Treatment of secondary acute myeloid leukemia in patients under 60 with CPX-351

A Phase II Study of CPX-351 in Younger Patients < 60 Years Old With Secondary Acute Myeloid Leukemia

Phase 2 Interventional Roswell Park Cancer Institute · NCT04269213

This study is testing a new treatment called CPX-351 to see if it can help younger patients with secondary acute myeloid leukemia feel better and live longer.

Quick facts

PhasePhase 2
Study typeInterventional
Enrollment46 (estimated)
Ages18 Years to 59 Years
SexAll
SponsorRoswell Park Cancer Institute Academic / other
Drugs / interventionschemotherapy
Locations4 sites (Omaha, Nebraska and 3 other locations)
Trial IDNCT04269213 on ClinicalTrials.gov

What this trial studies

This phase II trial evaluates the effectiveness of liposome-encapsulated daunorubicin-cytarabine (CPX-351) in treating secondary acute myeloid leukemia (AML) in patients younger than 60 years old. The study aims to determine the complete response rate, duration of response, event-free survival, overall survival, and the rate of patients proceeding to allogenic hematopoietic cell transplant. Participants will receive CPX-351 intravenously over a series of days, with follow-up assessments conducted for up to five years post-treatment. The trial includes both induction and consolidation phases to maximize treatment efficacy.

Who should consider this trial

Good fit: Ideal candidates are patients under 60 years old with newly diagnosed secondary acute myeloid leukemia, including therapy-related AML and AML with myelodysplastic syndrome.

Not a fit: Patients with primary acute myeloid leukemia or those with significant comorbidities that affect treatment tolerance may not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment could improve remission rates and overall survival for younger patients with secondary acute myeloid leukemia.

How similar studies have performed: Other studies have shown promise with similar chemotherapy approaches, but this specific combination in younger patients is being evaluated for the first time.

Eligibility criteria

Show full inclusion / exclusion criteria
Inclusion Criteria:

* Newly diagnosed:

  * Therapy-related acute myeloid leukemia (AML)
  * AML with antecedent myelodysplastic syndrome (MDS) or chronic myelomonocytic leukemia (CMML)
  * AML with MDS-related changes (as per World Health Organization \[WHO\])
* Have an Eastern Cooperative Oncology Group (ECOG) performance status of =\< 2
* Plasma creatinine =\< 1.5 x upper limit of normal (ULN)
* Total bilirubin \< 2.0 mg/dL
* Serum alanine aminotransferase and aspartate aminotransferase \< 3 x ULN
* Left ventricular ejection fraction by echocardiogram or multiple-gated acquisition \>= 50%
* Participants of child-bearing potential must agree to use adequate contraceptive methods (e.g., hormonal or barrier method of birth control; abstinence) prior to study entry. Should a woman become pregnant or suspect she is pregnant while she or her partner is participating in this study, she should inform her treating physician immediately
* Women of childbearing potential must have a negative serum or urine pregnancy test within 72 hours prior to enrollment and commit to two forms of birth control
* Men must use a latex condom during any sexual contact with women of childbearing potential
* Willing to adhere to protocol specific requirements
* Participant or legal representative must understand the investigational nature of this study and sign an Independent Ethics Committee/Institutional Review Board approved written informed consent form prior to receiving any study related procedure

Exclusion Criteria:

* Prior treatment of AML
* Known clinically active central nervous system (CNS) leukemia
* Core-binding factor leukemia
* Acute promyelocytic leukemia
* Uncontrolled other malignancy
* Prior anthracycline exposure \> 368 mg/m\^2 of daunorubicin or equivalent
* Cardiovascular disease resulting in heart failure (New York Heart Association class III or IV), unstable angina (angina symptoms at rest), or new onset angina (began within the last 3 months) or myocardial infarction within the past 6 months
* Hypersensitivity to cytarabine, daunorubicin, or liposomal drugs
* Known active HIV infection
* Known history of active hepatitis B or C infection
* Pre-existing liver disease (e.g. cirrhosis, chronic hepatitis B or C, nonalcoholic steatohepatitis, sclerosing cholangitis)
* Evidence of ongoing, uncontrolled systemic infection
* Pregnant or breastfeeding women
* Subject with concurrent severe and/or uncontrolled medical or psychiatric conditions that in the opinion of the investigator may impair the participation in the study or the evaluation of safety and/or efficacy
* History of Wilson disease or other copper-handling disorders
* Any condition which in the investigator's opinion deems the participant an unsuitable candidate to receive study drug

Where this trial is running

Omaha, Nebraska and 3 other locations

Study contacts

How to participate

  1. Review the eligibility criteria above with your treating physician.
  2. Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
  3. Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.
Conditions Acute Myeloid Leukemia Arising From Previous Myelodysplastic SyndromeAcute Myeloid Leukemia With Myelodysplasia-Related ChangesSecondary Acute Myeloid LeukemiaTherapy-Related Acute Myeloid Leukemia
Last reviewed 2026-06-13 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.