LeukemiaPhase 2NCT01720225
Decitabine Versus Azacitidine in Myelodysplastic Syndrome Patients With Low and Intermediate-1 Risk
A Phase 2 study testing Decitabine and Azacitidine in people with Leukemia, run by M.D. Anderson Cancer Center at 1 site in United States.
Trial summary
The goal of this clinical research study is to compare how two different drugs, decitabine and azacitidine, when given on a shorter than standard dosing schedule can help to control MDS. The safety of the drugs will also be studied. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Leukemia
- What is being tested?
- Decitabine and Azacitidine
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 113 participants
- Where
- 1 siteUnited States
- Study dates
- 6 Nov 2012to 8 Jan 2020
- 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 Institutional Review Board (IRB)-approved informed consent document.
- Age >/= than18 years
- de novo or secondary International Prostate Symptom Score (IPSS) low- or intermediate-1 - risk MDS, including CMML
- Eastern Cooperative Oncology Group (ECOG) performance status of </= 3 at study entry.
- Organ function as defined: Serum creatinine </= 3 x Upper Limit of Normal (ULN), Total bilirubin </= 2 x ULN, Alanine transaminase (ALT) (SGPT) </= 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
1 site in 1 country.
United States1 site
University of Texas MD Anderson Cancer Center
Houston, Texas, 77030
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) in Myelodysplastic Syndrome (MDS) Patients With Low and Intermediate-1 Risk Disease
The goal of this clinical research study is to compare how two different drugs, decitabine and azacitidine, when given on a shorter than standard dosing schedule can help to control MDS. The safety of the drugs will also be studied.
Decitabine is designed to damage the DNA (the genetic material) of cells, which may cause cancer cells to die.
Azacitidine is designed to block certain proteins in cancer cells whose job is to stop the function of the tumor-fighting proteins. By blocking the "bad" proteins, the tumor-fighting genes may be able to work better. This could cause the cancer cells to die.
Read the detailed description
Study Groups:
If you are found to be eligible to take part in this study, you will be randomly assigned (as in the flip of a coin) to 1 of 2 groups:
- If you are in Group 1, you will receive decitabine by vein over about 1 hour.
- If you are in Group 2, you will receive azacitidine either as an injection under your skin or through a vein. If by vein, the infusion will take about an hour.
At first, there will be an equal chance of being assigned to either group. However, as the study goes on and more information becomes available, the chance of being assigned to the group that has shown the most effectiveness will increase. However, once you are already enrolled and assigned to a group, you will not be eligible to change groups.
Study Drug Administration:
Each cycle is 28 days.
You will receive the study drug on Days 1-3 of every cycle and you will receive at least 2 cycles of study drug.
Study Visits:
Every 7-14 days, blood (about 2 tablespoons) will be drawn for routine tests.
At the end of Cycle 2, you will have a bone marrow biopsy and/or aspirate to check the status of the disease. This will then be repeated every 2-4 cycles until any point that the disease appears to have responded to the study drug, then as often as the study doctor thinks is necessary. To collect a bone marrow biopsy/aspirate, an area of the hip bone is numbed with anesthetic, and a small amount of bone and/or bone marrow is withdrawn through a large needle.
After Cycle 3:
If the study doctor decides it is acceptable, you may be allowed to receive treatment from your local cancer doctor. However, you have to return to Houston at least every 3 months for your study visits.
The frequency of the visits will depend on what the doctor thinks is in your best interest.
Length of Study:
You may continue taking the study drug for as long as the doctor thinks it is in your best interest. You will no longer be able to take the study drug if the disease gets worse, if intolerable side effects occur, or if you are unable to follow study directions.
Follow-Up Visits:
One (1) time every 3 months after your last dose of study drug, you will return to the clinic for a bone marrow aspiration to check the status of the disease.
Other Information:
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.
This is an investigational study. Decitabine and Azacitidine are both FDA approved and commercially available for use in patients with MDS.
Up to 120 patients will take part in this study. All will be enrolled at MD Anderson.
- Conditions
- Leukemia
- Being tested
- Decitabine, Azacitidine
- Keywords
- Leukemia, Myelodysplastic syndromes, MDS, Low and Intermediate-1 Risk Disease, Decitabine, DAC, Dacogen, Azacitidine, AZA, 5-azacytidine, 5-aza, Vidaza, 5-AZC, AZA-CR, Ladakamycin, NSC-102816
- 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.
DecitabineDrug
Also called DAC, Dacogen
20 mg/m2 by vein daily for 3 days (days 1-3) every 28 days.
AzacitidineDrug
Also called AZA, 5-azacytidine, 5-aza, Vidaza
75 mg/m2 subcutaneously or by vein daily for 3 days (days 1-3) every 28 days.
| Study group | Role | What they receive |
|---|---|---|
| Decitabine | Experimental | Patients randomized to receive Decitabine 20 mg/m2 by vein daily for 3 days (days 1-3) every 28 days. Receives: Drug: Decitabine |
| Azacitidine | Experimental | Patients randomized to receive Azacitidine 75 mg/m2 subcutaneously or by vein daily for 3 days (days 1-3) every 28 days. Receives: Drug: Azacitidine |
What is being measured
The primary outcome is the main question the study is designed to answer.
Participants With a Response
Overall Response = complete remission (CR) + partial remission (PR) + marrow CR (mCR) + hematologic improvement (HI). CR is normalization of peripheral blood and bone marrow with <5% bone marrow blasts, a peripheral blood granulocyte count > (1.0 x 10^9/L, and platelet count > 100 x 10^9/L). PR is same as CR except for the presence of 6-15% marrow blasts, or 50% reduction if <15% at start of treatment. Marrow CR is blasts </= 5% and decreased by >/=50% from baseline. HI is platelets increase by 50% and to above 30 x 10^9/L untransfused (if lower than that pre-therapy; or hemoglobin increase by 2 g/dl; or transfusion independent; or splenomegaly reduction by > 50%; or monocytosis reduction by > 50% if pretreatment > 5 X1 0^9/L.
Measured: 56 days
Secondary outcomes (1)
Number of Participants Who Became Transfusion Independent
Participants who were transfusion dependent at baseline prior to starting therapy on the Decitabine or Azacitidine arm will be analyzed for transfusion independence. Transfusion independence defined as being transfusion-free for ≥8 consecutive weeks between the first dose and study treatment discontinuation.
Measured: 8 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted2 Nov 2012 (estimated)
- Study start6 Nov 2012
- Primary completion8 Jan 2020
- Study completion8 Jan 2020
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.
- Publications (2)Papers the sponsor has linked to this study.
- 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
- 113 participants (actual)
Sponsor and investigators
- Lead sponsor
- M.D. Anderson Cancer Center
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Elias Jabbour, MD, Principal investigator, M.D. Anderson Cancer Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01720225
- Sponsor's protocol ID
- 2012-0507
- Other IDs
- NCI-2012-02215
- Status last verified
- Dec 2020
- First submitted
- 31 Oct 2012
- Last updated
- 24 Dec 2020
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Publications
Sasaki K, Jabbour E, Montalban-Bravo G, Darbaniyan F, Do KA, Class C, Short NJ, Kanagal-Shamana R, Kadia T, Borthakur G, Pemmaraju N, Cortes J, Ravandi F, Alvarado Y, Chien K, Komrokji R, Sekeres MA, Steensma DP, DeZern A, Roboz G, Soltysiak K, Yang H, Kantarjian HM, Garcia-Manero G. Low-Dose Decitabine versus Low-Dose Azacitidine in Lower-Risk MDS. NEJM Evid. 2022 Oct;1(10):EVIDoa2200034. doi: 10.1056/EVIDoa2200034. Epub 2022 Aug 9.
PubMed 38319837Jabbour E, Short NJ, Montalban-Bravo G, Huang X, Bueso-Ramos C, Qiao W, Yang H, Zhao C, Kadia T, Borthakur G, Pemmaraju N, Sasaki K, Estrov Z, Cortes J, Ravandi F, Alvarado Y, Komrokji R, Sekeres MA, Steensma DP, DeZern A, Roboz G, Kantarjian H, Garcia-Manero G. Randomized phase 2 study of low-dose decitabine vs low-dose azacitidine in lower-risk MDS and MDS/MPN. Blood. 2017 Sep 28;130(13):1514-1522. doi: 10.1182/blood-2017-06-788497. Epub 2017 Aug 3.
PubMed 28774880
Study at a glance
From the registry record, last updated by the sponsor on 24 Dec 2020. 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) in Myelodysplastic Syndrome (MDS) Patients With Low and Intermediate-1 Risk Disease
- ClinicalTrials.gov ID
- NCT01720225
- Acronym / sponsor ID
- 2012-0507
- Conditions
- Leukemia
- Keywords
- Leukemia, Myelodysplastic syndromes, MDS, Low and Intermediate-1 Risk Disease, Decitabine, DAC, Dacogen, Azacitidine
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 113 participants (actual)
- Lead sponsor
- M.D. Anderson Cancer CenterUniversity, hospital or other
- Study start
- 6 Nov 2012 (actual)
- Primary completion
- 8 Jan 2020 (actual)
- Study completion
- 8 Jan 2020 (actual)
- First posted
- 2 Nov 2012 (estimated)
- Results
- Posted 24 Dec 2020
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 two different drugs, decitabine and azacitidine, when given on a shorter than standard dosing schedule can help to control MDS. The safety of the drugs will also be studied.
Decitabine is designed to damage the DNA (the genetic material) of cells, which may cause cancer cells to die.
Azacitidine is designed to block certain proteins in cancer cells whose job is to stop the function of the tumor-fighting proteins. By blocking the "bad" proteins, the tumor-fighting genes may be able to work better. This could cause the cancer cells to die.
Detailed description
Study Groups:
If you are found to be eligible to take part in this study, you will be randomly assigned (as in the flip of a coin) to 1 of 2 groups:
- If you are in Group 1, you will receive decitabine by vein over about 1 hour.
- If you are in Group 2, you will receive azacitidine either as an injection under your skin or through a vein. If by vein, the infusion will take about an hour.
At first, there will be an equal chance of being assigned to either group. However, as the study goes on and more information becomes available, the chance of being assigned to the group that has shown the most effectiveness will increase. However, once you are already enrolled and assigned to a group, you will not be eligible to change groups.
Study Drug Administration:
Each cycle is 28 days.
You will receive the study drug on Days 1-3 of every cycle and you will receive at least 2 cycles of study drug.
Study Visits:
Every 7-14 days, blood (about 2 tablespoons) will be drawn for routine tests.
At the end of Cycle 2, you will have a bone marrow biopsy and/or aspirate to check the status of the disease. This will then be repeated every 2-4 cycles until any point that the disease appears to have responded to the study drug, then as often as the study doctor thinks is necessary. To collect a bone marrow biopsy/aspirate, an area of the hip bone is numbed with anesthetic, and a small amount of bone and/or bone marrow is withdrawn through a large needle.
After Cycle 3:
If the study doctor decides it is acceptable, you may be allowed to receive treatment from your local cancer doctor. However, you have to return to Houston at least every 3 months for your study visits.
The frequency of the visits will depend on what the doctor thinks is in your best interest.
Length of Study:
You may continue taking the study drug for as long as the doctor thinks it is in your best interest. You will no longer be able to take the study drug if the disease gets worse, if intolerable side effects occur, or if you are unable to follow study directions.
Follow-Up Visits:
One (1) time every 3 months after your last dose of study drug, you will return to the clinic for a bone marrow aspiration to check the status of the disease.
Other Information:
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.
This is an investigational study. Decitabine and Azacitidine are both FDA approved and commercially available for use in patients with MDS.
Up to 120 patients will take part in this study. All will be enrolled at MD Anderson.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Decitabine | Experimental | Drug: DecitabinePatients randomized to receive Decitabine 20 mg/m2 by vein daily for 3 days (days 1-3) every 28 days. |
| Azacitidine | Experimental | Drug: AzacitidinePatients randomized to receive Azacitidine 75 mg/m2 subcutaneously or by vein daily for 3 days (days 1-3) every 28 days. |
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 Institutional Review Board (IRB)-approved informed consent document.
- Age >/= than18 years
- de novo or secondary International Prostate Symptom Score (IPSS) low- or intermediate-1 - risk MDS, including CMML
- Eastern Cooperative Oncology Group (ECOG) performance status of </= 3 at study entry.
- Organ function as defined: Serum creatinine </= 3 x Upper Limit of Normal (ULN), Total bilirubin </= 2 x ULN, Alanine transaminase (ALT) (SGPT) </= 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 |
|---|---|---|
| DecitabineAlso: DAC, Dacogen20 mg/m2 by vein daily for 3 days (days 1-3) every 28 days. | Drug | Decitabine |
| AzacitidineAlso: AZA, 5-azacytidine, 5-aza, Vidaza75 mg/m2 subcutaneously or by vein daily for 3 days (days 1-3) every 28 days. | Drug | Azacitidine |
Primary outcome
Participants With a Response
Time frame: 56 days
Overall Response = complete remission (CR) + partial remission (PR) + marrow CR (mCR) + hematologic improvement (HI). CR is normalization of peripheral blood and bone marrow with <5% bone marrow blasts, a peripheral blood granulocyte count > (1.0 x 10^9/L, and platelet count > 100 x 10^9/L). PR is same as CR except for the presence of 6-15% marrow blasts, or 50% reduction if <15% at start of treatment. Marrow CR is blasts </= 5% and decreased by >/=50% from baseline. HI is platelets increase by 50% and to above 30 x 10^9/L untransfused (if lower than that pre-therapy; or hemoglobin increase by 2 g/dl; or transfusion independent; or splenomegaly reduction by > 50%; or monocytosis reduction by > 50% if pretreatment > 5 X1 0^9/L.
1 secondary outcome
Number of Participants Who Became Transfusion Independent
Time frame: 8 weeks
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 24 Dec 2020.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 38319837Sasaki K, Jabbour E, Montalban-Bravo G, Darbaniyan F, Do KA, Class C, Short NJ, Kanagal-Shamana R, Kadia T, Borthakur G, Pemmaraju N, Cortes J, Ravandi F, Alvarado Y, Chien K, Komrokji R, Sekeres MA, Steensma DP, DeZern A, Roboz G, Soltysiak K, Yang H, Kantarjian HM, Garcia-Manero G. Low-Dose Decitabine versus Low-Dose Azacitidine in Lower-Risk MDS. NEJM Evid. 2022 Oct;1(10):EVIDoa2200034. doi: 10.1056/EVIDoa2200034. Epub 2022 Aug 9.
- Derived from this study: PubMed 28774880Jabbour E, Short NJ, Montalban-Bravo G, Huang X, Bueso-Ramos C, Qiao W, Yang H, Zhao C, Kadia T, Borthakur G, Pemmaraju N, Sasaki K, Estrov Z, Cortes J, Ravandi F, Alvarado Y, Komrokji R, Sekeres MA, Steensma DP, DeZern A, Roboz G, Kantarjian H, Garcia-Manero G. Randomized phase 2 study of low-dose decitabine vs low-dose azacitidine in lower-risk MDS and MDS/MPN. Blood. 2017 Sep 28;130(13):1514-1522. doi: 10.1182/blood-2017-06-788497. Epub 2017 Aug 3.
- University of Texas MD Anderson Cancer Center WebsiteLink provided by the sponsor.
Related trials for Leukemia
RecruitingPhase 2
Tafasitamab With Acalabrutinib and Venetoclax for the Treatment of Chronic Lymphocytic Leukemia/Small Lymphocytic Lymphoma
Chronic Lymphocytic Leukemia, Chronic Lymphocytic Leukemia/Small Lymphocytic Lymphoma
Enrolling by invitationObservational study
Natural History Study of Early Life Exposures in Agriculture (ELEA)
Prostate Cancer, Leukemia
RecruitingPhase 1
A Study of CHM-029 in Participants With NPM1 Mutated, KMT2A or NUP98 Rearranged AML
AML (Acute Myeloid Leukemia)
RecruitingPhase 1
Vididencel in CML-CP Patients With MRD
CML (Chronic Myelogenous Leukemia)
RecruitingPhase 2
Testing New Therapies for Patients With Acute Myeloid Leukemia Who Are Newly Diagnosed and Have Not Yet Started Treatment (A MyeloMATCH Treatment Trial)
Acute Myeloid Leukemia, Acute Myeloid Leukemia Arising From Previous Myelodysplastic/Myeloproliferative Neoplasm
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.
Elias Jabbour, MD principal investigator
M.D. Anderson Cancer Center
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Decitabine and Azacitidine in people with Leukemia, run by M.D. Anderson Cancer Center at 1 site 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 24 Dec 2020. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site 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 24 Dec 2020). 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?
Participants With a Response (time frame: 56 days). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT01720225, which also lists 2 linked publications.
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 24 Dec 2020.