AMLPhase 2NCT03118466
Mitoxantrone, Etoposide, and Cytarabine (MEC) Plus Lenalidomide for Relapsed or Refractory Acute Myeloid Leukemia
A Phase 2 study testing Etoposide, Cytarabine and Lenalidomide in people with AML, run by Massachusetts General Hospital at 3 sites in United States.
Trial summary
This research study is evaluating how a drug called lenalidomide, given in combination with the standard chemotherapy regimen of Mitoxantrone, Etoposide, and Cytarabine, commonly referred to as MEC, works in individuals with either relapsed or refractory AML Read more about this study
- Who is it for?
- Ages 18 to 70 years
- Conditions the study is for
- AML
- What is being tested?
- Etoposide, Cytarabine and Lenalidomide
- Type of treatment
- Pill or oral medicine, Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 41 participants
- Where
- 3 sitesUnited States
- Study dates
- 25 Sep 2017to 18 Aug 2021
- 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 to 70 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Acute myelogenous leukemia diagnosed by WHO criteria with one of the following (patients with biphenotypic leukemia are eligible, provided that the treating physician determines an AML treatment regimen is appropriate)
- Primary refractory disease following > 1cycle of chemotherapy, (such as hypomethylating agent or induction chemotherapy)
- First relapse or higher. Patients with primary or secondary acute myelogenous leukemia are eligible.
- Age 18-70 years old
- LVEF > 50 %
- ECOG Performance status 0-2
Show all 20Show fewer
- Able to adhere to study schedule and other protocol requirements.
- Participants must have normal organ function as defined below, unless felt due to underlying disease and approved by the overall PI. Patients with Gilbert's disease may have total bilirubin up to < 3 x ULN.
- Creatinine < 2.0mg/dl
- Total bilirubin < 1.5 x ULN
- AST (SGOT) and ALT (SGPT) < 3 x ULN.
- Patients may receive hydroxyurea, steroids, or leukapheresis as necessary until Day 5 of treatment.
- Patients must give voluntary written informed consent and HIPAA authorization before performance of any study-related procedure not part of normal medical care, with the understanding that consent may be withdrawn by the subject at any time without prejudice to future medical care.
- Patients may have had prior treatment for MDS or AML, including prior lenalidomide for MDS or AML or another condition.
- Patient may have had prior autologous or allogeneic transplant (family member, unrelated donor, or cord blood) if there is at least 90 days between transplant and study entry.
- Patients may also have had donor lymphocyte infusion if there is at least 60 days between donor lymphocyte infusion and study entry.
- Patients on immunosuppression are also eligible.
- Females of childbearing potential (FCBP)† must have a negative serum or urine pregnancy test with a sensitivity of at least 50 mIU/mL prior to receiving treatment with lenalidomide, and must either commit to continued abstinence from heterosexual intercourse or begin TWO acceptable methods of birth control, one highly effective method and one additional effective method AT THE SAME TIME, at least 28 days before she starts taking lenalidomide. FCBP must also agree to ongoing pregnancy testing. Men must agree to use a latex condom during sexual contact with a FCBP even if they have had a successful vasectomy.
- Ability to understand and the willingness to sign a written informed consent document.
- All study participants must be registered into the mandatory Revlimid REMS ® program, and be willing and able to comply with the requirements of the REMs ® program. Females of reproductive potential must adhere to the scheduled pregnancy testing as required in the Revlimid REMS® program
Who cannot join
- Known hypersensitivity to thalidomide or lenalidomide (if applicable).
- The development of erythema nodosum if characterized by a desquamating rash while taking thalidomide or similar drugs.
- Known seropositive for human immunodeficiency virus (HIV). HIV testing is not required. Hepatitis testing is not required.
- Patients who have had a myocardial infarction within 6 months of enrollment or has New York Heart Association (NYHA) Class III or IV heart failure, uncontrolled angina, uncontrolled ventricular arrhythmias, or electrocardiographic evidence of acute ischemia or active conduction system abnormalities.
- Any serious medical condition laboratory abnormality or psychiatric illness that would prevent the subject from signing the consent form.
- Any condition, including laboratory abnormalities, that in the opinion of the investigator places the subject at unacceptable risk if he/she were to participate in the study or confounds the ability to interpret data from the study.
Show all 11Show fewer
- Patients with major surgery within 28 days prior to treatment.
- Patients with any serious medical or psychiatric illness that could, in the investigator's opinion, potentially interfere with the completion of treatment according to this protocol.
- Patient has received an investigational agent or cytotoxic chemotherapy (excluding hydroxyurea) within 7 days of study entry.
- Patients with acute promyelocytic leukemia.
- Females who are pregnant
Trial locations and contacts
3 sites in 1 country.
United States3 sites
Dana Farber Cancer Institute
Boston, Massachusetts, 02062
Beth Israel Deaconess Medical Center
Boston, Massachusetts, 02215
Massachusetts general Hospital
Boston, Massachusetts, 02215
About this study
Official title: Phase 2 Study of Mitoxantrone, Etoposide, and Cytarabine (MEC) Plus Lenalidomide for the Treatment of Adult Patients With Relapsed or Refractory Acute Myeloid Leukemia
This research study is evaluating how a drug called lenalidomide, given in combination with the standard chemotherapy regimen of Mitoxantrone, Etoposide, and Cytarabine, commonly referred to as MEC, works in individuals with either relapsed or refractory AML
Read the detailed description
This research study is a Phase II clinical trial. Phase II clinical trials test the safety and effectiveness of an investigational intervention to learn whether the intervention works in treating a specific disease. "Investigational" means that the intervention is being studied.
The FDA (the U.S. Food and Drug Administration) has not approved lenalidomide for this specific disease, but it has been approved for other uses, including for patients with multiple myeloma and some patients with myelodysplastic syndrome. This treatment is investigational because it is not approved by the FDA for patients with AML. Lenalidomide is a chemotherapy that also modulates the immune system, and is in a category of drugs called immunomodulatory drugs or IMIDs. Some research studies suggest that lenalidomide may be effective in patients with AML. Since the investigators know that many patients who receive MEC chemotherapy alone have less than desired response rates and overall shorter periods of remission (time free from leukemia) after treatment, the investigators are studying whether the addition of lenalidomide to MEC improves upon typical responses.
The combination of MEC (mitoxantrone, etoposide, and cytarabine) is a standard treatment option, commonly used for relapsed or refractory acute myeloid leukemia.
.
- Conditions
- AML
- Being tested
- Etoposide, Cytarabine, Lenalidomide, Mitoxantrone
- Keywords
- AML
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
EtoposideDrug
Also called Toposar
A Drug that interfere with the action of topoisomerase enzymes (topoisomerase I and II). Topoisomerase enzymes control the manipulation of the structure of DNA necessary for replication.
CytarabineDrug
Also called Depocyt
Cytarabine is an antimetabolite antineoplastic agent that inhibits the synthesis of DNA.
LenalidomideDrug
Also called REVLIMID
It may act by inhibiting the growth of new blood vessels (angiogenesis) in tumors, enhancing the status of the immune system, or decreasing cytokine and growth factor production
MitoxantroneDrug
Also called Novantrone
It interfere with cell reproduction
| Study group | Role | What they receive |
|---|---|---|
| Lenalidomide and MEC chemotherapy | Experimental | Lenalidomide is taken orally on a daily basis days 1-10. Mitoxantrone, Etoposide, and Cytarabine are administered intravenously on a daily basis for days 4 through 8 of the treatment. There is only one cycle of treatment in this study. Receives: Drug: Etoposide; Drug: Cytarabine; Drug: Lenalidomide; Drug: Mitoxantrone |
What is being measured
The primary outcome is the main question the study is designed to answer.
Complete Response Rate
Percentage of patients who have achieve CR or CRp after treatment.
- Morphologic Complete Remission (CR): Defined as morphologic leukemia-free state, including <5% blasts in Bone Marrow aspirate with marrow spicules, no persistent extramedullary disease, ANC >1000/mm3 and platelet count >100,000/mm3.
- Morphologic Complete Remission without platelet recovery (CRp): Defined as CR with the exception of platelet count < 100,000/mm3 (CRp).
Measured: up to 45 days
Secondary outcomes (6)
Number of Patients That Achieved ANC Recovery
The number of patients that achieved a neutrophil count of > 500/mm3 for 3 days within 45 of starting treatment
Measured: up to 45 days
Number of Patients That Achieved Platelet Recovery
The number of patients that achieved a stable platelet count > 20,000/mm3 for 3 days within 45 days of starting treatment
Measured: up to 45 days
Treatment-related Mortality
Cumulative number of deaths not related to persistent or relapsed leukemia during treatment within 50 days of the start of treatment.
Measured: 50 days
Transfusion Support: Number of Red Blood Cell and Platelet Transfusions
Number of red blood cell and platelet transfusions received within the first 50 days of treatment
Measured: 50 days
Overall Survival
Overall survival is defined as time from diagnosis of disease until date of death or censored on the last known date alive if patients are still alive.
Measured: Up to 3 years
Relapse-Free Survival
Relapse-Free Survival is defined as time from diagnosis of disease until date of relapse, death, or censored on the last known date alive if patients are still alive.Relapse is defined by morphological evidence of the original malignancy consistent with pre-treatment features.
Measured: Up to 3 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted18 Apr 2017
- Study start25 Sep 2017
- Primary completion29 Aug 2019
- Study completion18 Aug 2021
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 AMLStudies of the same condition, open and under way.
- More trials run by Massachusetts General HospitalThe 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
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 41 participants (actual)
Sponsor and investigators
- Lead sponsor
- Massachusetts General Hospital
- Sponsor type
- University, hospital or other
- Collaborators
- Celgene
- Responsible party
- Principal investigator, Andrew Mark Brunner, MD, Massachusetts General Hospital
- Investigators
- Andrew Brunner, MD, Principal investigator, Massachusetts General Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03118466
- Sponsor's protocol ID
- 16-574
- Status last verified
- Oct 2025
- First submitted
- 30 Mar 2017
- Last updated
- 15 Oct 2025
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 15 Oct 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phase 2 Study of Mitoxantrone, Etoposide, and Cytarabine (MEC) Plus Lenalidomide for the Treatment of Adult Patients With Relapsed or Refractory Acute Myeloid Leukemia
- ClinicalTrials.gov ID
- NCT03118466
- Acronym / sponsor ID
- 16-574
- Conditions
- AML
- Keywords
- AML
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 41 participants (actual)
- Lead sponsor
- Massachusetts General HospitalUniversity, hospital or other
- Collaborators
- Celgene
- Study start
- 25 Sep 2017 (actual)
- Primary completion
- 29 Aug 2019 (actual)
- Study completion
- 18 Aug 2021 (actual)
- First posted
- 18 Apr 2017 (actual)
- Results
- Posted 3 Feb 2021
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This research study is evaluating how a drug called lenalidomide, given in combination with the standard chemotherapy regimen of Mitoxantrone, Etoposide, and Cytarabine, commonly referred to as MEC, works in individuals with either relapsed or refractory AML
Detailed description
This research study is a Phase II clinical trial. Phase II clinical trials test the safety and effectiveness of an investigational intervention to learn whether the intervention works in treating a specific disease. "Investigational" means that the intervention is being studied.
The FDA (the U.S. Food and Drug Administration) has not approved lenalidomide for this specific disease, but it has been approved for other uses, including for patients with multiple myeloma and some patients with myelodysplastic syndrome. This treatment is investigational because it is not approved by the FDA for patients with AML. Lenalidomide is a chemotherapy that also modulates the immune system, and is in a category of drugs called immunomodulatory drugs or IMIDs. Some research studies suggest that lenalidomide may be effective in patients with AML. Since the investigators know that many patients who receive MEC chemotherapy alone have less than desired response rates and overall shorter periods of remission (time free from leukemia) after treatment, the investigators are studying whether the addition of lenalidomide to MEC improves upon typical responses.
The combination of MEC (mitoxantrone, etoposide, and cytarabine) is a standard treatment option, commonly used for relapsed or refractory acute myeloid leukemia.
.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Lenalidomide and MEC chemotherapy | Experimental | Drug: Etoposide, Drug: Cytarabine, Drug: Lenalidomide, Drug: MitoxantroneLenalidomide is taken orally on a daily basis days 1-10. Mitoxantrone, Etoposide, and Cytarabine are administered intravenously on a daily basis for days 4 through 8 of the treatment. There is only one cycle of treatment in this study. |
Who may qualify
- Ages
- 18 to 70 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (20 listed)
- Acute myelogenous leukemia diagnosed by WHO criteria with one of the following (patients with biphenotypic leukemia are eligible, provided that the treating physician determines an AML treatment regimen is appropriate)
- Age 18-70 years old
- LVEF > 50 %
- ECOG Performance status 0-2
- Able to adhere to study schedule and other protocol requirements.
Key exclusion criteria (11 listed)
- Known hypersensitivity to thalidomide or lenalidomide (if applicable).
- The development of erythema nodosum if characterized by a desquamating rash while taking thalidomide or similar drugs.
- Known seropositive for human immunodeficiency virus (HIV). HIV testing is not required. Hepatitis testing is not required.
- Patients who have had a myocardial infarction within 6 months of enrollment or has New York Heart Association (NYHA) Class III or IV heart failure, uncontrolled angina, uncontrolled ventricular arrhythmias, or electrocardiographic evidence of acute ischemia or active conduction system abnormalities.
- Any serious medical condition laboratory abnormality or psychiatric illness that would prevent the subject from signing the consent form.
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 |
|---|---|---|
| EtoposideAlso: ToposarA Drug that interfere with the action of topoisomerase enzymes (topoisomerase I and II). Topoisomerase enzymes control the manipulation of the structure of DNA necessary for replication. | Drug | Lenalidomide and MEC chemotherapy |
| CytarabineAlso: DepocytCytarabine is an antimetabolite antineoplastic agent that inhibits the synthesis of DNA. | Drug | Lenalidomide and MEC chemotherapy |
| LenalidomideAlso: REVLIMIDIt may act by inhibiting the growth of new blood vessels (angiogenesis) in tumors, enhancing the status of the immune system, or decreasing cytokine and growth factor production | Drug | Lenalidomide and MEC chemotherapy |
| MitoxantroneAlso: NovantroneIt interfere with cell reproduction | Drug | Lenalidomide and MEC chemotherapy |
Primary outcome
Complete Response Rate
Time frame: up to 45 days
Percentage of patients who have achieve CR or CRp after treatment. * Morphologic Complete Remission (CR): Defined as morphologic leukemia-free state, including <5% blasts in Bone Marrow aspirate with marrow spicules, no persistent extramedullary disease, ANC >1000/mm3 and platelet count >100,000/mm3. * Morphologic Complete Remission without platelet recovery (CRp): Defined as CR with the exception of platelet count < 100,000/mm3 (CRp).
6 secondary outcomes
Number of Patients That Achieved ANC Recovery
Time frame: up to 45 days
Number of Patients That Achieved Platelet Recovery
Time frame: up to 45 days
Treatment-related Mortality
Time frame: 50 days
Transfusion Support: Number of Red Blood Cell and Platelet Transfusions
Time frame: 50 days
Overall Survival
Time frame: Up to 3 years
Relapse-Free Survival
Time frame: Up to 3 years
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 15 Oct 2025.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Individual participant dataSharing planned: No
Related trials for AML
Not yet recruitingPhase 2
Revumenib, Azacitidine, and VENetoclax in Newly Diagnosed KMT2A-Rearranged AML
Leukemia Acute Myeloid
RecruitingPhase 4
Oral Azacitidine in Transplant-Eligible Patients With Acute Myeloid Leukemia (AML) Suffering From Health-Inequality
Acute Myeloid Leukemia
RecruitingPhase 1/2
The Lowest Effective Dose of Post-Transplantation Cyclophosphamide in Combination With Sirolimus and Mycophenolate Mofetil as Graft-Versus-Host Disease Prophylaxis After Reduced Intensity Conditioning and Peripheral Blood Stem Cell Transplantation
Peripheral Blood Stem Cell Transplantation, Hematopoietic Stem Cell Transplantation
Not yet recruitingObservational study
Risk Stratification Value of Deep NGS-MRD Detection in Acute Myeloid Leukemia Patients After Allo-HSCT
Measurable Residual Disease, Acute Myeloid Leukemia
RecruitingObservational study
Collecting Blood Samples From Patients With and Without Cancer to Evaluate Tests for Early Cancer Detection
Acute Lymphoblastic Leukemia, Acute Myeloid Leukemia
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.
Andrew Brunner, MD principal investigator
Massachusetts General Hospital
3 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 Etoposide, Cytarabine and Lenalidomide in people with AML, run by Massachusetts General Hospital at 3 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 20 inclusion and 11 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 Oct 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 3 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 3 Feb 2021). 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?
Complete Response Rate (time frame: up to 45 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 15 Oct 2025.