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Study of Azacytidine Followed by GM-CSF in Patients With Myelodysplastic Syndrome (MDS)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT01542684
Recruitment Status : Terminated (Slow Accrual)
First Posted : March 2, 2012
Results First Posted : April 30, 2014
Last Update Posted : April 30, 2014
Information provided by (Responsible Party):
M.D. Anderson Cancer Center

Brief Summary:

The goal of this clinical research study is to learn if the combination of azacitidine and GM-CSF can help to control MDS. The safety of these drugs will also be studied.

Azacitidine is designed to block certain proteins that stop the function of tumor-fighting genes. By blocking the "bad" proteins, the tumor-fighting genes may be able to work better.

Granulocyte-macrophage colony-stimulating factor (GM-CSF) is designed to help produce white blood cells. This may help to fight infections.

Condition or disease Intervention/treatment Phase
Leukemia Drug: Azacytidine Drug: GM-CSF Phase 2

Detailed Description:

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 azacitidine.

On Days 5-7 of every cycle, you will receive GM-CSF by vein over about 15 minutes or by injection.

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.

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 8 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Phase II Study of Azacytidine Followed by GM-CSF in Patients With Low- or Intermediate-1- Risk Myelodysplastic Syndrome (MDS)
Study Start Date : March 2012
Actual Primary Completion Date : April 2013
Actual Study Completion Date : April 2013

Resource links provided by the National Library of Medicine

Drug Information available for: Azacitidine

Arm Intervention/treatment
Experimental: Azacytidine + GM-CSF

Azacytidine administered intravenously (IV) or subcutaneously (SQ) at starting dose of 40 mg/m^2, daily for 4 days.

GM-CSF administered IV or subcutaneously at 250 mcg/m^2 one day (the next day) after completion of azacytidine treatment, for 3 consecutive days.

Each treatment cycle will last at least 4 weeks

Drug: Azacytidine
Starting dose: 40 mg/m^2 intravenously (IV) or subcutaneously (SQ) daily for 4 days.
Other Names:
  • 5-Azacytidine
  • 5-AZA
  • Vidaza
  • 5-AZC
  • AZA-CR
  • Ladakamycin
  • NSC-102816

Drug: GM-CSF
250 mcg/m^2 IV or SQ one day (the next day) after completion of azacytidine treatment, for 3 consecutive days.
Other Names:
  • Saragramostim
  • Leukine

Primary Outcome Measures :
  1. Overall Response Rate (ORR) [ Time Frame: Baseline up to 2 treatment cycles (8 weeks) ]
    ORR is percentage total participants with overall response (Complete Response (CR) or Partial Response (PR)) within two treatment cycles. Response based on modified International Working Group (IWG) criteria: Complete response - Bone marrow: 5% myeloblasts with normal maturation of all cell lines, Persistent dysplasia noted, Peripheral blood Hgb 11 g/dL, Platelets 100x109/L, Neutrophils 1.0x109/L, Blasts 0%. Partial response: All CR criteria if abnormal before treatment except: Bone marrow blasts decreased by 50% over pretreatment but still > 5% , Cellularity and morphology not relevant; Stable disease - Failure to achieve at least PR, but no evidence of progression for > 8 weeks; No Response or Failure - Death during treatment or disease progression characterized by worsening of cytopenias, increase in percentage of bone marrow blasts, or progression to a more advanced MDS French-American-British (FAB) classification subtype than pretreatme

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.

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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT01542684

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United States, Texas
UT MD Anderson Cancer Center
Houston, Texas, United States, 77030
Sponsors and Collaborators
M.D. Anderson Cancer Center
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Principal Investigator: Zeev Estrov, MD UT MD Anderson Cancer Center

Additional Information:
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Responsible Party: M.D. Anderson Cancer Center Identifier: NCT01542684     History of Changes
Other Study ID Numbers: 2011-1123
First Posted: March 2, 2012    Key Record Dates
Results First Posted: April 30, 2014
Last Update Posted: April 30, 2014
Last Verified: March 2014
Keywords provided by M.D. Anderson Cancer Center:
Myelodysplastic Syndrome
Additional relevant MeSH terms:
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Myelodysplastic Syndromes
Bone Marrow Diseases
Hematologic Diseases
Precancerous Conditions
Antimetabolites, Antineoplastic
Molecular Mechanisms of Pharmacological Action
Antineoplastic Agents
Enzyme Inhibitors
Immunologic Factors
Physiological Effects of Drugs