LeukemiaPhase 2NCT01786343
Decitabine for Older or Unfit Patients With Acute Myeloid Leukemia (AML)
A Phase 2 study testing Decitabine 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 well 2 different dosing schedules of decitabine may help control AML. Decitabine is designed to damage the DNA (the genetic material) of cells, which may cause cancer cells to die. Read more about this study
- Who is it for?
- Ages 60 years and older
- Conditions the study is for
- Leukemia
- What is being tested?
- Decitabine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 74 participants
- Where
- 1 siteUnited States
- Study dates
- 5 Feb 2013to 6 May 2019
- 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 60 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Patients with previously untreated AML (by the World Health Organization (WHO) criteria, i.e. >/= 20% blasts) Prior biologic therapies (such as growth factors) and targeted therapies administered for the treatment of prior myelodysplastic syndrome are allowed, with the exception of hypomethylating agents 5-azacytidine or decitabine. Patients must have been off such therapy for 1 week prior to entering this study and recovered from the toxic effects of that therapy, unless there is evidence of rapidly progressive disease. Hydroxyurea, and a single dose of cytarabine up to 3 g/m2, is permitted for control of counts prior to treatment.
- Patients >/= 60 are eligible if not a candidate for standard cytarabine plus anthracycline chemotherapy as determined by Kantarjian's score (Appendix D) Patients younger than 60 may also be included if felt not to be a candidate for intensive anthracycline plus cytarabine based chemotherapy.
- Performance 0-3 (ECOG).
- Adequate liver function (Total bilirubin of < 2 mg/dl) unless due to hemolysis, leukemia organ infiltration or Gilbert's syndrome and renal function (creatinine < 2.5 mg/dl).
- Signed informed consent
Who cannot join
- Nursing and pregnant females. Female patients of childbearing potential and male patients should practice effective methods of contraception such as double barrier method. Should a woman become pregnant or suspect she is pregnant while participating in this study, she should inform her treating physician immediately. Negative urine pregnancy test (women of childbearing potential)
- Active and uncontrolled infections.
- Uncontrolled intercurrent illness including, but not limited to, symptomatic congestive heart failure, unstable angina pectoris, active significant other cancers requiring chemotherapy and/or radiation therapy within past 6 months (excluding non-melanoma skin cancer) or psychiatric illness/social situations that would limit compliance with study requirements.
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: A Randomized Phase II Study of Two Schedules of Decitabine for Frontline Therapy of Older or Unfit Patients With Acute Myeloid Leukemia (AML)
The goal of this clinical research study is to compare how well 2 different dosing schedules of decitabine may help control AML.
Decitabine is designed to damage the DNA (the genetic material) of cells, which may cause 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 assigned to 1 of 2 dose levels of decitabine based on when you join this study. If you are among the first 20 participants, you will have an equal chance of being in either group. If you enroll after that, you will have an increasingly higher chance (51-100%) of being assigned to the group that had better results, depending on how much better that treatment arm is.
Study Drug Administration:
Each cycle is about 4-8 weeks, depending on the doctor's decision. In this study you will receive induction therapy to try to control the disease and cause remission (this is when tests and/or the doctor cannot find signs of the disease).
If you are in Group 1, you will receive decitabine by vein over about 1 hour for 5 days.
If you are in Group 2, you will receive decitabine by vein over about 1 hour for 10 days.
If the disease is in remission, you may receive more cycles (called maintenance) to help keep the disease under control. If you are in Group 2, you will receive 5 day dosing during maintenance, or when the doctor thinks it is in your best interest.
Your dose schedule or dose level may be changed if the doctor feels it is in your best interest.
Study Visits:
The following tests and procedures will be performed:
- Blood (about 2-3 teaspoons) will be drawn 1-2 times weekly for first cycle, then every 2-4 weeks after that. After the 6th cycle or sooner if the doctor decides, this blood draw will be performed only 1 time per cycle.
- Every 1-3 cycles, you will have a bone marrow aspiration/biopsy to check the status of the disease. Blood (about 2-3 teaspoons) may also be drawn for genetic testing if the disease is in remission and the doctor thinks it is needed.
Length of Treatment:
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.
Your participation in this study will be over after the follow-up phone calls.
Follow-Up:
After you stop the study treatment, you will be called by phone twice a year and asked how you are feeling. The phone calls should last about 5 minutes each time.
This is an investigational study. Decitabine is FDA approved and commercially available for the treatment of myelodysplastic syndrome (MDS). Its use to treat AML is investigational.
Up to 100 participants will be enrolled in this study. All will take part at MD Anderson.
- Conditions
- Leukemia
- Being tested
- Decitabine
- Keywords
- Leukemia, Acute myeloid leukemia, AML, Response rates, Decitabine, Dacogen
- Medical subject headings
- Leukemia, Leukemia, Myeloid, Acute
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
DecitabineDrug
Also called Dacogen
20 mg/m2 by vein daily for either 5 or 10 days.
| Study group | Role | What they receive |
|---|---|---|
| Decitabine - 5 Day Regimen | Experimental | Decitabine 20 mg/m2 by vein daily for 5 days. Receives: Drug: Decitabine |
| Decitabine - 10 Day Regimen | Experimental | Decitabine 20 mg/m2 by vein daily for 10 days. Receives: Drug: Decitabine |
What is being measured
The primary outcome is the main question the study is designed to answer.
Participants With a Response
Response is defined as Complete Response (CR) + Partial Remission (PR) + Complete Remission with incomplete recovery (CRi) + Clinical Benefit. CR is the normalization of the peripheral blood and bone marrow with </= 5% bone marrow blasts, a peripheral blood granulocyte count >/= (1.0 x 10^9/L, and a platelet count >/= 100 x 10^9/L). PR is the same as CR except for the presence of 6-15% marrow blasts, or 50% reduction if <15% at start of treatment. CRi meets all criteria for CR except for platelet recovery to >100 x 10^9/L and/or granulocyte count > (1.0 x 10^9/L). Clinical benefit is platelets increase by 50% and to above 30 x 10^9/L untransfused (if lower than that pretherapy); or granulocytes increase by 100% and to above 10^9/L (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 x 109/L.
Measured: Up to 3 months
Secondary outcomes (2)
Overall Survival
Time from date of treatment start until date of death due to any cause or last Follow-up.
Measured: Up to 5 years
Response Duration
The date of response to date of loss of response or last follow-up. Response is defined as Complete Response (CR) + Partial Remission (PR) + Complete Remission with incomplete recovery (CRi) + Clinical Benefit. CR is the normalization of the peripheral blood and bone marrow with </= 5% bone marrow blasts, a peripheral blood granulocyte count >/= (1.0 x 10^9/L, and a platelet count >/= 100 x 10^9/L). PR is the same as CR except for the presence of 6-15% marrow blasts, or 50% reduction if <15% at start of treatment. CRi meets all criteria for CR except for platelet recovery to >100 x 10^9/L and/or granulocyte count > (1.0 x 10^9/L). Clinical benefit is platelets increase by 50% and to above 30 x 10^9/L untransfused (if lower than that pretherapy); or granulocytes increase by 100% and to above 10^9/L (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
Measured: Up to 5 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted7 Feb 2013 (estimated)
- Study start5 Feb 2013
- Primary completion6 May 2019
- Study completion6 May 2019
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 (1)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
- 74 participants (actual)
Sponsor and investigators
- Lead sponsor
- M.D. Anderson Cancer Center
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Farhad Ravandi-Kashani, MD, Principal investigator, M.D. Anderson Cancer Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01786343
- Sponsor's protocol ID
- 2012-1017
- Other IDs
- NCI-2013-00463
- Status last verified
- Apr 2020
- First submitted
- 5 Feb 2013
- Last updated
- 15 Apr 2020
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Publications
Short NJ, Kantarjian HM, Loghavi S, Huang X, Qiao W, Borthakur G, Kadia TM, Daver N, Ohanian M, Dinardo CD, Estrov Z, Kanagal-Shamanna R, Maiti A, Benton CB, Bose P, Alvarado Y, Jabbour E, Kornblau SM, Pemmaraju N, Jain N, Gasior Y, Richie MA, Pierce S, Cortes J, Konopleva M, Garcia-Manero G, Ravandi F. Treatment with a 5-day versus a 10-day schedule of decitabine in older patients with newly diagnosed acute myeloid leukaemia: a randomised phase 2 trial. Lancet Haematol. 2019 Jan;6(1):e29-e37. doi: 10.1016/S2352-3026(18)30182-0. Epub 2018 Dec 10.
PubMed 30545576
Study at a glance
From the registry record, last updated by the sponsor on 15 Apr 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Randomized Phase II Study of Two Schedules of Decitabine for Frontline Therapy of Older or Unfit Patients With Acute Myeloid Leukemia (AML)
- ClinicalTrials.gov ID
- NCT01786343
- Acronym / sponsor ID
- 2012-1017
- Conditions
- Leukemia
- Keywords
- Leukemia, Acute myeloid leukemia, AML, Response rates, Decitabine, Dacogen
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 74 participants (actual)
- Lead sponsor
- M.D. Anderson Cancer CenterUniversity, hospital or other
- Study start
- 5 Feb 2013 (actual)
- Primary completion
- 6 May 2019 (actual)
- Study completion
- 6 May 2019 (actual)
- First posted
- 7 Feb 2013 (estimated)
- Results
- Posted 15 Apr 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 well 2 different dosing schedules of decitabine may help control AML.
Decitabine is designed to damage the DNA (the genetic material) of cells, which may cause cancer cells to die.
Detailed description
Study Groups:
If you are found to be eligible to take part in this study, you will be assigned to 1 of 2 dose levels of decitabine based on when you join this study. If you are among the first 20 participants, you will have an equal chance of being in either group. If you enroll after that, you will have an increasingly higher chance (51-100%) of being assigned to the group that had better results, depending on how much better that treatment arm is.
Study Drug Administration:
Each cycle is about 4-8 weeks, depending on the doctor's decision. In this study you will receive induction therapy to try to control the disease and cause remission (this is when tests and/or the doctor cannot find signs of the disease).
If you are in Group 1, you will receive decitabine by vein over about 1 hour for 5 days.
If you are in Group 2, you will receive decitabine by vein over about 1 hour for 10 days.
If the disease is in remission, you may receive more cycles (called maintenance) to help keep the disease under control. If you are in Group 2, you will receive 5 day dosing during maintenance, or when the doctor thinks it is in your best interest.
Your dose schedule or dose level may be changed if the doctor feels it is in your best interest.
Study Visits:
The following tests and procedures will be performed:
- Blood (about 2-3 teaspoons) will be drawn 1-2 times weekly for first cycle, then every 2-4 weeks after that. After the 6th cycle or sooner if the doctor decides, this blood draw will be performed only 1 time per cycle.
- Every 1-3 cycles, you will have a bone marrow aspiration/biopsy to check the status of the disease. Blood (about 2-3 teaspoons) may also be drawn for genetic testing if the disease is in remission and the doctor thinks it is needed.
Length of Treatment:
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.
Your participation in this study will be over after the follow-up phone calls.
Follow-Up:
After you stop the study treatment, you will be called by phone twice a year and asked how you are feeling. The phone calls should last about 5 minutes each time.
This is an investigational study. Decitabine is FDA approved and commercially available for the treatment of myelodysplastic syndrome (MDS). Its use to treat AML is investigational.
Up to 100 participants will be enrolled in this study. All will take part 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 - 5 Day Regimen | Experimental | Drug: DecitabineDecitabine 20 mg/m2 by vein daily for 5 days. |
| Decitabine - 10 Day Regimen | Experimental | Drug: DecitabineDecitabine 20 mg/m2 by vein daily for 10 days. |
Who may qualify
- Ages
- 60 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (5 listed)
- Patients with previously untreated AML (by the World Health Organization (WHO) criteria, i.e. >/= 20% blasts) Prior biologic therapies (such as growth factors) and targeted therapies administered for the treatment of prior myelodysplastic syndrome are allowed, with the exception of hypomethylating agents 5-azacytidine or decitabine. Patients must have been off such therapy for 1 week prior to entering this study and recovered from the toxic effects of that therapy, unless there is evidence of rapidly progressive disease. Hydroxyurea, and a single dose of cytarabine up to 3 g/m2, is permitted for control of counts prior to treatment.
- Patients >/= 60 are eligible if not a candidate for standard cytarabine plus anthracycline chemotherapy as determined by Kantarjian's score (Appendix D) Patients younger than 60 may also be included if felt not to be a candidate for intensive anthracycline plus cytarabine based chemotherapy.
- Performance 0-3 (ECOG).
- Adequate liver function (Total bilirubin of < 2 mg/dl) unless due to hemolysis, leukemia organ infiltration or Gilbert's syndrome and renal function (creatinine < 2.5 mg/dl).
- Signed informed consent
Key exclusion criteria (3 listed)
- Nursing and pregnant females. Female patients of childbearing potential and male patients should practice effective methods of contraception such as double barrier method. Should a woman become pregnant or suspect she is pregnant while participating in this study, she should inform her treating physician immediately. Negative urine pregnancy test (women of childbearing potential)
- Active and uncontrolled infections.
- Uncontrolled intercurrent illness including, but not limited to, symptomatic congestive heart failure, unstable angina pectoris, active significant other cancers requiring chemotherapy and/or radiation therapy within past 6 months (excluding non-melanoma skin cancer) or psychiatric illness/social situations that would limit compliance with study requirements.
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: Dacogen20 mg/m2 by vein daily for either 5 or 10 days. | Drug | Decitabine - 10 Day Regimen, Decitabine - 5 Day Regimen |
Primary outcome
Participants With a Response
Time frame: Up to 3 months
Response is defined as Complete Response (CR) + Partial Remission (PR) + Complete Remission with incomplete recovery (CRi) + Clinical Benefit. CR is the normalization of the peripheral blood and bone marrow with </= 5% bone marrow blasts, a peripheral blood granulocyte count >/= (1.0 x 10^9/L, and a platelet count >/= 100 x 10^9/L). PR is the same as CR except for the presence of 6-15% marrow blasts, or 50% reduction if <15% at start of treatment. CRi meets all criteria for CR except for platelet recovery to >100 x 10^9/L and/or granulocyte count > (1.0 x 10^9/L). Clinical benefit is platelets increase by 50% and to above 30 x 10^9/L untransfused (if lower than that pretherapy); or granulocytes increase by 100% and to above 10^9/L (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 x 109/L.
2 secondary outcomes
Overall Survival
Time frame: Up to 5 years
Response Duration
Time frame: Up to 5 years
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 15 Apr 2020.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 30545576Short NJ, Kantarjian HM, Loghavi S, Huang X, Qiao W, Borthakur G, Kadia TM, Daver N, Ohanian M, Dinardo CD, Estrov Z, Kanagal-Shamanna R, Maiti A, Benton CB, Bose P, Alvarado Y, Jabbour E, Kornblau SM, Pemmaraju N, Jain N, Gasior Y, Richie MA, Pierce S, Cortes J, Konopleva M, Garcia-Manero G, Ravandi F. Treatment with a 5-day versus a 10-day schedule of decitabine in older patients with newly diagnosed acute myeloid leukaemia: a randomised phase 2 trial. Lancet Haematol. 2019 Jan;6(1):e29-e37. doi: 10.1016/S2352-3026(18)30182-0. Epub 2018 Dec 10.
- 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.
Farhad Ravandi-Kashani, 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 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 5 inclusion and 3 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 15 Apr 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 15 Apr 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: Up to 3 months). 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 NCT01786343, which also lists 1 linked publication.
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 15 Apr 2020.