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Phase II Decitabine (DAC) Versus Azacitidine (AZA) in 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.
 
ClinicalTrials.gov Identifier: NCT02269280
Recruitment Status : Active, not recruiting
First Posted : October 21, 2014
Last Update Posted : July 22, 2020
Sponsor:
Collaborator:
National Cancer Institute (NCI)
Information provided by (Responsible Party):
M.D. Anderson Cancer Center

Tracking Information
First Submitted Date  ICMJE October 13, 2014
First Posted Date  ICMJE October 21, 2014
Last Update Posted Date July 22, 2020
Actual Study Start Date  ICMJE October 13, 2014
Estimated Primary Completion Date October 30, 2026   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: October 16, 2014)
Event Free Survival (EFS) [ Time Frame: 56 days ]
Event free survival (EFS) defined as the time from beginning of treatment till an event occurs or last follow-up. For transfusion independent patients, the events includes lack of response, requirement of blood transfusion, progression to advanced stages of disease, transformation into AML, discontinuation of therapy due to side effects, and death. For transfusion dependent patients, the events includes lack of response, progression to advanced stages of disease, transformation into AML, discontinuation of therapy due to side effects, and death.
Original Primary Outcome Measures  ICMJE Same as current
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: October 16, 2014)
Overall improvement rate (OIR) [ Time Frame: 56 days ]
Overall improvement rate (OIR), defined as complete remission (CR), partial remission (PR), marrow CR (mCR), or hematologic improvement (HI), measured using each patient's best response with the 2 different agents. Response assessed using the modified MDS International Working Group 2006 criteria. The best response within the first two cycles will be the OIR for each treatment arm that will be used in the adaptive randomization algorithm.
Original Secondary Outcome Measures  ICMJE Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Phase II Decitabine (DAC) Versus Azacitidine (AZA) in Myelodysplastic Syndrome (MDS)
Official Title  ICMJE Phase II Randomized Study of Lower Doses of Decitabine (DAC; 20 mg/m2 IV Daily for 3 Days Every Month) Versus Azacitidine (AZA; 75 mg/m2 SC/IV Daily for 3 Days Every Month) Versus Azacitidine (AZA; 75 mg/m2 SC/IV Daily for 5 Days Every Month) in MDS Patients With Low and Intermediate-1 Risk Disease Transfusion-Dependent Versus Best Supportive Care (BSC) in MDS Patients With Low and Intermediate-1 Risk Disease Transfusion-Independent
Brief Summary

The goal of this clinical research study is to compare how 2 different drugs, decitabine and azacitidine, when given on a shorter than standard dosing schedule, may help to control MDS. The safety of each study drug given on these schedules will also be studied.

This is an investigational study. Decitabine and azacitidine are both FDA approved and commercially available for use in patients with MDS. Giving these drugs on a different schedule than is standard is considered investigational.

The study doctor can tell you how the study drugs are designed to work.

Up to 240 participants will be enrolled in this multicenter study. Up to 157 will take part at MD Anderson.

Detailed Description

Study Groups and Study Drug Administration:

Each cycle is approximately 28 days.

If you are found to be eligible to take part in this study, you will be randomly assigned (as in the roll of dice) to 1 of 4 groups:

  • If you are in Group 1, you will receive decitabine by vein over about 1 hour on Days 1-3 of every cycle.
  • If you are in Group 2, you will receive azacitidine either as an injection under your skin or by vein on Days 1-3 of every cycle.
  • If you are in Group 3, you will receive azacitidine either as an injection under your skin or by vein on Days 1-5 of every cycle.
  • If you are in Group 4, you will receive the standard of care. The study doctor can explain the treatment you will receive and the risks involved.

Transfusion-dependent participants will be randomly assigned to 1 of 3 groups

This is done because no one knows if one study group is better, the same, or worse than the other group. If you are among the first 20 participants, you will have an equal chance of being in any of the groups. If you enroll after that, you will have a higher chance of being assigned to the group that has had better results.

However, once you are assigned to a group, you will not be allowed to change groups.

You may be given other drugs to help prevent side effects. The study staff will tell you about these drugs, how they will be given, and the possible risks.

Study Visits:

One (1) time each month, blood (about 2 tablespoons) will be drawn for routine tests.

At the end of Cycle 2, then every 3 cycles for the first year, then every 6 cycles, you will have a bone marrow biopsy and/or aspirate to check the status of the disease and for cytogenetic testing.

After Cycle 1, if the study doctor decides it is acceptable, you may be allowed to receive treatment from your local cancer doctor. However, you must return to Houston at least every 3 cycles for study visits. How often these visits will occur will depend on what the doctor thinks is in your best interest.

Length of Study:

You may continue taking the study drug or standard therapy for as long as the doctor thinks it is in your best interest. You will no longer be able to take the study drug or standard therapy if the disease gets worse, if intolerable side effects occur, or if you are unable to follow study directions.

Follow-Up:

When you are off-treatment, every 6 -12 months for up to 5 years, you will be called by a member of the study staff. You will be asked about any side effects you may be having. The phone calls will take about 5-10 minutes.

Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 2
Study Design  ICMJE Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE Leukemia
Intervention  ICMJE
  • Drug: Azacitidine (AZA) Days 1 - 3
    Azacitidine 75 mg/m2 by vein or subcutaneously daily for 3 days (days 1-3) approximately every 28 days.
    Other Names:
    • 5-Azacytidine
    • 5-AZA
    • Vidaza
    • 5-AZC
    • AZA-CR
    • Ladakamycin
    • NSC-102816
    • Azacytidine
  • Drug: Decitabine (DAC)
    Decitabine 20 mg/m2 by vein for 3 days (days 1-3) approximately every 28 days.
    Other Name: Dacogen
  • Other: Best Supportive Care (BSC)
    Participants receive standard of care as chosen by study doctor. Best supportive care for transfusion-independent participants only.
  • Drug: Azacitidine (AZA) Days 1 - 5
    Azacitidine 75 mg/m2 by vein or subcutaneously daily for 5 days (days 1-5) approximately every 28 days.
    Other Names:
    • 5-Azacytidine
    • 5-AZA
    • Vidaza
    • 5-AZC
    • AZA-CR
    • Ladakamycin
    • NSC-102816
    • Azacytidine
Study Arms  ICMJE
  • Experimental: Azacitidine (AZA) - Days 1 - 3
    Azacitidine (AZA) Azacitidine 75 mg/m2 by vein or subcutaneously daily for 3 days (days 1-3) approximately every 28 days.
    Intervention: Drug: Azacitidine (AZA) Days 1 - 3
  • Experimental: Azacitidine (AZA) - Days 1 - 5
    Azacitidine (AZA) 75 mg/m2 by vein or subcutaneously daily for 5 days (days 1-5) approximately every 28 days.
    Intervention: Drug: Azacitidine (AZA) Days 1 - 5
  • Experimental: Decitabine (DAC)
    Decitabine 20 mg/m2 by vein for 3 days (days 1-3) approximately every 28 days.
    Intervention: Drug: Decitabine (DAC)
  • Best Supportive Care (BSC)
    Participants receive standard of care as chosen by study doctor. Best supportive care for transfusion-independent participants only.
    Intervention: Other: Best Supportive Care (BSC)
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 Active, not recruiting
Actual Enrollment  ICMJE
 (submitted: December 23, 2019)
268
Original Estimated Enrollment  ICMJE
 (submitted: October 16, 2014)
240
Estimated Study Completion Date  ICMJE October 30, 2026
Estimated Primary Completion Date October 30, 2026   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

Inclusion Criteria:

  1. Sign an IRB-approved informed consent document.
  2. Age >/= 18 years.
  3. IPSS low- or intermediate-1-risk MDS, including CMML-1
  4. ECOG performance status of </= 3 at study entry.
  5. Organ function defined as: Serum creatinine </= 2 mg/dL; Total bilirubin </= 2 x ULN; ALT (SGPT) </= 2 x ULN; AST (SGOT) </= 2 x ULN
  6. Women of childbearing potential must have a negative serum or urine pregnancy test within 7 days and will also need to use contraceptives. Men must agree not to father a child and agree to use a condom if his partner is of child bearing potential.

Exclusion Criteria:

  1. Breast feeding females
  2. Prior therapy with decitabine or azacitidine
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 NCT02269280
Other Study ID Numbers  ICMJE 2014-0112
NCI-2014-02339 ( Registry Identifier: CTRP (Clinical Trial Reporting Program) )
2014-0112 ( Other Identifier: M D Anderson Cancer Center )
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE Not Provided
Responsible Party M.D. Anderson Cancer Center
Study Sponsor  ICMJE M.D. Anderson Cancer Center
Collaborators  ICMJE National Cancer Institute (NCI)
Investigators  ICMJE
Principal Investigator: Guillermo Garcia-Manero M.D. Anderson Cancer Center
PRS Account M.D. Anderson Cancer Center
Verification Date December 2019

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