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Phase II Study of Azacytidine Followed by GM-CSF in Patients With Low- or Intermediate-1- Risk Myelodysplastic Syndrome (MDS)

Phase 2
18 Years
Not Enrolling

Thank you

Trial Information

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.

Study Visits:

One (1) time each week during every cycle, blood (about 2-3 teaspoons) will be drawn for
routine tests.

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
considered investigational.

Up to 40 patients will take part in this study. All will be enrolled at MD Anderson.

Inclusion Criteria:

1. Patients with low- or intermediate-1-risk MDS according to the International
Prognostic Scoring System (IPSS) classification

2. Signed informed consent indicating that patients are aware of the investigational
nature of this study in keeping with the policies of UT MD Anderson Cancer Center.

3. Age >/= 18 years old.

4. Prior therapy with growth factor support, lenalidomide, or other investigational
agents is allowed.

5. Previously untreated patients are eligible for this study.

Exclusion Criteria:

1. Any previous adverse reaction (>/= Common Terminology Criteria for Adverse Events
(CTCAE) grade 2) to GM-CSF.

2. Prior treatment with azacytidine or decitabine.

3. Unresolved diarrhea >/= CTCAE grade 2.

Type of Study:


Study Design:

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

Outcome Measure:

Overall Response Rate

Outcome Description:

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.

Outcome Time Frame:

8 weeks

Safety Issue:


Principal Investigator

Zeev Estrov, MD

Investigator Role:

Principal Investigator

Investigator Affiliation:

UT MD Anderson Cancer Center


United States: Institutional Review Board

Study ID:




Start Date:

March 2012

Completion Date:

April 2013

Related Keywords:

  • Leukemia
  • Leukemia
  • Myelodysplastic Syndrome
  • MDS
  • Azacytidine
  • 5-Azacytidine
  • 5-AZA
  • Vidaza
  • 5-AZC
  • Aza-CR
  • Ladakamycin
  • NSC-102816
  • GM-CSF
  • Sargramostim
  • Leukine
  • Leukemia
  • Myelodysplastic Syndromes
  • Preleukemia



UT MD Anderson Cancer Center Houston, Texas  77030