Acute Myeloid LeukemiaPhase 2NCT04802161
Comparing the Addition of an Anti-Cancer Drug, Pomalidomide, to the Usual Chemotherapy Treatment (Daunorubicin and Cytarabine Liposome) in Newly Diagnosed Acute Myeloid Leukemia With Myelodysplastic Syndrome-Related Changes
A Phase 2 study testing Biospecimen Collection, Bone Marrow Aspiration and Biopsy and Liposome-encapsulated Daunorubicin-Cytarabine in people with Acute Myeloid Leukemia and Acute Myeloid Leukemia Post Cytotoxic Therapy, run by National Cancer Institute (NCI) at 11 sites in United States.
Trial summary
This phase II trial studies the effect of adding pomalidomide to usual chemotherapy treatment (daunorubicin and cytarabine liposome) in treating patients with newly diagnosed acute leukemia with myelodysplastic syndrome-related changes. Read more about this study
- Who is it for?
- Ages 18 to 75 years
- Conditions the study is for
- Acute Myeloid Leukemia, Acute Myeloid Leukemia Post Cytotoxic Therapy, Acute Myeloid Leukemia With Multilineage Dysplasia
- What is being tested?
- Biospecimen Collection, Bone Marrow Aspiration and Biopsy and Liposome-encapsulated Daunorubicin-Cytarabine
- Type of treatment
- Injection or infusion, Surgery or procedure
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 49 participants
- Where
- 11 sitesUnited States
- Study dates
- 24 Aug 2022to 25 Apr 2027
- 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 75 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Pathological confirmation of AML as defined by histologic, morphologic, or cytological evidence/confirmation of >= 20% blasts in bone marrow aspirate and/or biopsy
- Must meet criteria for t-AML or AML with MRC as defined by the 5th Edition of the World Health Organization (WHO) Classification of Myeloid Neoplasms or the International Consensus Classification (ICC) of Myeloid Neoplasms. Patients must meet one of the following criteria:
- Therapy-related AML (AML derived from prior chemotherapy or radiation therapy)
- AML originating from prior hematologic malignancy (MDS, CMML, or MPN)
- AML with myelodysplasia-related cytogenetic abnormalities:
- One of the following cytogenetic abnormalities:
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- Complex karyotype (3 or more unrelated chromosomal abnormalities in the absence of other class-defining recurrent genetic abnormalities as defined by WHO or ICC)
- -7/del(7q)
- Del(5q)/t(5q)/add(5q)
- +8
- i(17q)
- -17/add(17p) or del(17p)
- Del(20q)
- -13/del(13q)
- Del(11q)
- Del(12p)/t(12p)/add(12p)
- idicX(q13)
- AML with myelodysplasia-related mutations: Must have a mutation in one of the following genes:
- ASXL1
- BCOR
- EZH2
- RUNX1
- SF3B1
- SRSF2
- STAG2
- U2AF1
- ZRSR2
- No prior treatment for AML other than cytoreductive doses of hydroxyurea or leukapheresis
- Age >= 18 and =< 75 years on day of signing informed consent are eligible who are planned for intensive chemotherapy. Because no dosing or adverse event data are currently available on the use of pomalidomide in combination with daunorubicin and cytarabine liposome in patients < 18 years of age, children are excluded from this study. Patients > 75 years are not candidates for intensive chemotherapy with daunorubicin and cytarabine liposome
- Eastern Cooperative Oncology Group (ECOG) performance status of 0-2 (Karnofsky >= 60%)
- Total bilirubin =< 1.5 institutional upper limit of normal (ULN) unless due to leukemic infiltration, Gilbert's Syndrome, or hemolysis
- Aspartate aminotransferase (AST) (serum glutamic-oxaloacetic transaminase [SGOT])/alanine aminotransferase (ALT) (serum glutamate pyruvate transaminase [SGPT]) =< 5 x institutional ULN
- Creatinine >= 30 ml/min creatinine clearance by Cockcroft-gault
- Left ventricular ejection fraction (LVEF) >= 50%
- Human immunodeficiency virus (HIV)-infected patients on effective anti-retroviral therapy with undetectable viral load within 6 months are eligible for this trial
- For patients with evidence of chronic hepatitis B virus (HBV) infection, the HBV viral load must be undetectable on suppressive therapy, if indicated
- Patients with a history of hepatitis C virus (HCV) infection must have been treated and cured with undetectable HCV viral load. For patients with HCV infection who are currently on treatment, they are eligible if they have an undetectable HCV viral load
- Patients with known history or current symptoms of cardiac disease, or history of treatment with cardiotoxic agents, should have a clinical risk assessment of cardiac function using the New York Heart Association Functional Classification. To be eligible for this trial, patients should be class 2B or better
- Females of childbearing potential (FCBP), defined as a female who: 1) has reached menarche at some point, 2) has not undergone a hysterectomy or bilateral oophorectomy; or 3) has not been naturally postmenopausal for at least 24 consecutive months (i.e., has had menses at any time in the preceding 24 consecutive months) must have a negative pregnancy test 72 hours prior to the start of study therapy. For FCBPs in Arm A, they must have a negative serum or urine pregnancy test with a sensitivity of at least 25 mIU/mL within 10 - 14 days prior to and again within 24 hours of starting pomalidomide 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 starting pomalidomide. 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 vasectomy. All patients must be counseled at a minimum of every 28 days about pregnancy precautions and risks of fetal exposure
- Ability to understand and the willingness to sign a written informed consent document. Participants with impaired decision-making capacity (IDMC) who have a legally-authorized representative (LAR) and/or family member available will also be eligible
Who cannot join
- Patients with Wilson's Disease or Copper-related metabolic disorders
- Absolute blast count > 30 x 10^9/L (cytoreduction with leukapheresis or hydroxyurea can be used to achieve absolute blast count < 30 x 10^9/L prior to day 1 of treatment)
- Cumulative daunorubicin lifetime exposure > 330 mg/m^2 and > 180 mg/m^2 with prior mediastinal radiation therapy
- Patients with known active central nervous system leukemia should be excluded from this clinical trial because they often develop progressive neurologic dysfunction that would confound the evaluation of neurologic and other adverse events. Patients receiving intrathecal chemotherapy prophylaxis should receive pomalidomide >= 3 days after administration
- Patients with uncontrolled intercurrent illness including, but not limited to, active and uncontrolled infection, symptomatic congestive heart failure, unstable angina pectoris, and cardiac arrhythmia. Patients with infection under active treatment and controlled with antibiotics are eligible
- Known additional malignancy (with the exception of prior hematologic malignancies that have transformed to AML) that is active and/or progressive requiring treatment; exceptions include basal cell or squamous cell skin cancer, in situ cervical cancer or patients receiving maintenance treatments without active disease (for example, hormonal therapy for breast cancer or prostate cancer or other adjuvant chemotherapy approaches). Anti-cancer therapy as above should be discontinued > 72 hours prior to day 1 of treatment
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- Patients with psychiatric illness/social situations that would limit compliance with study requirements
- Receipt of prior allogeneic stem cell transplant
- Administration of any therapy for MDS, CMML, or MPN (conventional or unconventional) must be completed by 2 weeks prior to treatment with daunorubicin and cytarabine liposome. Use of strong CYP1A2 inhibitors should be avoided
- Patients who are receiving any other investigational agents
- History of allergic reactions attributed to compounds of similar chemical or biologic composition to pomalidomide (e.g. lenalidomide, thalidomide) or daunorubicin and cytarabine liposome or their excipients
- Development of erythema nodosum if characterized by a desquamating rash while taking thalidomide, lenalidomide, or similar drugs in the past
- Pregnant women are excluded from this study because pomalidomide is an immunomodulatory agent with the potential for teratogenic or abortifacient effects. Because there is an unknown but potential risk for adverse events in nursing infants secondary to treatment of the mother with pomalidomide, breastfeeding should be discontinued if the mother is treated with pomalidomide. These potential risks may also apply to other agents used in this study. Women of childbearing potential must be willing to undergo pregnancy testing
- Any other medical condition that in the opinion of investigator would place patient at increased risk for toxicity during pomalidomide treatment (i.e. history of recurrent or serious thromboembolic events such as massive pulmonary embolism)
Trial locations and contacts
11 sites in 1 country.
11 sites in 1 country
United States11 sites
Emory University Hospital/Winship Cancer Institute
Atlanta, Georgia, 30322
Johns Hopkins University/Sidney Kimmel Cancer Center
Baltimore, Maryland, 21287
UNC Lineberger Comprehensive Cancer Center
Chapel Hill, North Carolina, 27599
University of Virginia Cancer Center
Charlottesville, Virginia, 22908
University of Cincinnati Cancer Center-UC Medical Center
Cincinnati, Ohio, 45219
Ohio State University Comprehensive Cancer Center
Columbus, Ohio, 43210
University of Kansas Cancer Center
Kansas City, Kansas, 66160
NYP/Weill Cornell Medical Center
New York, New York, 10065
University of Cincinnati Cancer Center-West Chester
West Chester, Ohio, 45069
University of Kansas Hospital-Westwood Cancer Center
Westwood, Kansas, 66205
Wake Forest University Health Sciences
Winston-Salem, North Carolina, 27157
About this study
Official title: Randomized Phase 2 Study of Daunorubicin and Cytarabine Liposome + Pomalidomide Versus Daunorubicin and Cytarabine Liposome in Newly Diagnosed AML With MDS-Related Changes
This phase II trial studies the effect of adding pomalidomide to usual chemotherapy treatment (daunorubicin and cytarabine liposome) in treating patients with newly diagnosed acute leukemia with myelodysplastic syndrome-related changes. Pomalidomide may stop the growth of blood vessels, stimulate the immune system, and kill cancer cells. Chemotherapy drugs, such as daunorubicin and cytarabine liposome, work in different ways to stop the growth of cancer cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. Adding pomalidomide to chemotherapy treatment with daunorubicin and cytarabine liposome may be effective in improving some treatment outcomes in patients with newly diagnosed acute leukemia with myelodysplastic syndrome-related changes.
Read the detailed description
PRIMARY OBJECTIVES:
- To establish recommended phase 2 dose (RP2D) of pomalidomide after liposome-encapsulated daunorubicin-cytarabine (daunorubicin and cytarabine liposome) induction.
II. To compare the rate of overall complete response (CR)/complete response with incomplete hematologic recovery (CRi) with daunorubicin and cytarabine liposome + pomalidomide versus daunorubicin and cytarabine liposome alone in newly diagnosed acute myeloid leukemia (AML) with preexisting myelodysplastic syndrome (MDS), chronic myelomonocytic leukemia (CMML), or myeloproliferative neoplasm (MPN); therapy-related AML (t-AML); or AML with myelodysplasia-related changes (MRC) based on cytogenetics or morphologic dysplasia.
SECONDARY OBJECTIVES:
- To evaluate and compare rates of CR (full hematologic recovery) between daunorubicin and cytarabine liposome + pomalidomide versus daunorubicin and cytarabine liposome alone in newly diagnosed AML with preexisting MDS, CMML or MPN, t-AML or AML with MRC.
II. To evaluate and compare toxicities (including treatment-related mortality) of daunorubicin and cytarabine liposome + pomalidomide versus daunorubicin and cytarabine liposome alone in newly diagnosed AML with preexisting MDS, CMML or MPN, t-AML, or AML with MRC based on cytogenetics or morphologic dysplasia.
III. To detect and compare the presence of minimal residual disease (MRD) by flow cytometry in those who achieve CR/CRi with daunorubicin and cytarabine liposome + pomalidomide versus daunorubicin and cytarabine liposome alone in newly diagnosed AML with preexisting MDS, CMML or MPN, t-AML, or AML with MRC based on cytogenetics or morphologic dysplasia.
IV. To compare median event-free survival (EFS) of daunorubicin and cytarabine liposome + pomalidomide versus daunorubicin and cytarabine liposome alone in newly diagnosed AML with preexisting MDS, CMML or MPN, t-AML, or AML with MRC based on cytogenetics or morphologic dysplasia.
- To compare median overall survival (OS) of daunorubicin and cytarabine liposome + pomalidomide versus daunorubicin and cytarabine liposome alone in newly diagnosed AML with preexisting MDS, CMML or MPN, t-AML, or AML with MRC based on cytogenetics or morphologic dysplasia.
VI. To compare median and 2-year disease-free survival (DFS) after CR/CRi with daunorubicin and cytarabine liposome + pomalidomide versus daunorubicin and cytarabine liposome alone in newly diagnosed AML with preexisting MDS, CMML or MPN, t-AML, or AML with MRC based on cytogenetics or morphologic dysplasia.
VII. To compare rates of allogeneic stem cell transplant (SCT) after daunorubicin and cytarabine liposome + pomalidomide versus daunorubicin and cytarabine liposome alone in newly diagnosed AML with preexisting MDS, CMML or MPN, t-AML, or AML with MRC based on cytogenetics or morphologic dysplasia.
EXPLORATORY OBJECTIVES:
- To assess for molecular biomarkers, Aiolos expression, and immune correlates of response with daunorubicin and cytarabine liposome + pomalidomide versus daunorubicin and cytarabine liposome alone in newly diagnosed AML with preexisting MDS, CMML or MPN, t-AML, or AML with MRC based on cytogenetics or morphologic dysplasia.
II. To assess for differences in MRD by molecular based platforms in daunorubicin and cytarabine liposome + pomalidomide versus daunorubicin and cytarabine liposome alone in newly diagnosed AML with preexisting MDS, CMML or MPN, t-AML, or AML with MRC based on cytogenetics or morphologic dysplasia.
OUTLINE: Patients are randomized to 1 of 2 arms.
ARM A:
INDUCTION: Patients receive liposome-encapsulated daunorubicin-cytarabine intravenously (IV) over 90 minutes on days 1, 3, and 5 and then pomalidomide orally (PO) once daily (QD) beginning between days 21-30 for 14 days in the absence of disease progression or unacceptable toxicity. Patients who do not respond, may receive a second cycle of liposome-encapsulated daunorubicin-cytarabine IV over 90 minutes on days 1 and 3 in the absence of disease progression or unacceptable toxicity.
CONSOLIDATION: Patients who achieve CR/CRi receive liposome-encapsulated daunorubicin-cytarabine IV over 90 minutes on days 1 and 3. Treatment repeats every 21 days for 2 cycles in the absence of disease progression and unacceptable toxicity. Patients also undergo bone marrow aspirate and biopsy and collection of blood samples throughout all phases of the trial.
ARM B:
INDUCTION: Patients receive liposome-encapsulated daunorubicin-cytarabine intravenously (IV) over 90 minutes on days 1, 3, and 5 in the absence of disease progression or unacceptable toxicity. Patients who do not respond, may receive a second cycle of liposome-encapsulated daunorubicin-cytarabine IV over 90 minutes on days 1 and 3 in the absence of disease progression or unacceptable toxicity.
CONSOLIDATION: Patients who achieve CR/CRi receive liposome-encapsulated daunorubicin-cytarabine IV over 90 minutes on days 1 and 3. Treatment repeats every 21 days for 2 cycles in the absence of disease progression and unacceptable toxicity. Patients also undergo bone marrow aspirate and biopsy and collection of blood samples throughout all phases of the trial.
After completion of study treatment, patients are followed up for 30 days, then up to 5 years after the start of induction therapy or until death, whichever occurs first.
- Conditions
- Acute Myeloid Leukemia, Acute Myeloid Leukemia Post Cytotoxic Therapy, Acute Myeloid Leukemia With Multilineage Dysplasia, Chronic Myelomonocytic Leukemia, Myelodysplastic Syndrome, Myeloproliferative Neoplasm
- Being tested
- Biospecimen Collection, Bone Marrow Aspiration and Biopsy, Liposome-encapsulated Daunorubicin-Cytarabine, Pomalidomide
- Medical subject headings
- Leukemia, Myeloid, Acute, Leukemia, Myelomonocytic, Chronic, Myelodysplastic Syndromes, Myeloproliferative Disorders
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Biospecimen CollectionProcedure or surgery
Also called Biological Sample Collection, Biospecimen Collected, Sample Collection, Specimen Collection
Undergo collection of blood samples
Bone Marrow Aspiration and BiopsyProcedure or surgery
Undergo bone marrow aspirate and biopsy
Liposome-encapsulated Daunorubicin-CytarabineDrug
Also called CPX 351, CPX-351, CPX351, Cytarabine and Daunorubicin Liposomal
Given IV
PomalidomideDrug
Also called 4-Aminothalidomide, Actimid, CC 4047, CC-4047
Given PO
| Study group | Role | What they receive |
|---|---|---|
| Arm A (daunorubicin and cytarabine liposome, pomalidomide) | Experimental | INDUCTION: Patients receive liposome-encapsulated daunorubicin-cytarabine IV over 90 minutes on days 1, 3, and 5 and then pomalidomide PO QD beginning between days 21-30 for 14 days in the absence of disease progression or unacceptable toxicity. Patients who do not respond, may receive a second cycle of liposome-encapsulated daunorubicin-cytarabine IV over 90 minutes on days 1 and 3 in the absence of disease progression or unacceptable toxicity.
CONSOLIDATION: Patients who achieve CR/CRi receive liposome-encapsulated daunorubicin-cytarabine IV over 90 minutes on days 1 and 3. Treatment repeats every 21 days for 2 cycles in the absence of disease progression and unacceptable toxicity. Patients also undergo bone marrow aspirate and biopsy and collection of blood samples throughout all phases of the trial. Receives: Procedure: Biospecimen Collection; Procedure: Bone Marrow Aspiration and Biopsy; Drug: Liposome-encapsulated Daunorubicin-Cytarabine; Drug: Pomalidomide |
| Arm B (daunorubicin and cytarabine liposome) | Active comparator | INDUCTION: Patients receive liposome-encapsulated daunorubicin-cytarabine IV over 90 minutes on days 1, 3, and 5 in the absence of disease progression or unacceptable toxicity. Patients who do not respond, may receive a second cycle of liposome-encapsulated daunorubicin-cytarabine IV over 90 minutes on days 1 and 3 in the absence of disease progression or unacceptable toxicity.
CONSOLIDATION: Patients who achieve CR/CRi receive liposome-encapsulated daunorubicin-cytarabine IV over 90 minutes on days 1 and 3. Treatment repeats every 21 days for 2 cycles in the absence of disease progression and unacceptable toxicity. Patients also undergo bone marrow aspirate and biopsy and collection of blood samples throughout all phases of the trial. Receives: Procedure: Biospecimen Collection; Procedure: Bone Marrow Aspiration and Biopsy; Drug: Liposome-encapsulated Daunorubicin-Cytarabine |
What is being measured
The primary outcome is the main question the study is designed to answer.
Rate of Complete Response (CR)/Complete Response With Incomplete Hematologic Recovery (CRi)
Measured: Up to 2 years
Secondary outcomes (8)
CR With Full Hematologic Recovery (Absolute Neutrophil Count > 1 x 10^9/L and Platelets > 100 x 10^9/L)
Will be assessed and compared with both arms A and B.
Measured: Up to 5 years
Incidence of Adverse Events
Will be assessed by Common Terminology Criteria for Adverse Events (CTCAE) version (v.) 5.0 and compared descriptively between Arms A and B. Serious adverse events are denoted by "[SAE]".
Measured: Up to 30 days after last dose, up to 2 years
Complete Response (CR) Without Minimal Residual Disease (MRD)
Will assess CR without MRD by flow cytometry via Hematologics, Inc. compare descriptively between Arms A and B.
Measured: Up to 5 years
Event-free Survival
Will be compared between Arms A and B.
Measured: From day 1 of liposome-encapsulated daunorubicin-cytarabine until no response is achieved, relapse or death, will be assessed for up to 5 years
Disease-free Survival (DFS)
Will be compared between Arms A and B.
Measured: From CR/CRi until relapse or death, assessed at 2 years
Disease-free Survival
Will be compared between Arms A and B.
Measured: From CR/CRi until relapse or death, assessed up to 5 years
Overall Survival (OS)
Will be compared between Arms A and B.
Measured: From randomization until death or last follow-up, assessed up to 5 years
Rate of Allogeneic Stem Cell Transplantation
Will be compared between Arms A and B.
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 posted17 Mar 2021
- Study start24 Aug 2022
- Primary completion21 Nov 2024
- Study completion25 Apr 2027 (estimated)
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 Acute Myeloid LeukemiaStudies of the same condition, open and under way.
- More trials run by National Cancer Institute (NCI)The 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
- 49 participants (actual)
Sponsor and investigators
- Lead sponsor
- National Cancer Institute (NCI)
- Sponsor type
- US National Institutes of Health
- Responsible party
- Sponsor
- Investigators
- Joshua F Zeidner, Principal investigator, Ohio State University Comprehensive Cancer Center LAO
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04802161
- Sponsor's protocol ID
- NCI-2021-01961
- Other IDs
- NCI-2021-01961, 10434, 10434, UM1CA186712
- Status last verified
- May 2026
- First submitted
- 16 Mar 2021
- Last updated
- 30 Jun 2026
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Yes: NCI is committed to sharing data in accordance with NIH policy. For more details on how clinical trial data is shared, access the link to the NIH data sharing policy page.
Study at a glance
From the registry record, last updated by the sponsor on 30 Jun 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Randomized Phase 2 Study of Daunorubicin and Cytarabine Liposome + Pomalidomide Versus Daunorubicin and Cytarabine Liposome in Newly Diagnosed AML With MDS-Related Changes
- ClinicalTrials.gov ID
- NCT04802161
- Acronym / sponsor ID
- NCI-2021-01961
- Conditions
- Acute Myeloid Leukemia, Acute Myeloid Leukemia Post Cytotoxic Therapy, Acute Myeloid Leukemia With Multilineage Dysplasia, Chronic Myelomonocytic Leukemia, Myelodysplastic Syndrome, Myeloproliferative Neoplasm
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 49 participants (actual)
- Lead sponsor
- National Cancer Institute (NCI)US National Institutes of Health
- Study start
- 24 Aug 2022 (actual)
- Primary completion
- 21 Nov 2024 (actual)
- Study completion
- 25 Apr 2027 (estimated)
- First posted
- 17 Mar 2021 (actual)
- Results
- Posted 19 Dec 2025
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This phase II trial studies the effect of adding pomalidomide to usual chemotherapy treatment (daunorubicin and cytarabine liposome) in treating patients with newly diagnosed acute leukemia with myelodysplastic syndrome-related changes. Pomalidomide may stop the growth of blood vessels, stimulate the immune system, and kill cancer cells. Chemotherapy drugs, such as daunorubicin and cytarabine liposome, work in different ways to stop the growth of cancer cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. Adding pomalidomide to chemotherapy treatment with daunorubicin and cytarabine liposome may be effective in improving some treatment outcomes in patients with newly diagnosed acute leukemia with myelodysplastic syndrome-related changes.
Detailed description
PRIMARY OBJECTIVES:
- To establish recommended phase 2 dose (RP2D) of pomalidomide after liposome-encapsulated daunorubicin-cytarabine (daunorubicin and cytarabine liposome) induction.
II. To compare the rate of overall complete response (CR)/complete response with incomplete hematologic recovery (CRi) with daunorubicin and cytarabine liposome + pomalidomide versus daunorubicin and cytarabine liposome alone in newly diagnosed acute myeloid leukemia (AML) with preexisting myelodysplastic syndrome (MDS), chronic myelomonocytic leukemia (CMML), or myeloproliferative neoplasm (MPN); therapy-related AML (t-AML); or AML with myelodysplasia-related changes (MRC) based on cytogenetics or morphologic dysplasia.
SECONDARY OBJECTIVES:
- To evaluate and compare rates of CR (full hematologic recovery) between daunorubicin and cytarabine liposome + pomalidomide versus daunorubicin and cytarabine liposome alone in newly diagnosed AML with preexisting MDS, CMML or MPN, t-AML or AML with MRC.
II. To evaluate and compare toxicities (including treatment-related mortality) of daunorubicin and cytarabine liposome + pomalidomide versus daunorubicin and cytarabine liposome alone in newly diagnosed AML with preexisting MDS, CMML or MPN, t-AML, or AML with MRC based on cytogenetics or morphologic dysplasia.
III. To detect and compare the presence of minimal residual disease (MRD) by flow cytometry in those who achieve CR/CRi with daunorubicin and cytarabine liposome + pomalidomide versus daunorubicin and cytarabine liposome alone in newly diagnosed AML with preexisting MDS, CMML or MPN, t-AML, or AML with MRC based on cytogenetics or morphologic dysplasia.
IV. To compare median event-free survival (EFS) of daunorubicin and cytarabine liposome + pomalidomide versus daunorubicin and cytarabine liposome alone in newly diagnosed AML with preexisting MDS, CMML or MPN, t-AML, or AML with MRC based on cytogenetics or morphologic dysplasia.
- To compare median overall survival (OS) of daunorubicin and cytarabine liposome + pomalidomide versus daunorubicin and cytarabine liposome alone in newly diagnosed AML with preexisting MDS, CMML or MPN, t-AML, or AML with MRC based on cytogenetics or morphologic dysplasia.
VI. To compare median and 2-year disease-free survival (DFS) after CR/CRi with daunorubicin and cytarabine liposome + pomalidomide versus daunorubicin and cytarabine liposome alone in newly diagnosed AML with preexisting MDS, CMML or MPN, t-AML, or AML with MRC based on cytogenetics or morphologic dysplasia.
VII. To compare rates of allogeneic stem cell transplant (SCT) after daunorubicin and cytarabine liposome + pomalidomide versus daunorubicin and cytarabine liposome alone in newly diagnosed AML with preexisting MDS, CMML or MPN, t-AML, or AML with MRC based on cytogenetics or morphologic dysplasia.
EXPLORATORY OBJECTIVES:
- To assess for molecular biomarkers, Aiolos expression, and immune correlates of response with daunorubicin and cytarabine liposome + pomalidomide versus daunorubicin and cytarabine liposome alone in newly diagnosed AML with preexisting MDS, CMML or MPN, t-AML, or AML with MRC based on cytogenetics or morphologic dysplasia.
II. To assess for differences in MRD by molecular based platforms in daunorubicin and cytarabine liposome + pomalidomide versus daunorubicin and cytarabine liposome alone in newly diagnosed AML with preexisting MDS, CMML or MPN, t-AML, or AML with MRC based on cytogenetics or morphologic dysplasia.
OUTLINE: Patients are randomized to 1 of 2 arms.
ARM A:
INDUCTION: Patients receive liposome-encapsulated daunorubicin-cytarabine intravenously (IV) over 90 minutes on days 1, 3, and 5 and then pomalidomide orally (PO) once daily (QD) beginning between days 21-30 for 14 days in the absence of disease progression or unacceptable toxicity. Patients who do not respond, may receive a second cycle of liposome-encapsulated daunorubicin-cytarabine IV over 90 minutes on days 1 and 3 in the absence of disease progression or unacceptable toxicity.
CONSOLIDATION: Patients who achieve CR/CRi receive liposome-encapsulated daunorubicin-cytarabine IV over 90 minutes on days 1 and 3. Treatment repeats every 21 days for 2 cycles in the absence of disease progression and unacceptable toxicity. Patients also undergo bone marrow aspirate and biopsy and collection of blood samples throughout all phases of the trial.
ARM B:
INDUCTION: Patients receive liposome-encapsulated daunorubicin-cytarabine intravenously (IV) over 90 minutes on days 1, 3, and 5 in the absence of disease progression or unacceptable toxicity. Patients who do not respond, may receive a second cycle of liposome-encapsulated daunorubicin-cytarabine IV over 90 minutes on days 1 and 3 in the absence of disease progression or unacceptable toxicity.
CONSOLIDATION: Patients who achieve CR/CRi receive liposome-encapsulated daunorubicin-cytarabine IV over 90 minutes on days 1 and 3. Treatment repeats every 21 days for 2 cycles in the absence of disease progression and unacceptable toxicity. Patients also undergo bone marrow aspirate and biopsy and collection of blood samples throughout all phases of the trial.
After completion of study treatment, patients are followed up for 30 days, then up to 5 years after the start of induction therapy or until death, whichever occurs first.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Arm A (daunorubicin and cytarabine liposome, pomalidomide) | Experimental | Procedure: Biospecimen Collection, Procedure: Bone Marrow Aspiration and Biopsy, Drug: Liposome-encapsulated Daunorubicin-Cytarabine, Drug: PomalidomideINDUCTION: Patients receive liposome-encapsulated daunorubicin-cytarabine IV over 90 minutes on days 1, 3, and 5 and then pomalidomide PO QD beginning between days 21-30 for 14 days in the absence of disease progression or unacceptable toxicity. Patients who do not respond, may receive a second cycle of liposome-encapsulated daunorubicin-cytarabine IV over 90 minutes on days 1 and 3 in the absence of disease progression or unacceptable toxicity. CONSOLIDATION: Patients who achieve CR/CRi receive liposome-encapsulated daunorubicin-cytarabine IV over 90 minutes on days 1 and 3. Treatment repeats every 21 days for 2 cycles in the absence of disease progression and unacceptable toxicity. Patients also undergo bone marrow aspirate and biopsy and collection of blood samples throughout all phases of the trial. |
| Arm B (daunorubicin and cytarabine liposome) | Active comparator | Procedure: Biospecimen Collection, Procedure: Bone Marrow Aspiration and Biopsy, Drug: Liposome-encapsulated Daunorubicin-CytarabineINDUCTION: Patients receive liposome-encapsulated daunorubicin-cytarabine IV over 90 minutes on days 1, 3, and 5 in the absence of disease progression or unacceptable toxicity. Patients who do not respond, may receive a second cycle of liposome-encapsulated daunorubicin-cytarabine IV over 90 minutes on days 1 and 3 in the absence of disease progression or unacceptable toxicity. CONSOLIDATION: Patients who achieve CR/CRi receive liposome-encapsulated daunorubicin-cytarabine IV over 90 minutes on days 1 and 3. Treatment repeats every 21 days for 2 cycles in the absence of disease progression and unacceptable toxicity. Patients also undergo bone marrow aspirate and biopsy and collection of blood samples throughout all phases of the trial. |
Who may qualify
- Ages
- 18 to 75 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (40 listed)
- Pathological confirmation of AML as defined by histologic, morphologic, or cytological evidence/confirmation of >= 20% blasts in bone marrow aspirate and/or biopsy
- Must meet criteria for t-AML or AML with MRC as defined by the 5th Edition of the World Health Organization (WHO) Classification of Myeloid Neoplasms or the International Consensus Classification (ICC) of Myeloid Neoplasms. Patients must meet one of the following criteria:
- No prior treatment for AML other than cytoreductive doses of hydroxyurea or leukapheresis
- Age >= 18 and =< 75 years on day of signing informed consent are eligible who are planned for intensive chemotherapy. Because no dosing or adverse event data are currently available on the use of pomalidomide in combination with daunorubicin and cytarabine liposome in patients < 18 years of age, children are excluded from this study. Patients > 75 years are not candidates for intensive chemotherapy with daunorubicin and cytarabine liposome
- Eastern Cooperative Oncology Group (ECOG) performance status of 0-2 (Karnofsky >= 60%)
Key exclusion criteria (14 listed)
- Patients with Wilson's Disease or Copper-related metabolic disorders
- Absolute blast count > 30 x 10^9/L (cytoreduction with leukapheresis or hydroxyurea can be used to achieve absolute blast count < 30 x 10^9/L prior to day 1 of treatment)
- Cumulative daunorubicin lifetime exposure > 330 mg/m^2 and > 180 mg/m^2 with prior mediastinal radiation therapy
- Patients with known active central nervous system leukemia should be excluded from this clinical trial because they often develop progressive neurologic dysfunction that would confound the evaluation of neurologic and other adverse events. Patients receiving intrathecal chemotherapy prophylaxis should receive pomalidomide >= 3 days after administration
- Patients with uncontrolled intercurrent illness including, but not limited to, active and uncontrolled infection, symptomatic congestive heart failure, unstable angina pectoris, and cardiac arrhythmia. Patients with infection under active treatment and controlled with antibiotics are eligible
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 |
|---|---|---|
| Biospecimen CollectionAlso: Biological Sample Collection, Biospecimen Collected, Sample Collection, Specimen CollectionUndergo collection of blood samples | Procedure or surgery | Arm A (daunorubicin and cytarabine liposome, pomalidomide), Arm B (daunorubicin and cytarabine liposome) |
| Bone Marrow Aspiration and BiopsyUndergo bone marrow aspirate and biopsy | Procedure or surgery | Arm A (daunorubicin and cytarabine liposome, pomalidomide), Arm B (daunorubicin and cytarabine liposome) |
| Liposome-encapsulated Daunorubicin-CytarabineAlso: CPX 351, CPX-351, CPX351, Cytarabine and Daunorubicin LiposomalGiven IV | Drug | Arm A (daunorubicin and cytarabine liposome, pomalidomide), Arm B (daunorubicin and cytarabine liposome) |
| PomalidomideAlso: 4-Aminothalidomide, Actimid, CC 4047, CC-4047Given PO | Drug | Arm A (daunorubicin and cytarabine liposome, pomalidomide) |
Primary outcome
Rate of Complete Response (CR)/Complete Response With Incomplete Hematologic Recovery (CRi)
Time frame: Up to 2 years
8 secondary outcomes
CR With Full Hematologic Recovery (Absolute Neutrophil Count > 1 x 10^9/L and Platelets > 100 x 10^9/L)
Time frame: Up to 5 years
Incidence of Adverse Events
Time frame: Up to 30 days after last dose, up to 2 years
Complete Response (CR) Without Minimal Residual Disease (MRD)
Time frame: Up to 5 years
Event-free Survival
Time frame: From day 1 of liposome-encapsulated daunorubicin-cytarabine until no response is achieved, relapse or death, will be assessed for up to 5 years
Disease-free Survival (DFS)
Time frame: From CR/CRi until relapse or death, assessed at 2 years
Disease-free Survival
Time frame: From CR/CRi until relapse or death, assessed up to 5 years
Overall Survival (OS)
Time frame: From randomization until death or last follow-up, assessed up to 5 years
Rate of Allogeneic Stem Cell Transplantation
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 30 Jun 2026.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Individual participant dataSharing planned: Yes. NCI is committed to sharing data in accordance with NIH policy. For more details on how clinical trial data is shared, access the link to the NIH data sharing policy page.
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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.
Joshua F Zeidner principal investigator
Ohio State University Comprehensive Cancer Center LAO
11 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 Biospecimen Collection, Bone Marrow Aspiration and Biopsy and Liposome-encapsulated Daunorubicin-Cytarabine in people with Acute Myeloid Leukemia and Acute Myeloid Leukemia Post Cytotoxic Therapy, run by National Cancer Institute (NCI) at 11 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 40 inclusion and 14 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: under way, not looking for participants, as of the sponsor's last update on 30 Jun 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 11 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 19 Dec 2025). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Rate of Complete Response (CR)/Complete Response With Incomplete Hematologic Recovery (CRi) (time frame: Up to 2 years). 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 30 Jun 2026.