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
Randomized Phase 2 Study of Daunorubicin and Cytarabine Liposome + Pomalidomide Versus Daunorubicin and Cytarabine Liposome in Newly Diagnosed AML With MDS-Related Changes
3 other identifiers
interventional
50
1 country
11
Brief 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. 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.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P25-P50 for phase_2
Started Aug 2022
Typical duration for phase_2
11 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
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Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
March 16, 2021
CompletedFirst Posted
Study publicly available on registry
March 17, 2021
CompletedStudy Start
First participant enrolled
August 24, 2022
CompletedPrimary Completion
Last participant's last visit for primary outcome
November 21, 2024
CompletedResults Posted
Study results publicly available
December 19, 2025
CompletedStudy Completion
Last participant's last visit for all outcomes
April 25, 2026
CompletedDecember 19, 2025
December 1, 2025
2.2 years
March 16, 2021
November 21, 2025
December 9, 2025
Conditions
Outcome Measures
Primary Outcomes (1)
Rate of Complete Response (CR)/Complete Response With Incomplete Hematologic Recovery (CRi)
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)
Up to 5 years
Incidence of Adverse Events
Up to 30 days after last dose, up to 2 years
Complete Response (CR) Without Minimal Residual Disease (MRD)
Up to 5 years
Event-free Survival
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)
From CR/CRi until relapse or death, assessed at 2 years
- +3 more secondary outcomes
Study Arms (2)
Arm A (daunorubicin and cytarabine liposome, pomalidomide)
EXPERIMENTALINDUCTION: 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 COMPARATORINDUCTION: 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.
Interventions
Undergo collection of blood samples
Undergo bone marrow aspirate and biopsy
Given IV
Given PO
Eligibility Criteria
You may qualify if:
- 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:
- Complex karyotype (3 or more unrelated chromosomal abnormalities in the absence of other class-defining recurrent genetic abnormalities as defined by WHO or ICC)
- /del(7q)
- Del(5q)/t(5q)/add(5q)
- i(17q)
- /add(17p) or del(17p)
- Del(20q)
- /del(13q)
- Del(11q)
- Del(12p)/t(12p)/add(12p)
- +24 more criteria
You may not qualify if:
- 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
- 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)
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (11)
Emory University Hospital/Winship Cancer Institute
Atlanta, Georgia, 30322, United States
University of Kansas Cancer Center
Kansas City, Kansas, 66160, United States
University of Kansas Hospital-Westwood Cancer Center
Westwood, Kansas, 66205, United States
Johns Hopkins University/Sidney Kimmel Cancer Center
Baltimore, Maryland, 21287, United States
NYP/Weill Cornell Medical Center
New York, New York, 10065, United States
UNC Lineberger Comprehensive Cancer Center
Chapel Hill, North Carolina, 27599, United States
Wake Forest University Health Sciences
Winston-Salem, North Carolina, 27157, United States
University of Cincinnati Cancer Center-UC Medical Center
Cincinnati, Ohio, 45219, United States
Ohio State University Comprehensive Cancer Center
Columbus, Ohio, 43210, United States
University of Cincinnati Cancer Center-West Chester
West Chester, Ohio, 45069, United States
University of Virginia Cancer Center
Charlottesville, Virginia, 22908, United States
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Intervention Hierarchy (Ancestors)
Results Point of Contact
- Title
- Dr. Joshua F. Zeidner
- Organization
- University of North Carolina, Lineberger Comprehensive Cancer Center
Study Officials
- PRINCIPAL INVESTIGATOR
Joshua F Zeidner
Ohio State University Comprehensive Cancer Center LAO
Publication Agreements
- PI is Sponsor Employee
- No
- Restriction Type
- LTE60
- Restrictive Agreement
- Yes
Study Design
- Study Type
- interventional
- Phase
- phase 2
- Allocation
- RANDOMIZED
- Masking
- NONE
- Purpose
- TREATMENT
- Intervention Model
- PARALLEL
- Sponsor Type
- NIH
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
March 16, 2021
First Posted
March 17, 2021
Study Start
August 24, 2022
Primary Completion
November 21, 2024
Study Completion
April 25, 2026
Last Updated
December 19, 2025
Results First Posted
December 19, 2025
Record last verified: 2025-12
Data Sharing
- IPD Sharing
- Will share
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.