CPX-351 in Treating Patients With Newly Diagnosed, High-Risk Acute Myeloid Leukemia

NCT02286726 | Completed Phase 2 Results DMC FDA Drug

• 2 other identifiers: 2014-0548NCI-2014-02529
• Study Type: interventional
• Target: 56
• Locations: 1 country
• Sites: 1

Brief Summary

This phase II trial studies the best dose and how well liposomal cytarabine-daunorubicin CPX-351 (CPX-351) works in treating patients with newly diagnosed acute myeloid leukemia and who are at risk for not responding well to treatment. Liposomal cytarabine-daunorubicin CPX-351 combines two chemotherapy drugs that are known to help each other work better, and may work to stop the growth of cancer cells by blocking the cells from dividing.

Conditions

Acute Myeloid Leukemia; Acute Myeloid Leukemia Arising From Previous Myelodysplastic Syndrome; Secondary Acute Myeloid Leukemia

Outcome Measures

Primary Outcomes (1)

• Number of Participants With a Response

  Response is defined as Complete response (CR) or CR with incomplete blood count recovery (CRi) rate: Complete Remission (CR) is Bone marrow blasts \<5%; absence of blasts with Auer rods; absence of extramedullary disease; absolute neutrophil count \>1.0 x 10\^9/L (1000/μL); platelet count \>100 x 10\^9/L (100,000/μL). Complete Response with incomplete blood count recovery (CRi) is All CR criteria except for residual neutropenia (\<1.0 x 10\^9/L \[1000/μL\]) and/or thrombocytopenia (\<100 x 10\^9/L \[100,000/μL\]).

  Up to 8 weeks (after induction therapy)

Secondary Outcomes (1)

• Number of Participants Who Experienced Dose-limiting Toxicity (DLT)

  Up to day 60

Study Arms (3)

Arm I (lower-dose (50 units/m^2) CPX-351)

EXPERIMENTAL

Patients receive lower-dose liposomal cytarabine-daunorubicin CPX-351 IV over 90 minutes on days 1, 3, and 5 of a 28-day course. Patients with persistent disease may receive a second course with treatment on days 1 and 3.

Other: Laboratory Biomarker Analysis; Drug: Liposome-encapsulated Daunorubicin-Cytarabine

Arm II (intermediate-dose (75 units/m^2) CPX-351)

EXPERIMENTAL

Patients receive intermediate-dose liposomal cytarabine-daunorubicin CPX-351 IV over 90 minutes on days 1, 3, and 5. Patients with persistent disease may receive a second course with treatment on days 1 and 3.

Other: Laboratory Biomarker Analysis; Drug: Liposome-encapsulated Daunorubicin-Cytarabine

Arm III (standard-dose (100 units/m^2) CPX-351)

EXPERIMENTAL

Patients receive standard-dose liposomal cytarabine-daunorubicin CPX-351 IV over 90 minutes on days 1, 3, and 5. Patients with persistent disease may receive a second course with treatment on days 1 and 3.

Other: Laboratory Biomarker Analysis; Drug: Liposome-encapsulated Daunorubicin-Cytarabine

Interventions

Laboratory Biomarker Analysis OTHER

Correlative studies

Arm I (lower-dose (50 units/m^2) CPX-351); Arm II (intermediate-dose (75 units/m^2) CPX-351); Arm III (standard-dose (100 units/m^2) CPX-351)

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

Arm I (lower-dose (50 units/m^2) CPX-351); Arm II (intermediate-dose (75 units/m^2) CPX-351); Arm III (standard-dose (100 units/m^2) CPX-351)

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Ability to understand and voluntarily sign an informed consent form
• Pathological diagnosis of AML according to World Health Organization (WHO) criteria (with at least 20% blasts in the peripheral blood or bone marrow): newly diagnosed de novo AML; except for acute promyelocytic leukemia (APL); newly diagnosed secondary AML, defined as having a history of an antecedent hematologic disorder (myelodysplastic syndromes \[MDS\], myeloproliferative disease \[MPD\] or history of cytotoxic treatment for non-hematologic malignancy) or apparent de novo AML with MDS-associated karyotype
• Eastern Cooperative Oncology Group (ECOG) performance status 0-3
• Serum creatinine =\< 2.0 mg/dL
• Serum total bilirubin =\< 2.0 mg/dL
• Serum alanine aminotransferase \< 3 times the upper limit of normal (ULN); Note: If elevated liver enzymes are related to disease alanine aminotransferase (ALT) should be \< 5 times ULN
• To be considered at high risk for induction mortality patients must have 1 or 2 of the following risk factors (patients \>= 60 must have at least 1 risk factor, patients \< 60 must have at least 2 risk factors) present; at least one risk factor in every patient must be an AML-related factor:
• AML-related factors include:
• Antecedent hematologic disorder (AHD) (MDS, chronic myelomonocytic leukemia \[CMML\], or MPD) or history of exposure to cytotoxic chemotherapy \[therapy-related (t)-AML\]), or WHO-defined AML with MDS-related changes or apparent de novo AML with MDS-associated karyotype
• Unfavorable cytogenetics as defined by the European Leukemia Net
• Patient-related factors:
• Age \>= 70
• ECOG performance status (PS) \>= 2
• Co-morbidities:
• Serum creatinine \> 1.3 g/dL

You may not qualify if:

• Patients with history of second malignancy are eligible if they have documentation of disease stability, off therapy, based on computed tomography (CT) scan or other measures for the 6 months prior to entry in core
• Any serious medical condition or psychiatric illness that would prevent the patient from providing informed consent
• Chemotherapy or other investigational anticancer therapeutic drugs in the two weeks prior to study entry; in the event of rapidly proliferative disease, however, the use of hydroxyurea is permitted up to 24 hours before study entry in core
• Evidence of active central nervous system (CNS) leukemia
• Pregnant or lactating women
• Uncontrolled infection; to be eligible, patients receiving treatment for an infection (antibiotic, antifungal or antiviral treatment) must be afebrile (\< 38.3 degrees Celsius \[C\]) and without hemodynamic instability or dyspnea from pneumonia for \> 48 hours (hrs) prior to the start of induction therapy
• Hypersensitivity to cytarabine, daunorubicin or liposomal products
• History of Wilson's disease or other copper-metabolism disorder

Sponsors & Collaborators

• M.D. Anderson Cancer Center: lead
• National Cancer Institute (NCI): collaborator

Study Sites (1)

M D Anderson Cancer Center

Houston, Texas, 77030, United States

Location

Related Links

• University of Texas MD Anderson Cancer Center Website

MeSH Terms

Conditions

Leukemia, Myeloid, Acute

Interventions

CPX-351; Injections; Cytarabine

Condition Hierarchy (Ancestors)

Leukemia, Myeloid; Leukemia; Neoplasms by Histologic Type; Neoplasms; Hematologic Diseases; Hemic and Lymphatic Diseases

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

Results Point of Contact

• Title: Ghayas Issa C. MD./Assistant Professor
• Organization: The University of Texas MD Anderson Cancer Center

GCIssa@mdanderson.org

713-745-6798

Study Officials

• Ghayas Issa

  M.D. Anderson Cancer Center

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: Yes

Study Design

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

Study Record Dates

• First Submitted: October 31, 2014
• First Posted: November 10, 2014
• Study Start: May 4, 2015
• Primary Completion: January 22, 2020
• Study Completion: January 22, 2020
• Last Updated: March 4, 2026
• Results First Posted: January 14, 2021

Record last verified: 2025-04

Locations

US (1)