Phase II Decitabine (DAC) Versus Azacitidine (AZA) in Myelodysplastic Syndrome (MDS)

NCT ID: NCT02269280

Last Updated: 2025-08-07

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE2
• Total Enrollment: 185 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2014-10-13
• Study Completion Date: 2024-07-25

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.

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.

Conditions

Leukemia

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

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.

Group Type: EXPERIMENTAL

Azacitidine (AZA) Days 1 - 3

Intervention Type: DRUG

Azacitidine 75 mg/m2 by vein or subcutaneously daily for 3 days (days 1-3) approximately every 28 days.

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.

Group Type: EXPERIMENTAL

Azacitidine (AZA) Days 1 - 5

Intervention Type: DRUG

Azacitidine 75 mg/m2 by vein or subcutaneously daily for 5 days (days 1-5) approximately every 28 days.

Decitabine (DAC)

Decitabine 20 mg/m2 by vein for 3 days (days 1-3) approximately every 28 days.

Group Type: EXPERIMENTAL

Decitabine (DAC)

Intervention Type: DRUG

Decitabine 20 mg/m2 by vein for 3 days (days 1-3) approximately every 28 days.

Best Supportive Care (BSC)

Participants receive standard of care as chosen by study doctor. Best supportive care for transfusion-independent participants only.

Group Type: OTHER

Best Supportive Care (BSC)

Intervention Type: OTHER

Participants receive standard of care as chosen by study doctor. Best supportive care for transfusion-independent participants only.

Interventions

Azacitidine (AZA) Days 1 - 3

Azacitidine 75 mg/m2 by vein or subcutaneously daily for 3 days (days 1-3) approximately every 28 days.

Intervention Type: DRUG

Decitabine (DAC)

Decitabine 20 mg/m2 by vein for 3 days (days 1-3) approximately every 28 days.

Intervention Type: DRUG

Best Supportive Care (BSC)

Participants receive standard of care as chosen by study doctor. Best supportive care for transfusion-independent participants only.

Intervention Type: OTHER

Azacitidine (AZA) Days 1 - 5

Azacitidine 75 mg/m2 by vein or subcutaneously daily for 5 days (days 1-5) approximately every 28 days.

Intervention Type: DRUG

Other Intervention Names

5-Azacytidine; 5-AZA; Vidaza; 5-AZC; AZA-CR; Ladakamycin; NSC-102816; Azacytidine; Dacogen; 5-Azacytidine; 5-AZA; Vidaza; 5-AZC; AZA-CR; Ladakamycin; NSC-102816; Azacytidine

Eligibility Criteria

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

• Minimum Eligible Age: 18 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

National Cancer Institute (NCI)

NIH

Sponsor Role: collaborator

M.D. Anderson Cancer Center

OTHER

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Guillermo Garcia-Manero

Role: PRINCIPAL_INVESTIGATOR

M.D. Anderson Cancer Center

Locations

Moffitt Cancer Center

Tampa, Florida, United States

Johns Hopkins University/Sidney Kimmel Cancer Center

Baltimore, Maryland, United States

Dana-Farber Cancer Institute

Boston, Massachusetts, United States

NYP/Weill Cornell Medical Center

New York, New York, United States

Cleveland Clinic Foundation

Cleveland, Ohio, United States

M D Anderson Cancer Center

Houston, Texas, United States

Countries

United States

Provided Documents

Document Type: Study Protocol and Statistical Analysis Plan

View Document

Related Links

http://www.mdanderson.org

University of Texas MD Anderson Cancer Center Website

Other Identifiers

NCI-2014-02339

Identifier Type: REGISTRY

Identifier Source: secondary_id

2014-0112

Identifier Type: OTHER

Identifier Source: secondary_id

2014-0112

Identifier Type: -

Identifier Source: org_study_id