CPX-351 for the Treatment of Secondary Acute Myeloid Leukemia in Patients Younger Than 60 Years Old

NCT04269213 | Active Not Recruiting Phase 2 DMC FDA Drug

• 2 other identifiers: I 501719NCI-2019-08946
• Study Type: interventional
• Target: 21
• Locations: 1 country
• Sites: 4

Brief Summary

This phase II trial studies how well liposome-encapsulated daunorubicin-cytarabine (CPX-351) works in treating patients with secondary acute myeloid leukemia who are younger than 60 years old. Drugs used in chemotherapy, such as CPX-351, 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.

Conditions

Secondary Acute Myeloid Leukemia; Acute Myeloid Leukemia Arising From Previous Myelodysplastic Syndrome; Acute Myeloid Leukemia With Myelodysplasia-Related Changes; Therapy-Related Acute Myeloid Leukemia

Outcome Measures

Primary Outcomes (1)

• Complete response rate (morphological complete remission [CR] and incomplete blood count recovery [CRi])

  Defined by the International Working Group Criteria. Will be summarized using frequencies and relative frequencies.

  At day 45

Secondary Outcomes (5)

• CR + CRi duration

  Time from CR or CRi until relapse or last follow-up, assessed up to 5 years

• Event free survival

  Time from treating until disease progression/relapse, death due to disease, or last follow-up, assessed up to 5 years

• Overall survival

  Time from treatment until death due to any cause or last follow-up, assessed up to 5 years

• Allogeneic hematopoietic cell transplant rate

  Up to 5 years

• Incidence of adverse events

  Up to 5 years

Study Arms (1)

Treatment (CPX-351)

EXPERIMENTAL

INDUCTION: 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. RE-INDUCTION: Patients who do not achieve remission receive liposome-encapsulated daunorubicin-cytarabine IV over 90 minutes on days 1 and 3 in the absence of disease progression or unacceptable toxicity. CONSOLIDATION: Beginning 5-8 weeks after the start of the last induction, patients who achieve CR receive liposome-encapsulated daunorubicin-cytarabine IV over 90 minutes on days 1 and 3. Treatment repeats every 45 days for up to 2 cycles in the absence of disease progression or unacceptable toxicity.

Drug: Liposome-encapsulated Daunorubicin-Cytarabine

Interventions

Liposome-encapsulated Daunorubicin-Cytarabine DRUG

Given IV

Also known as: CPX-351, Cytarabine-Daunorubicin Liposome for Injection, Liposomal AraC-Daunorubicin CPX-351, Liposomal Cytarabine-Daunorubicin, Liposome-encapsulated Combination of Daunorubicin and Cytarabine, Vyxeos

Treatment (CPX-351)

Eligibility Criteria

• Age: 18 Years - 59 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Roswell Park Cancer Institute: lead
• Jazz Pharmaceuticals: collaborator

Study Sites (4)

University of Nebraska Medical Center

Omaha, Nebraska, 68198, United States

Location

Roswell Park Cancer Institute

Buffalo, New York, 14263, United States

Location

SUNY Upstate Medical Center

Syracuse, New York, 13210, United States

Location

Allegheny Health Network Cancer Institute - West Penn Hospital

Pittsburgh, Pennsylvania, 15224, United States

Location

MeSH Terms

Interventions

CPX-351; Injections; Cytarabine

Intervention Hierarchy (Ancestors)

Drug Administration Routes; Drug Therapy; Therapeutics; Cytidine; Pyrimidine Nucleosides; Pyrimidines; Heterocyclic Compounds, 1-Ring; Heterocyclic Compounds; Arabinonucleosides; Nucleosides; Nucleic Acids, Nucleotides, and Nucleosides

Study Officials

• Steven Green, MD

  Roswell Park Cancer Institute

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: February 11, 2020
• First Posted: February 13, 2020
• Study Start: July 29, 2021
• Primary Completion: April 10, 2025
• Study Completion (Estimated): January 29, 2027
• Last Updated: December 23, 2025

Record last verified: 2025-12

Locations

US (4)