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

This study has been terminated.
(Slow Accrual)
Sponsor:
Information provided by (Responsible Party):
M.D. Anderson Cancer Center
ClinicalTrials.gov Identifier:
NCT01542684
First received: February 27, 2012
Last updated: March 28, 2014
Last verified: March 2014
  Purpose

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 Intervention Phase
Leukemia
Drug: Azacytidine
Drug: GM-CSF
Phase 2

Study Type: Interventional
Study Design: Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: 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)

Resource links provided by NLM:


Further study details as provided by M.D. Anderson Cancer Center:

Primary Outcome Measures:
  • Overall Response Rate (ORR) [ Time Frame: Baseline up to 2 treatment cycles (8 weeks) ] [ Designated as safety issue: No ]
    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


Enrollment: 8
Study Start Date: March 2012
Study Completion Date: April 2013
Primary Completion Date: April 2013 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
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

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.

  Eligibility

Ages Eligible for Study:   18 Years and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

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.
  Contacts and Locations
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, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01542684

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

Additional Information:
No publications provided

Responsible Party: M.D. Anderson Cancer Center
ClinicalTrials.gov Identifier: NCT01542684     History of Changes
Other Study ID Numbers: 2011-1123
Study First Received: February 27, 2012
Results First Received: March 28, 2014
Last Updated: March 28, 2014
Health Authority: United States: Institutional Review Board

Keywords provided by M.D. Anderson Cancer Center:
Leukemia
Myelodysplastic Syndrome
MDS
Azacytidine
5-Azacytidine
5-AZA
Vidaza
5-AZC
Aza-CR
Ladakamycin
NSC-102816
GM-CSF
Sargramostim
Leukine

Additional relevant MeSH terms:
Leukemia
Myelodysplastic Syndromes
Preleukemia
Syndrome
Bone Marrow Diseases
Disease
Hematologic Diseases
Neoplasms
Neoplasms by Histologic Type
Pathologic Processes
Precancerous Conditions
Azacitidine
Antimetabolites
Antimetabolites, Antineoplastic
Antineoplastic Agents
Enzyme Inhibitors
Molecular Mechanisms of Pharmacological Action
Pharmacologic Actions
Therapeutic Uses

ClinicalTrials.gov processed this record on October 20, 2014