Skip to main content

CPXSMN: CPX-351 in Pediatric Patients with Secondary Myeloid Neoplasms

A Prospective, Multicenter, Single-Arm Pilot Study of CPX-351 (VYXEOS) in Individuals < 22 Years with Secondary Myeloid Neoplasms


Leukemia / Lymphoma

Hematological Disorders

Diseases Treated:

Eligibility Overview:

  • 1–22 years old
  • Treatment-related or secondary MDS/AML 
  1. It is rare for pediatric patients treated for primary cancers or hematologic diseases to develop secondary myeloid malignancies such as myelodysplastic syndrome (MDS) or acute myeloid leukemia (AML). Those who do have second malignancies often have poor prognoses, with survival rates below 30%.

    Traditionally, patients with secondary myeloid neoplasms (SMNs) are treated with intensive chemotherapy followed by hematopoietic cell transplantation. However, most of these patients have already received significant amounts of chemotherapy or were chronically ill. Early mortality and morbidity are high.

    This phase 2 clinical trial will study the treatment of secondary AML and MDS with a new FDA-approved drug, CPX-351 (VYXEOS). This lipid-based vesicle contains 2 commonly used induction chemotherapy drugs, daunorubicin and cytarabine. The lipid delivery method minimizes cardiotoxicity until the drug reaches cancer cells in the bone marrow, spleen, and liver. This drug has already shown promise in some patients with SMNs.

    Primary Objectives

    This study will:

    • Examine the efficacy of CPX-351 for treatment of secondary MDS and AML
    • Examine side effects (toxicity) of this drug
    • Study how the body reacts to CPX-351
    • Study the biology and genetics of patients with SMNs
    • Estimate the 3-year survival of patients receiving 1 or 2 courses of CPX-351 followed by hematopoietic stem cell transplantation

    Eligibility Criteria

    Inclusion criteria include:

    • 1–22 years old
    • Treatment-related or secondary MDS/AML
    • Performance status for an Eastern Cooperative Oncology Group (ECOG) score of 0, 1 or 2 Karnowski for patients older than 16 and Lansky for patients younger than 16
    • Adequate renal, liver, cardiac, and CNS function
    • Recovered from acute toxic effects of prior chemotherapy, immunotherapy, HSCT, or radiotherapy

    Exclusion criteria include:

    • Down syndrome
    • Dyskeratosis congenita (telomeropathy)
    • Copper-related metabolic disorders
    • Philadelphia chromosome-positive myeloid neoplasms
    • Active uncontrolled infection
    • Receiving medications for left ventricular systolic dysfunction
    • Active HBV and HCV infections
    • Prior treatment with drugs that cause heart damage
    • Allergy to daunorubicin and/or cytarabine
    • Currently receiving another investigational drug
    • Pregnant or breastfeeding

    Study Sites

    St. Jude Children’s Research Hospital, Memphis, Tennessee
    Other collaborating sites

  2. About this study

    Some patients treated for cancer or blood diseases may get cancer again. This second cancer may come from the disease or its treatment.

    We want to study the effects of a new drug, CPX-351, to treat 2 kinds of secondary cancers:

    • Myelodysplastic syndrome (MDS)
    • Acute myeloid leukemia (AML)

    We aim to:

    • Study how well CPX-351 treats MDS and AML
    • Learn more about the side effects of CPX-351
    • Learn more about how the body reacts to CPX-351
    • Learn more about the biology and genetics of patients with MDS and AML

    In this study patients will be treated with:

    •  2 rounds of CPX-351

    A stem cell transplant or other therapies may follow this treatment. We will study DNA changes in your bone marrow and blood.

    Purpose of this clinical trial

    This study will help us understand the effects of CPX-351 treatment in patients with MDS and AML.


    To take part in this study, patients must:

    • Be 1–22 years old
    • Have 1 of the following:
      • MDS or AML as a second cancer  
      • MDS or AML that was caused by earlier treatments
    • Pass tests for kidney, liver, heart, brain, and spinal cord function
    • Have recovered from earlier treatments  

    Patients are not eligible for this study if they are:

    • Taking another new drug as part of a research study
    • Taking medicine for a heart problem known as left ventricular systolic dysfunction
    • Pregnant or breastfeeding
  3. CPXSMN  Quick View
    Sponsor St. Jude Children's Research Hospital Identifier NCT05656248
    Trial start date December 2022
    Estimated enrollment

    10 St. Jude patients, 15 patients from other institutions

    Study type Prospective Phase 2 

    Secondary myeloid neoplasms (SMNs), acute myeloid leukemia (AML) and myelodysplastic syndrome (MDS)

    Ages 1–22 years old
    Site Principal Investigator

    Raul Ribeiro, MD

    Study site St. Jude Children’s Research Hospital
    For a consultation or to discuss St. Jude Physician/Patient Referral Office

St. Jude Children’s Research Hospital
262 Danny Thomas Place
Memphis, TN 38105  USA
Voice: 1-888-226-4343 or 901-595-4055
24-Hour Emergency Access Pager: 1-800-349-4334

The above information is intended to provide only a basic description about a research protocol that may be currently active at St. Jude. The details made available here may not be the most up-to-date information on protocols used by St. Jude. To receive full details about a protocol and its status and or use at St. Jude, a physician must contact St. Jude directly.