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Acute Myeloid Leukemia (AML) in RemissionPhase 1/2NCT04990102

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

A Phase 1/2 study testing CPX-351 in people with Acute Myeloid Leukemia (AML) in Remission, run by Georgetown University at 3 sites in United States.

Recruiting

Looking for participants now. Contact the study team or a site below to ask about taking part.

Sites and contacts
Official record
Search all trials

Trial 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. Read more about this study

Who is it for?
Ages 18 years and older
What is being tested?
CPX-351
Type of treatment
Injection or infusion
Could I receive a placebo?
No, everyone receives an active treatment
How many people
24 participants
Where
3 sitesUnited States
Study dates
22 May 2023to Dec 2026
Phase
Phase 1/2
Are there results?
Not yet: the study is under way

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 years and older
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • 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)
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  • 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

Who cannot join

  • 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
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  • 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))

Trial locations and contacts

3 sites in 1 country. Contact the study team or a site near you to ask about taking part.

United States3 sites, 3 recruiting

  • John Theurer Cancer Center at Hackensack University Medical Center

    Hackensack, New Jersey, 07601

    Oncology Clinical Research Referral Office, 551-996-1777, OncologyResearchReferral@hmhn.org

    James McCloskey, MD

    Recruiting

  • University of Pennsylvania

    Philadelphia, Pennsylvania, 19104

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

    Catherine Lai, MD

    Recruiting

  • Georgetown Lombardi Comprehensive Cancer Center

    Washington D.C., District of Columbia, 20007

    Courtney Brown, RN, 202-687-0354, courtney.brown@georgetown.edu

    Kimberley Doucette, MD

    Recruiting

Other trials in United States

About this study

Official title: Phase IB/II of CPX-351 as Maintenance Therapy in AML Patients Ineligible for Bone Marrow Transplantation

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.

Being tested
CPX-351
Keywords
Acute Myeloid Leukemia, Remission
Medical subject headings
Leukemia, Myeloid, Acute

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • CPX-351Drug

    Also called Vyxeos

    Daunorubicin 8.8mg/m2 + cytarabine 20mg/m2

Study groups in NCT04990102
Study groupRoleWhat they receive
CPX-351ExperimentalDose 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.
Receives: Drug: CPX-351

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Maximum tolerate dose (Phase 1)

    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.

    Measured: 1 cycle (28 day cycle)

  2. Inicidence of Treatment Emergent Adverse events (Phase 2)

    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.

    Measured: 6 cycles (28 day cycles)

Secondary outcomes (4)

  1. Incidence of Treatment Emergent Adverse Events (Phase 1)

    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.

    Measured: 6 cycles (28 day Cycle)

  2. Overall Survival (Phase 1 and 2)

    Determine the efficacy of CPX-351 as maintenance treatment in newly diagnosed AML patients who have achieved remission after induction treatment as measured by overall survival of subjects.

    Measured: 1 year after end of treatment

  3. Event free survival (Phase 1 and 2)

    Determine efficacy of CPX-351 as maintenance treatment in newly diagnosed AML patients who have achieved remission after induction treatment as measured by event free survival.

    Measured: 1 year after end of treatment

  4. Relapse Free Survival (Phase 1 and 2)

    Determine efficacy of CPX-351 as maintenance treatment in newly diagnosed AML patients who have achieved remission after induction treatment as measured by relapse free survival.

    Measured: 1 year after end of treatment

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted4 Aug 2021
  2. Study start22 May 2023
  3. Primary completionDec 2026 (estimated)
  4. Study completionDec 2026 (estimated)

Resources

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 1/2
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Sequential
Model description
Dose de-escalation of CPX-351
Masking
None (open label)
Enrolment
24 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT04990102
Sponsor's protocol ID
STUDY00003287
Status last verified
Mar 2026
First submitted
26 Jul 2021
Last updated
23 Mar 2026
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
No

Questions about this trial

What is this clinical trial studying?

A Phase 1/2 study testing CPX-351 in people with Acute Myeloid Leukemia (AML) in Remission, run by Georgetown University 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 15 inclusion and 24 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: looking for participants now, as of the sponsor's last update on 23 Mar 2026. 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 combined phase mean?

Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.

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?

This study is still under way. Results are not available in the source record yet.

How can a doctor refer a patient or contact the research team?

Each site lists its own contact: choose the nearest one under Trial locations. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Maximum tolerate dose (Phase 1) (time frame: 1 cycle (28 day cycle)). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT04990102.

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 23 Mar 2026.

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