic Myelomonocytic Leukemia

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Trial Information

Phase II Randomized Study of Three Different Schedules of Low-Dose Decitabine (5-AZA-2'-Deoxycytidine) in Myelodysplastic Syndrome (MDS)

Treatment: Methylation is a change that occurs to Deoxyribonucleic acid (DNA) that has an
effect on gene usage in human cells. Abnormal methylation is very common in leukemias.
Decitabine is a new drug that blocks DNA methylation.

Before treatment starts, a physical exam, blood tests (between 4-6 tablespoons), and a bone
marrow study will be done. To collect a bone marrow sample, an area of the hip or chest
bone is numbed with anesthetic and a small amount of bone marrow is withdrawn through a
large needle. Women able to have children must have a negative blood or urine pregnancy

When this study began, participants were randomly assigned (as in the toss of a coin) to one
of 3 treatment groups. The assignment to one of the 3 schedules was adjusted according to
how well patients respond to treatment. About 17 patients were assigned to each group for
the first 50 patients.

Participants in the first group received decitabine intravenously (IV--through a needle in
their vein) over one hour, once a day, for 10 days. Treatment was given every 4 to 8 weeks
depending on how well their blood counts recovered. Participants in the second group
received decitabine as an IV infusion over one hour, once a day, for 5 days. Treatment was
given every 4 to 8 weeks. Participants who received decitabine by vein got the same total
dose per course. Participants in the third group received decitabine by subcutaneous (SQ)
injections (injections given under the skin) twice a day for 5 days. As in the first and
second group, treatment was given every 4 to 8 weeks.

After 65 patients were enrolled on this study, it was decided that the 5-day IV schedule was
the best of the 3 schedules. The study will now continue with all new patients receiving
the 5 -day IV decitabine treatment. If you are now enrolling on the study, you will be
placed in this treatment group, instead of being randomly assigned to a treatment group.

Participants who are already on study and who are receiving the 5-day SQ schedule or the
10-day IV schedule, will be given the option to change to the 5-day IV schedule at the start
of their next course of study drug treatment, since this is considered the new "standard"
schedule on this particular study.

If you choose to take part in this study and begin receiving the study treatment described
above, your response to treatment will be checked after completing 8 weeks of therapy. If
the response to treatment is good, treatment with decitabine will continue. Decitabine
treatment may be continued for up to 24 courses, or as long as it is judged best to control
the leukemia.

During this study, you will need to visit your doctor for a physical exam and vital signs.
The frequency of doctor visits will vary depending on your physical condition, but will be
required at least once a month.

Blood tests (about 2 teaspoons) will be done about every week during the first 6-8 weeks of
treatment, then every 1 to 2 weeks for the length of the study. The blood samples will be
used for routine lab tests. Periodic bone marrow samples will also be taken to check cells
related to the disease before, during, and after completion of this study.

Patients will be taken off study if the disease gets worse or intolerable side effects

This is an investigational study. Decitabine is not yet Food and Drug Administration (FDA)
approved.Up to 133 participants will be treated in this study. All will be enrolled at M.
D. Anderson.

Inclusion Criteria:

1. MDS and 5% or more marrow blasts, or IPSS risk intermediate 1-2 or high risk; or
chronic myelomonocytic leukemia

2. Performance status 0-2 (Eastern Cooperative Oncology Group (ECOG) scale); adequate
hepatic (bilirubin < 2 mg/dl) and renal functions (creatinine <2mg/dl); New York
Heart Association (NYHA) cardiac status III-IV excluded.

3. Signed informed consent

4. No prior intensive combination chemotherapy or high-dose ara-C (>/= 1g/m2 per dose).
Prior biologic therapies, targeted therapies and single agent chemotherapy allowed.

5. Patients must have been off chemotherapy for 2 weeks prior to entering this study and
recovered from the toxic effects of that therapy, unless there is evidence of rapidly
progressive disease. Use of Hydroxyurea for patients with rapidly proliferative
disease is allowed for the first two weeks on therapy.

Exclusion Criteria:

1. Nursing and pregnant females are excluded. Patients of childbearing potential should
practice effective methods of contraception. Should a woman become pregnant or
suspect she is pregnant while participating in this study, she should inform her
treating physician immediately.

2. Patients with active and uncontrolled infections

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

Type of Study:


Study Design:

Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Open Label, Primary Purpose: Treatment

Outcome Measure:

Participant Responses

Outcome Description:

Objective responses by International Working Group criteria: 'Complete Response' (CR) defined as Normalization of the peripheral blood and bone marrow with <5% bone marrow blasts, a peripheral blood granulocyte count > (1.0 x 109/ L, and a platelet count > 100 x 109/L); 'Other Response' including Partial Remission (PR) defined as above, except for the presence of 6-15% marrow blasts, or 50% reduction if <15% at start of treatment combined with participants who meet all criteria for CR except for platelet recovery to >100 x 109/L; and 'No Response'.

Outcome Time Frame:

Response to treatment after 8 weeks of therapy

Safety Issue:


Principal Investigator

Hagop M Kantarjian, MD

Investigator Role:

Principal Investigator

Investigator Affiliation:

M.D. Anderson Cancer Center


United States: Food and Drug Administration

Study ID:




Start Date:

October 2003

Completion Date:

May 2009

Related Keywords:

  • Myelodysplastic Syndrome
  • Chronic Myelomonocytic Leukemia
  • Myelodysplastic Syndrome
  • Chronic Myelomonocytic Leukemia
  • MDS
  • Decitabine
  • Dacogen
  • Methylation
  • Leukemia
  • Leukemia, Myelomonocytic, Chronic
  • Myelodysplastic Syndromes
  • Preleukemia
  • Leukemia, Myelomonocytic, Acute



University of Texas - MD Anderson Cancer Center Houston, Texas  77030-4009