Acute Myeloid LeukemiaPhase 2NCT01305499
A Trial to Evaluate Two Schedules of MS275 in Combination With 5AC in Elderly Patients With Acute Myeloid Leukemia (AML)
A Phase 2 study testing Entinostat days 3, 10, 5AC and Entinostat days 10,17 in people with Acute Myeloid Leukemia, run by Hetty Carraway at 4 sites in United States.
Trial summary
This research is being done to help us learn how to best use new drugs which may be active against acute myeloid leukemia (AML). Two study drugs will be tested: 5AC (5-azacitidine) and entinostat. 5AC improves blood counts in 50 - 60% of patients with MDS and has also shown promise in AML. Read more about this study
- Who is it for?
- Ages 60 years and older
- Conditions the study is for
- Acute Myeloid Leukemia
- What is being tested?
- Entinostat days 3, 10, 5AC and Entinostat days 10,17
- Type of treatment
- Pill or oral medicine, Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 52 participants
- Where
- 4 sitesUnited States
- Study dates
- 1 Jul 2011to 30 Sep 2024
- Phase
- Phase 2
- Are there results?
- None posted in the registry
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
- One of the following:
- Untreated AML in (de novo or treatment related) patients in the following categories:
- Medical conditions that compromise the ability to give cytotoxic chemotherapy as the primary modality.
- Patients who decline cytotoxic chemotherapy.
- Patients with AML who have relapsed despite one prior regimen
- ECOG performance status 0, 1, or 2
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- Patients must not have untreated active infections at the time of study entry.
- Normal organ function as defined below:
- Creatinine < 2 mg/dl.
- Total serum bilirubin within institutional limits unless due to hemolysis, Gilbert's syndrome, or ineffective erythropoiesis.
- AST(SGOT)/ALT(SGPT) ≤2.5 X institutional upper limit of normal.
- Life expectancy of at least three months.
- Patients must be informed of the investigational nature of the treatment, results that might be expected, and potential toxicities. They must be able to understand and give informed written consent according to federal and institutional guidelines.
- Declined or ineligible for potentially curative options such as allogeneic stem cell transplant.
- No chemotherapy or study drugs for >3 weeks prior to starting study.
- Women of childbearing potential should be advised to avoid becoming pregnant and men should be advised to not father a child while receiving treatment. All men and women of childbearing potential must use acceptable methods of birth control throughout the study as described below:
- Females of childbearing potential: Recommendation is for 2 effective contraceptive methods during the study. Adequate forms of contraception are double-barrier methods (condoms with spermicidal jelly or foam and diaphragm with spermicidal jelly or foam), oral, depo provera, or injectable contraceptives, intrauterine devices, and tubal ligation.
- Male patients with female partners who are of childbearing potential: Recommendation is for male and partner to use at least 2 effective contraceptive methods, as described above, during the study or to abstain.
Who cannot join
- Any of the Following:
- Treatment for acute myeloid leukemia (AML), including hematopoietic growth factors, < 3 weeks prior to study registration. Exception: Hydroxyurea may be administered to patients with WBC > 30,000/µL
- Diagnosis of acute promyelocytic leukemia (APL)
- Radiotherapy < 4 weeks prior to study registration
- Failure to recover (to < grade 1) from all adverse events associated with prior therapy.
- Valproic acid < 2 weeks prior to study registration.
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- Hypersensitivity to azacytidine, deoxyazacytidine, mannitol, entinostat or components of the entinostat tablet
- Any advanced malignant hepatic tumor(s)
- Prior therapy with demethylating agents for leukemia treatment within the last four months.
- Clinical evidence of CNS or pulmonary leukostasis, disseminated intravascular coagulation, or central nervous system leukemia.
- Serious or uncontrolled medical conditions.
- Concurrent use of any other investigational agents.
- Known HIV-positive patients.
- Pregnancy or breast feeding
- Male and female patients who are fertile who do not agree to use an effective barrier methods of birth control (i.e. abstinence) to avoid pregnancy during the study and for a minimum of 30 days after study treatment.
Trial locations and contacts
4 sites in 1 country.
United States4 sites
Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins
Baltimore, Maryland, 21231
University Hospitals Cleveland Medical Center, Case Comprehensive Cancer Center
Cleveland, Ohio, 44106
Cleveland Clinic Taussig Cancer institute, Case Comprehensive Cancer Center
Cleveland, Ohio, 44195
Yale School of Medicine
New Haven, Connecticut, 06520
About this study
Official title: A Randomized Phase II Trial to Simultaneously Evaluate Two Schedules of the Histone Deacetylase Inhibitor Entinostat in Combination With 5-Azacytidine (5AC, NSC 102816) in Elderly Patients With Acute Myeloid Leukemia (AML)
This research is being done to help us learn how to best use new drugs which may be active against acute myeloid leukemia (AML). Two study drugs will be tested: 5AC (5-azacitidine) and entinostat. 5AC improves blood counts in 50 - 60% of patients with MDS and has also shown promise in AML. Entinostat has undergone early testing in patients with MDS and AML. It has decreased the blast count in some patients' blood and bone marrow and has improved the blood counts in some patients. The combinations of these two classes of drugs are well tolerated and appear to work well together in laboratory tests.
A recent study at Johns Hopkins University administered 5AC and entinostat in an overlapping schedule to patients with myelodysplastic syndrome (MDS), Chronic myelomonocytic leukemia (CMMoL), and AML. The impressive results from this study have led to another phase II trial to further examine this drug combination versus 5AC alone in these patients. In this study, we want to see how the timing of when 5AC and entinostat are given affects the magnitude of the disease response.
Read the detailed description
- To estimate the major response rate (complete and partial responses by the International Working Group (IWG) response criteria) in patients with AML who are >= 60 years old and unable to tolerate or decline cytotoxic chemotherapy or patients who have relapsed despite one prior regimen and are treated with (a) 5AC 50mg/m2 subcutaneously/intravenously for 10 days on days 1 - 10 of a 28 day cycle given in combination with entinostat 8 mg (flat dose) administered orally on days 3 and10 of each cycle or (b) the same regimen of 5AC with entinostat given on days 10 and 17.
- To estimate the overall response rate (complete, partial, and hematologic improvement- major by IWG criteria) following treatment with two different dose schedules of 5-Azacytidine and entinostat in patients with AML >= 60 years old who are unable to tolerate or decline cytotoxic chemotherapy or those who have relapsed despite one prior regimen.
The secondary objectives of the study are:
- To identify changes in gene promoter methylation and gene expression in response to combination therapy with 5AC and entinostat and compare the dynamics and kinetics of these alterations in promoter methylation and gene re-expression in the two different dosing schedules.
- To evaluate the effect of entinostat on the induction of hyperacetylation of histones from peripheral blood and/or bone marrow samples.
- To evaluate changes in DNA damage in response to combination therapy using gammaH2AX determination by western blotting.
- To evaluate immune parameters after exposure to 5AC and entinostat when given at either dosing schedule and to evaluate these in relation to clinical outcomes.
- To evaluate duration of response.
- Conditions
- Acute Myeloid Leukemia
- Being tested
- Entinostat days 3, 10, 5AC, Entinostat days 10,17
- Keywords
- AML
- Medical subject headings
- Leukemia, Myeloid, Acute
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Entinostat days 3, 10Drug
Also called MS275
Given orally on days 3, 10
5ACDrug
Also called 5-azacitidine
Entinostat days 10,17Drug
Also called MS275
Given orally on days 10, 17
| Study group | Role | What they receive |
|---|---|---|
| A: 5AC days 1-10 / entinostat days 3, 10 | Experimental | Arm A will be given an overlapping schedule of drugs with 5AC given at 50mg/m2 subcutaneously daily for 10 days on days 1 - 10 of a 28 day cycle and entinostat given at a flat dose of 8 mg orally on days 3 and 10. Receives: Drug: Entinostat days 3, 10; Drug: 5AC |
| B: 5AC days 1-10 / entinostat days 10,17 | Experimental | In Arm B the agents will be administered sequentially with 5AC given at 50mg/m2 subcutaneously daily for 10 days on days 1 - 10 of a 28 day cycle followed by entinostat at a 8 mg flat dose on days 10 and 17. Receives: Drug: 5AC; Drug: Entinostat days 10,17 |
What is being measured
The primary outcome is the main question the study is designed to answer.
To estimate the major response rate in patients with AML who are ≥ 60 years old and unable to tolerate or decline cytotoxic chemotherapy or patients who have relapsed despite one prior regimen.
Measured: Up to 15 cycles (420 days)
To estimate the overall response rate following treatment in patients with AML ≥ 60 years old who are unable to tolerate or decline cytotoxic chemotherapy or those who have relapsed despite one prior regimen.
Measured: Up to 15 cycles (420 days)
Secondary outcomes (5)
To identify changes in gene promoter methylation and gene expression in response to combination therapy and compare the dynamics and kinetics of these alterations in promoter methylation and gene re-expression in the two different dosing schedules.
Measured: Up to 15 cycles (420 days)
To evaluate the effect of entinostat on the induction of hyperacetylation of histones from peripheral blood and/or bone marrow samples.
Measured: Up to 15 cycles (420 days)
To evaluate changes in DNA damage in response to combination therapy using gammaH2AX determination by western blotting.
Measured: Up to 15 cycles (420 days)
To evaluate the pharmacodynamics of 5AC and entinostat when given at either dosing schedule and to evaluate these in relation to pharmacokinetic and clinical outcomes.
Measured: Up to 15 cycles (420 days)
To evaluate duration of response.
Measured: Up to 15 cycles (420 days)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted28 Feb 2011 (estimated)
- Study start1 Jul 2011
- Primary completion30 Sep 2021
- Study completion30 Sep 2024 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Acute Myeloid LeukemiaStudies of the same condition, open and under way.
- 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
- 52 participants (actual)
Sponsor and investigators
- Lead sponsor
- Hetty Carraway
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor-investigator, Hetty Carraway, Case Comprehensive Cancer Center
- Investigators
- Hetty Carraway, MD, Principal investigator, Cleveland Clinic Taussig Cancer institute, Case Comprehensive Cancer Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01305499
- Sponsor's protocol ID
- CASE2914
- Other IDs
- 15-851
- Status last verified
- May 2024
- First submitted
- 25 Feb 2011
- Last updated
- 28 May 2024
- Results posted
- No
- 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 28 May 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Randomized Phase II Trial to Simultaneously Evaluate Two Schedules of the Histone Deacetylase Inhibitor Entinostat in Combination With 5-Azacytidine (5AC, NSC 102816) in Elderly Patients With Acute Myeloid Leukemia (AML)
- ClinicalTrials.gov ID
- NCT01305499
- Acronym / sponsor ID
- CASE2914
- Conditions
- Acute Myeloid Leukemia
- Keywords
- AML
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 52 participants (actual)
- Lead sponsor
- Hetty CarrawayUniversity, hospital or other
- Study start
- 1 Jul 2011 (actual)
- Primary completion
- 30 Sep 2021 (actual)
- Study completion
- 30 Sep 2024 (estimated)
- First posted
- 28 Feb 2011 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This research is being done to help us learn how to best use new drugs which may be active against acute myeloid leukemia (AML). Two study drugs will be tested: 5AC (5-azacitidine) and entinostat. 5AC improves blood counts in 50 - 60% of patients with MDS and has also shown promise in AML. Entinostat has undergone early testing in patients with MDS and AML. It has decreased the blast count in some patients' blood and bone marrow and has improved the blood counts in some patients. The combinations of these two classes of drugs are well tolerated and appear to work well together in laboratory tests.
A recent study at Johns Hopkins University administered 5AC and entinostat in an overlapping schedule to patients with myelodysplastic syndrome (MDS), Chronic myelomonocytic leukemia (CMMoL), and AML. The impressive results from this study have led to another phase II trial to further examine this drug combination versus 5AC alone in these patients. In this study, we want to see how the timing of when 5AC and entinostat are given affects the magnitude of the disease response.
Detailed description
- To estimate the major response rate (complete and partial responses by the International Working Group (IWG) response criteria) in patients with AML who are >= 60 years old and unable to tolerate or decline cytotoxic chemotherapy or patients who have relapsed despite one prior regimen and are treated with (a) 5AC 50mg/m2 subcutaneously/intravenously for 10 days on days 1 - 10 of a 28 day cycle given in combination with entinostat 8 mg (flat dose) administered orally on days 3 and10 of each cycle or (b) the same regimen of 5AC with entinostat given on days 10 and 17.
- To estimate the overall response rate (complete, partial, and hematologic improvement- major by IWG criteria) following treatment with two different dose schedules of 5-Azacytidine and entinostat in patients with AML >= 60 years old who are unable to tolerate or decline cytotoxic chemotherapy or those who have relapsed despite one prior regimen.
The secondary objectives of the study are:
- To identify changes in gene promoter methylation and gene expression in response to combination therapy with 5AC and entinostat and compare the dynamics and kinetics of these alterations in promoter methylation and gene re-expression in the two different dosing schedules.
- To evaluate the effect of entinostat on the induction of hyperacetylation of histones from peripheral blood and/or bone marrow samples.
- To evaluate changes in DNA damage in response to combination therapy using gammaH2AX determination by western blotting.
- To evaluate immune parameters after exposure to 5AC and entinostat when given at either dosing schedule and to evaluate these in relation to clinical outcomes.
- To evaluate duration of response.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| A: 5AC days 1-10 / entinostat days 3, 10 | Experimental | Drug: Entinostat days 3, 10, Drug: 5ACArm A will be given an overlapping schedule of drugs with 5AC given at 50mg/m2 subcutaneously daily for 10 days on days 1 - 10 of a 28 day cycle and entinostat given at a flat dose of 8 mg orally on days 3 and 10. |
| B: 5AC days 1-10 / entinostat days 10,17 | Experimental | Drug: 5AC, Drug: Entinostat days 10,17In Arm B the agents will be administered sequentially with 5AC given at 50mg/m2 subcutaneously daily for 10 days on days 1 - 10 of a 28 day cycle followed by entinostat at a 8 mg flat dose on days 10 and 17. |
Who may qualify
- Ages
- 60 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (18 listed)
- One of the following:
- Patients with AML who have relapsed despite one prior regimen
- ECOG performance status 0, 1, or 2
- Patients must not have untreated active infections at the time of study entry.
- Normal organ function as defined below:
Key exclusion criteria (15 listed)
- Any of the Following:
- Prior therapy with demethylating agents for leukemia treatment within the last four months.
- Clinical evidence of CNS or pulmonary leukostasis, disseminated intravascular coagulation, or central nervous system leukemia.
- Serious or uncontrolled medical conditions.
- Concurrent use of any other investigational agents.
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 |
|---|---|---|
| Entinostat days 3, 10Also: MS275Given orally on days 3, 10 | Drug | A: 5AC days 1-10 / entinostat days 3, 10 |
| 5ACAlso: 5-azacitidine | Drug | A: 5AC days 1-10 / entinostat days 3, 10, B: 5AC days 1-10 / entinostat days 10,17 |
| Entinostat days 10,17Also: MS275Given orally on days 10, 17 | Drug | B: 5AC days 1-10 / entinostat days 10,17 |
Primary outcome
To estimate the major response rate in patients with AML who are ≥ 60 years old and unable to tolerate or decline cytotoxic chemotherapy or patients who have relapsed despite one prior regimen.
Time frame: Up to 15 cycles (420 days)
To estimate the overall response rate following treatment in patients with AML ≥ 60 years old who are unable to tolerate or decline cytotoxic chemotherapy or those who have relapsed despite one prior regimen.
Time frame: Up to 15 cycles (420 days)
5 secondary outcomes
To identify changes in gene promoter methylation and gene expression in response to combination therapy and compare the dynamics and kinetics of these alterations in promoter methylation and gene re-expression in the two different dosing schedules.
Time frame: Up to 15 cycles (420 days)
To evaluate the effect of entinostat on the induction of hyperacetylation of histones from peripheral blood and/or bone marrow samples.
Time frame: Up to 15 cycles (420 days)
To evaluate changes in DNA damage in response to combination therapy using gammaH2AX determination by western blotting.
Time frame: Up to 15 cycles (420 days)
To evaluate the pharmacodynamics of 5AC and entinostat when given at either dosing schedule and to evaluate these in relation to pharmacokinetic and clinical outcomes.
Time frame: Up to 15 cycles (420 days)
To evaluate duration of response.
Time frame: Up to 15 cycles (420 days)
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 28 May 2024.
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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.
Hetty Carraway, MD principal investigator
Cleveland Clinic Taussig Cancer institute, Case Comprehensive Cancer Center
4 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 Entinostat days 3, 10, 5AC and Entinostat days 10,17 in people with Acute Myeloid Leukemia, run by Hetty Carraway at 4 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 18 inclusion and 15 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: status not confirmed, as of the sponsor's last update on 28 May 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 4 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?
Results are not available in the source record.
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?
To estimate the major response rate in patients with AML who are ≥ 60 years old and unable to tolerate or decline cytotoxic chemotherapy or patients who have relapsed despite one prior regimen. (time frame: Up to 15 cycles (420 days)). The study lists 2 primary outcomes in all. 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 28 May 2024.