Phase IB/II CPX-351 for Relapse Prevention in AML
This study is testing a drug called CPX-351 (a combination of daunorubicin and cytarabine) to see if it can help prevent acute myeloid leukemia (AML) from coming back after treatment. It's for adults aged 18 and older who have been newly diagnosed with AML and are currently in remission (meaning their cancer is not detectable). The study aims to find the safest dose of CPX-351 and to see how often side effects occur. Participants will receive CPX-351 for up to 6 cycles, as long as their AML remains in remission. The study plans to enroll 24 participants, but its current recruitment status is unclear.
- Study design
- This is a Phase IB/II study with a 3+3 dose de-escalation design, meaning the dose of CPX-351 may be lowered based on how participants tolerate it. It plans to enroll 24 participants.
- What's involved
- Participants will receive CPX-351 treatment for up to 6 cycles, with each cycle lasting 28 days. The drug will be given on day 1 and day 3 of each cycle, or possibly only on day 1 depending on the dose de-escalation.
- Compensation
- Not stated in the trial record.
- Follow-up
- The study will measure the incidence of treatment-emergent adverse events for up to 6 cycles (28-day cycles).
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NCT04990102
Phase IB/II of CPX-351 for Relapse Prevention in AML
Recruiting
PHASE1Ages 18+InterventionalTreatmentGeorgetown UniversityInvestigator-initiated
~24 participants
Updated 2026-03-23 on ClinicalTrials.gov
What's tested:CPX-351
At a glance
Recruiting sites
3 of 3 listed sites are recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
Maximum tolerate dose (Phase 1)
Measured over 1 cycle (28 day cycle)
+1 more outcome measured
Conditions
Where it's being run
3 sites across 3 statesDistrict of Columbia1
New Jersey1
Pennsylvania1
Study leadership
- Kimberley Doucette, MD · PRINCIPAL_INVESTIGATOR · Georgetown University
Who to contact
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Do you actually qualify for this trial?
Add a private profile and we'll compare every criterion below against your situation — and tell you which ones are met, uncertain, or excluding.
Eligibility criteria
Inclusion
Newly diagnosed patients \> 18 years of age
Patients must be in CR or CRh (complete remission with partial count recovery).
Must have received ANY induction treatment with standard consolidation or hypomethylating agent (HMA) + venetoclax, for up to 6 cycles or no more than 12 cycles of treatment.
Must be able to start therapy within 3 months of last documented CR
De novo or secondary AML/treatment related AML (non-M3) including AML with myelodysplasia-related changes (MRC), histologically confirmed
Patients must be ineligible for allogeneic BMT (for any reason including poor performance status, patient's preference, favorable AML not a candidate for transplant, or comorbidities and age precluding from transplant etc)
Cardiac ejection fraction ≥ 50% by transthoracic echocardiography or MUGA scan
Adequate hepatic and renal function defined as:
Serum aspartate transaminase (AST) or alanine transaminase (ALT) ≤ 3 x upper limit of normal (ULN)
Serum aspartate transaminase (AST) or alanine transaminase (ALT) ≤ 3 is permissible if due to disease.
Bilirubin ≤3 x ULN (unless bilirubin rise is due to Gilbert's syndrome or of non-hepatic origin)
Estimated Creatinine Clearance ≥30 ml/min (Cockcroft-Gault based on actual weight) (See Appendix A)
Eastern Cooperative Oncology Group (ECOG) performance status of ≤ 3 (Appendix A)
Female subjects who are of non-reproductive potential (i.e., post-menopausal by history - no menses for ≥1 year; OR history of hysterectomy; OR history of bilateral tubal ligation; OR history of bilateral oophorectomy). Female subjects of childbearing potential must have a negative serum pregnancy test upon study entry.
Male and female subjects who agree to use highly effective methods of birth control (e.g., condoms, implants, injectables, combined oral contraceptives, some intrauterine devices \[IUDs\], sexual abstinence, or sterilized partner) during the period of therapy and for at least 6 months after the last dose of study drug
Known bleeding disorders (e.g., von Willebrand's disease) or hemophilia
Known active infection with hepatitis C virus (HCV) or hepatitis B virus (HBV).
Subjects who are positive for hepatitis B core antibody, hepatitis B surface antigen, hepatitis C antibody, must have a negative polymerase chain reaction (PCR) result for the respective disease before enrollment. Those who are PCR positive will be excluded.
Any uncontrolled active systemic infection.
Any life-threatening illness, medical condition, or organ system dysfunction that, in the investigator's opinion, could compromise the subject's safety or put the study outcomes at undue risk.
Currently active, clinically significant cardiovascular disease, such as uncontrolled arrhythmia or Class 3 or 4 congestive heart failure as defined by the New York Heart Association Functional Classification; or a history of myocardial infarction, unstable angina, or acute coronary syndrome within 6 months prior to randomization.
Known CNS involvement by leukemia
Erythema multiforme, toxic epidermal necrolysis, or Stevens-Johnson syndrome
Lactating or pregnant.
Unwilling or unable to participate in all required study evaluations and procedures.
Unable to understand the purpose and risks of the study and to provide a signed and dated informed consent form (ICF) and authorization to use protected health information (in accordance with national and local subject privacy regulations).
Currently active, clinically significant hepatic impairment (≥ moderate hepatic impairment according to the Child Pugh classification (class B or C))
Exclusion
Prior allogeneic transplant
Previous cumulative anthracycline (doxorubicin equivalent) dose equal to or greater than 345 mg/m2, and for patients with prior mediastinal XRT, anthracycline dose equal to or greater than 295 mg/m2
Acute promyelocytic leukemia \[t(15;17)\]
If patient is unable to sign informed consent due to any serious medical condition, laboratory abnormality or psychiatric illness
Patients with evidence of uncontrolled current myocardial impairment (e.g. unstable ischemic heart disease, uncontrolled arrhythmia, symptomatic valvular dysfunction not controlled on medical therapy, uncontrolled hypertensive heart disease, and uncontrolled congestive heart failure)
History of Wilson's disease or other copper-related disorders
History of allergic reactions attributed to compounds of similar composition to cytarabine and daunorubicin or liposomal products
History of other malignancies, except:
Malignancy treated with curative intent and with no known active disease present for ≥ 3 years before the first dose of study drug and felt to be at low risk for recurrence by treating physician.
Adequately treated non-melanoma skin cancer or lentigo maligna without evidence of disease.
Adequately treated low risk prostate cancer or carcinoma in situ without evidence of disease.
What this trial measures
- Maximum tolerate dose (Phase 1)1 cycle (28 day cycle)
Number of subjects with Dose Limiting toxicities will be used to determine the recommended phase II dose of CPX-351 to be used in the maintenance setting for newly diagnosed AML in complete remission.
- Inicidence of Treatment Emergent Adverse events (Phase 2)6 cycles (28 day cycles)
To determine the safety, tolerability and toxicity of CPX-351 in the maintenance setting for newly diagnosed AML in complete remission by analyzing the incidence of treatment emergent adverse events reported.