Recruiting

Phase 1

Phase 2

CPX-351Phase IB/II of CPX-351 for Relapse Prevention in AML

Sponsor:

Georgetown University

Code:

NCT04990102

Conditions

Acute Myeloid Leukemia (AML) in Remission

Eligibility Criteria

Sex: All

Age: 18+

Healthy Volunteers: Not accepted

Interventions

CPX-351

Study Details

Brief summary:

This is a phase IB/II study with a 3+3 dose de-escalation study design. Patients will continue maintenance treatment with CPX-351 for 6 cycles on D1 and D3, as long as patient remains in CR. The dose de-escalation will be one dose given on D1 only, every 28 days pending toxicity. The maximum tolerated dose will be used for the phase II expansion portion of the study.

Conditions

Acute Myeloid Leukemia (AML) in Remission

Study ID

NCT04990102

Start date

May 22, 2023

Status verified date

Mar, 2026

Completion date

Dec, 2026

Anticipated

Primary completion date

Dec, 2026

Anticipated

Eligibility Criteria

Sex: All

Age: 18+

Healthy Volunteers: Not accepted

Inclusion Criteria:

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

Exclusion Criteria:

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.
Unresolved toxicities from prior anti-cancer therapy, defined as having not resolved to Common Terminology Criteria for Adverse Event (CTCAE, version 4.03), grade ≤1, or to the levels dictated in the inclusion/exclusion criteria with the exception of alopecia.
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))

Study Design

Enrollment

24 participants

Anticipated

Intervention Model

Sequential

Primary purpose

Treatment

Interventions and Outcome Measures

Arms

experimental: CPX-351

Dose Level 1: CPX-351 administered through intravenou infusion on Day 1 and Day 3 of 28 day cycle for 6 cycles or Dose Level -1: CPX-351 administered through intravenous infusion on Day 1 of each 28 day cycle for 6 cycles.

Interventions

CPX-351

Daunorubicin 8.8mg/m2 + cytarabine 20mg/m2

Primary outcome measure

Maximum tolerate dose (Phase 1) [ Time Frame: 1 cycle (28 day cycle) ]
Inicidence of Treatment Emergent Adverse events (Phase 2) [ Time Frame: 6 cycles (28 day cycles) ]

Central Contacts and Locations

Locations

Georgetown Lombardi Comprehensive Cancer Center

Recruiting

Washington D.C., District of Columbia, United States, 20007

Contacts

Courtney Brown, RN

202-687-0354 courtney.brown@georgetown.edu

Principal Investigator:

Kimberley Doucette, MD

John Theurer Cancer Center at Hackensack University Medical Center

Recruiting

Hackensack, New Jersey, United States, 07601

Contacts

Oncology Clinical Research Referral Office

551-996-1777 OncologyResearchReferral@hmhn.org

Principal Investigator:

James McCloskey, MD

University of Pennsylvania

Recruiting

Philadelphia, Pennsylvania, United States, 19104

Contacts

Fatme Chaloub

215-906-8435 Fatme.Chaloub@pennmedicine.upenn.edu

Principal Investigator:

Catherine Lai, MD

More Information

Sponsor

Georgetown University

Last update posted

Mar 23, 2026

Last verified

Mar, 2026

Keywords

Acute Myeloid Leukemia
Remission

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-08. This information was provided to ClinicalTrials.gov by Georgetown University on 2026-03-23.