LeukemiaPhase 2NCT02269280
Phase II Decitabine (DAC) Versus Azacitidine (AZA) in Myelodysplastic Syndrome (MDS)
A Phase 2 study testing Azacitidine (AZA) Days 1 - 3, Decitabine (DAC) and Azacitidine (AZA) Days 1 - 5 in people with Leukemia, run by M.D. Anderson Cancer Center at 6 sites in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Leukemia
- What is being tested?
- Azacitidine (AZA) Days 1 - 3, Decitabine (DAC) and Azacitidine (AZA) Days 1 - 5
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 185 participants
- Where
- 6 sitesUnited States
- Study dates
- 13 Oct 2014to 25 Jul 2024
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Sign an IRB-approved informed consent document.
- Age >/= 18 years.
- IPSS low- or intermediate-1-risk MDS, including CMML-1
- ECOG performance status of </= 3 at study entry.
- Organ function defined as: Serum creatinine </= 2 mg/dL; Total bilirubin </= 2 x ULN; ALT (SGPT) </= 2 x ULN; AST (SGOT) </= 2 x ULN
- 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.
Who cannot join
- Breast feeding females
- Prior therapy with decitabine or azacitidine
Trial locations and contacts
6 sites in 1 country.
6 sites in 1 country
United States6 sites
Johns Hopkins University/Sidney Kimmel Cancer Center
Baltimore, Maryland, 21287
Dana-Farber Cancer Institute
Boston, Massachusetts, 02215
Cleveland Clinic Foundation
Cleveland, Ohio, 44195
M D Anderson Cancer Center
Houston, Texas, 77030
NYP/Weill Cornell Medical Center
New York, New York, 10065
Moffitt Cancer Center
Tampa, Florida, 33612
About this study
Official title: 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
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.
Read the 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
- Being tested
- Azacitidine (AZA) Days 1 - 3, Decitabine (DAC), Best Supportive Care (BSC), Azacitidine (AZA) Days 1 - 5
- Keywords
- Leukemia, Myelodysplastic syndrome, MDS, Low or intermediate-1 risk, Transfusion-dependent, Transfusion-independent, Best supportive care, BSC, Azacitidine, 5-Azacytidine, 5-AZA, Vidaza, 5-AZC, AZA-CR, Ladakamycin, NSC-102816, Azacytidine, Decitabine, Dacogen
- Medical subject headings
- Leukemia, Myelodysplastic Syndromes
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Azacitidine (AZA) Days 1 - 3Drug
Also called 5-Azacytidine, 5-AZA, Vidaza, 5-AZC
Azacitidine 75 mg/m2 by vein or subcutaneously daily for 3 days (days 1-3) approximately every 28 days.
Decitabine (DAC)Drug
Also called Dacogen
Decitabine 20 mg/m2 by vein for 3 days (days 1-3) approximately every 28 days.
Best Supportive Care (BSC)Other
Participants receive standard of care as chosen by study doctor. Best supportive care for transfusion-independent participants only.
Azacitidine (AZA) Days 1 - 5Drug
Also called 5-Azacytidine, 5-AZA, Vidaza, 5-AZC
Azacitidine 75 mg/m2 by vein or subcutaneously daily for 5 days (days 1-5) approximately every 28 days.
| Study group | Role | What they receive |
|---|---|---|
| Azacitidine (AZA) - Days 1 - 3 | Experimental | Azacitidine (AZA) Azacitidine 75 mg/m2 by vein or subcutaneously daily for 3 days (days 1-3) approximately every 28 days. Receives: Drug: Azacitidine (AZA) Days 1 - 3 |
| Azacitidine (AZA) - Days 1 - 5 | Experimental | Azacitidine (AZA) 75 mg/m2 by vein or subcutaneously daily for 5 days (days 1-5) approximately every 28 days. Receives: Drug: Azacitidine (AZA) Days 1 - 5 |
| Decitabine (DAC) | Experimental | Decitabine 20 mg/m2 by vein for 3 days (days 1-3) approximately every 28 days. Receives: Drug: Decitabine (DAC) |
| Best Supportive Care (BSC) | Other | Participants receive standard of care as chosen by study doctor. Best supportive care for transfusion-independent participants only. Receives: Other: Best Supportive Care (BSC) |
What is being measured
The primary outcome is the main question the study is designed to answer.
Event Free Survival (EFS)
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.
Measured: Up to 8 years, 9 months and 12 days
Secondary outcomes (1)
Number of Participants With Overall Improvement
Overall improvement, 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.
Measured: Up to 8 years, 9 months and 12 days
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted21 Oct 2014 (estimated)
- Study start13 Oct 2014
- Primary completion25 Jul 2024
- Study completion25 Jul 2024
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for LeukemiaStudies of the same condition, open and under way.
- More trials run by M.D. Anderson Cancer CenterThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 185 participants (actual)
Sponsor and investigators
- Lead sponsor
- M.D. Anderson Cancer Center
- Sponsor type
- University, hospital or other
- Collaborators
- National Cancer Institute (NCI)
- Responsible party
- Sponsor
- Investigators
- Guillermo Garcia-Manero, Principal investigator, M.D. Anderson Cancer Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02269280
- Sponsor's protocol ID
- 2014-0112
- Other IDs
- NCI-2014-02339, 2014-0112
- Status last verified
- Jul 2025
- First submitted
- 13 Oct 2014
- Last updated
- 7 Aug 2025
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 7 Aug 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- 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
- ClinicalTrials.gov ID
- NCT02269280
- Acronym / sponsor ID
- 2014-0112
- Conditions
- Leukemia
- Keywords
- Leukemia, Myelodysplastic syndrome, MDS, Low or intermediate-1 risk, Transfusion-dependent, Transfusion-independent, Best supportive care, BSC
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 185 participants (actual)
- Lead sponsor
- M.D. Anderson Cancer CenterUniversity, hospital or other
- Collaborators
- National Cancer Institute (NCI)
- Study start
- 13 Oct 2014 (actual)
- Primary completion
- 25 Jul 2024 (actual)
- Study completion
- 25 Jul 2024 (actual)
- First posted
- 21 Oct 2014 (estimated)
- Results
- Posted 7 Aug 2025
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 4
| Arm | Type | Receives |
|---|---|---|
| Azacitidine (AZA) - Days 1 - 3 | Experimental | Drug: Azacitidine (AZA) Days 1 - 3Azacitidine (AZA) Azacitidine 75 mg/m2 by vein or subcutaneously daily for 3 days (days 1-3) approximately every 28 days. |
| Azacitidine (AZA) - Days 1 - 5 | Experimental | Drug: Azacitidine (AZA) Days 1 - 5Azacitidine (AZA) 75 mg/m2 by vein or subcutaneously daily for 5 days (days 1-5) approximately every 28 days. |
| Decitabine (DAC) | Experimental | Drug: Decitabine (DAC)Decitabine 20 mg/m2 by vein for 3 days (days 1-3) approximately every 28 days. |
| Best Supportive Care (BSC) | Other | Other: Best Supportive Care (BSC)Participants receive standard of care as chosen by study doctor. Best supportive care for transfusion-independent participants only. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (6 listed)
- Sign an IRB-approved informed consent document.
- Age >/= 18 years.
- IPSS low- or intermediate-1-risk MDS, including CMML-1
- ECOG performance status of </= 3 at study entry.
- Organ function defined as: Serum creatinine </= 2 mg/dL; Total bilirubin </= 2 x ULN; ALT (SGPT) </= 2 x ULN; AST (SGOT) </= 2 x ULN
Key exclusion criteria (2 listed)
- Breast feeding females
- Prior therapy with decitabine or azacitidine
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Azacitidine (AZA) Days 1 - 3Also: 5-Azacytidine, 5-AZA, Vidaza, 5-AZCAzacitidine 75 mg/m2 by vein or subcutaneously daily for 3 days (days 1-3) approximately every 28 days. | Drug | Azacitidine (AZA) - Days 1 - 3 |
| Decitabine (DAC)Also: DacogenDecitabine 20 mg/m2 by vein for 3 days (days 1-3) approximately every 28 days. | 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. | Other | Best Supportive Care (BSC) |
| Azacitidine (AZA) Days 1 - 5Also: 5-Azacytidine, 5-AZA, Vidaza, 5-AZCAzacitidine 75 mg/m2 by vein or subcutaneously daily for 5 days (days 1-5) approximately every 28 days. | Drug | Azacitidine (AZA) - Days 1 - 5 |
Primary outcome
Event Free Survival (EFS)
Time frame: Up to 8 years, 9 months and 12 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.
1 secondary outcome
Number of Participants With Overall Improvement
Time frame: Up to 8 years, 9 months and 12 days
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 7 Aug 2025.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- University of Texas MD Anderson Cancer Center WebsiteLink provided by the sponsor.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Guillermo Garcia-Manero principal investigator
M.D. Anderson Cancer Center
6 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Azacitidine (AZA) Days 1 - 3, Decitabine (DAC) and Azacitidine (AZA) Days 1 - 5 in people with Leukemia, run by M.D. Anderson Cancer Center at 6 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 2 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 7 Aug 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 6 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 7 Aug 2025). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Event Free Survival (EFS) (time frame: Up to 8 years, 9 months and 12 days). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 7 Aug 2025.