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

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ClinicalTrials.gov Identifier: NCT01542684
Recruitment Status : Terminated (Slow Accrual)
First Posted : March 2, 2012
Results First Posted : April 30, 2014
Last Update Posted : April 30, 2014
Sponsor:
Information provided by (Responsible Party):
M.D. Anderson Cancer Center

Tracking Information
First Submitted Date  ICMJE February 27, 2012
First Posted Date  ICMJE March 2, 2012
Results First Submitted Date  ICMJE March 28, 2014
Results First Posted Date  ICMJE April 30, 2014
Last Update Posted Date April 30, 2014
Study Start Date  ICMJE March 2012
Actual Primary Completion Date April 2013   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: March 28, 2014)
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
Original Primary Outcome Measures  ICMJE
 (submitted: March 1, 2012)
Overall Response Rate [ Time Frame: 8 weeks ]
Overall response rate defined as overall response (OR), complete response (CR) + partial response (PR) within two treatment cycles. Overall response rate based on the modified International Working Group (IWG) criteria.
Change History Complete list of historical versions of study NCT01542684 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures  ICMJE Not Provided
Original Secondary Outcome Measures  ICMJE Not Provided
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Study of Azacytidine Followed by GM-CSF in Patients With Myelodysplastic Syndrome (MDS)
Official Title  ICMJE Phase II Study of Azacytidine Followed by GM-CSF in Patients With Low- or Intermediate-1- Risk Myelodysplastic Syndrome (MDS)
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.

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.

Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 2
Study Design  ICMJE Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE Leukemia
Intervention  ICMJE
  • 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
Study Arms  ICMJE 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

Interventions:
  • Drug: Azacytidine
  • Drug: GM-CSF
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Terminated
Actual Enrollment  ICMJE
 (submitted: April 23, 2013)
8
Original Estimated Enrollment  ICMJE
 (submitted: March 1, 2012)
40
Actual Study Completion Date  ICMJE April 2013
Actual Primary Completion Date April 2013   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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.
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 18 Years and older   (Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT01542684
Other Study ID Numbers  ICMJE 2011-1123
Has Data Monitoring Committee No
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE Not Provided
Responsible Party M.D. Anderson Cancer Center
Study Sponsor  ICMJE M.D. Anderson Cancer Center
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Principal Investigator: Zeev Estrov, MD UT MD Anderson Cancer Center
PRS Account M.D. Anderson Cancer Center
Verification Date March 2014

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP