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Study for Newly Diagnosed AML in Children and Young Adults

This study compares standard chemotherapy to new treatments for children and young adults (up to 21 years old) with newly diagnosed acute myeloid leukemia (AML). Researchers are looking at liposome-encapsulated daunorubicin-cytarabine (CPX-351) and/or gilteritinib. CPX-351 combines two chemotherapy drugs, daunorubicin and cytarabine, in a way that might make them stay in the bone marrow longer and potentially reduce heart problems. The study aims to see if these new treatments improve event-free survival (how long patients live without their cancer returning or getting worse). You must have newly diagnosed AML to be eligible. The study plans to enroll 1186 participants, but its current status is unclear.

Study design
This interventional study plans to enroll 1186 participants. It compares different treatment arms for newly diagnosed AML.
What's involved
You would undergo blood sample collection, bone marrow aspiration, and bone marrow biopsy. Some participants may also undergo allogeneic hematopoietic stem cell transplantation.
Compensation
Not stated in the trial record.
Follow-up
Event-free survival will be measured for up to 3 years.

AI-generated from the public study record. Only the study team can confirm whether you're eligible — confirm details with them before making decisions.

NCT04293562

A Study to Compare Standard Chemotherapy to Therapy With CPX-351 and/or Gilteritinib for Patients With Newly Diagnosed AML With or Without FLT3 Mutations

Recruiting
PHASE3Ages 6–21InterventionalTreatment
Children's Oncology Group
~1,400 participants
Updated 2026-09-17 on ClinicalTrials.gov
What's tested:Allogeneic Hematopoietic Stem Cell TransplantationAsparaginase Erwinia chrysanthemiBiospecimen CollectionBone Marrow AspirationBone Marrow BiopsyComputed Tomography

At a glance

Recruiting sites
188 of 206 listed sites are recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
Event-free survival (EFS)
Measured over Up to 3 years

Conditions

Acute Myeloid Leukemia

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Where you'd take part

This study runs at 206 sites. They're the same protocol — you choose where, and that choice sets who your contact draft is addressed to.

  • AdventHealth Orlando

    Orlando, Floridastudy coordinator listed

    Recruiting

  • Advocate Children's Hospital-Oak Lawn

    Oak Lawn, Illinoisstudy coordinator listed

    Recruiting

  • Advocate Children's Hospital-Park Ridge

    Park Ridge, Illinoisstudy coordinator listed

    Recruiting

  • Albany Medical Center

    Albany, New Yorkstudy coordinator listed

    Recruiting

  • Alberta Children's Hospital

    Calgary, Alberta, Canadastudy coordinator listed

    Recruiting

  • Alfred I duPont Hospital for Children

    Wilmington, Delawarestudy coordinator listed

    Recruiting

  • Alliance for Childhood Diseases/Cure 4 the Kids Foundation

    Las Vegas, Nevadastudy coordinator listed

    Recruiting

  • Arkansas Children's Hospital

    Little Rock, Arkansasstudy coordinator listed

    Recruiting

Sites open and close at different times, so the status above is per site — it can differ from the study's overall status.

Study leadership

  • Todd M Cooper · PRINCIPAL_INVESTIGATOR · Children's Oncology Group

Who to contact

Site Public Contact
[email protected]
205-638-9285
Email the study team

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Eligibility criteria

Inclusion

All patients must be enrolled on APEC14B1 and consented to Eligibility Screening (Part A) prior to enrollment and treatment on AAML1831. Bone marrow and/or blood must have been submitted to the BPC via APEC14B1 for testing of FLT3 markers. Patients without samples submitted for FLT3 testing are not eligible
Patients must be at least 6 months of age and less than 22 years of age at the time of study enrollment
Patient must be newly diagnosed with de novo AML according to the 2016 World Health Organization (WHO) classification with or without extramedullary disease
Patient must have 1 of the following:
\>= 20% bone marrow blasts (obtained within 14 days prior to enrollment)
In cases where extensive fibrosis may result in a dry tap, blast count can be obtained from touch imprints or estimated from an adequate bone marrow core biopsy
\< 20% bone marrow blasts with one or more of the genetic abnormalities associated with childhood/young adult AML as provided in the protocol (sample obtained within 14 days prior to enrollment)
A complete blood count (CBC) documenting the presence of at least 1,000/uL (i.e., a white blood cell \[WBC\] count \>= 10,000/uL with \>= 10% blasts or a WBC count of \>= 5,000/uL with \>= 20% blasts) circulating leukemic cells (blasts) if a bone marrow aspirate or biopsy is pending or cannot be performed (performed within 7 days prior to enrollment)
ARM C: Patient must be \>= 2 years of age at the time of Late Callback
ARM C: Patient must have FLT3/ITD allelic ratio \> 0.1 as reported by Molecular Oncology
ARM C: Patient does not have any congenital long QT syndrome or congenital heart block
ARM C: Females of reproductive potential must agree to use effective contraception during treatment and for at least 6 months after the last dose of gilteritinib
ARM C: Lactating women must agree not to breastfeed during treatment with gilteritinib and for 2 months after the last dose of gilteritinib
ARM C: Males of reproductive potential must agree to use effective contraception during treatment and for at least 4 months after the last dose of gilteritinib
ARM D: Patient must be \>= 2 years of age at the time of Late Callback
ARM D: Patient must have one of the clinically relevant non-ITD FLT3 activating mutations as reported by Foundation Medicine
ARM D: Females of reproductive potential must agree to use effective contraception during treatment and for at least 6 months after the last dose of gilteritinib
ARM D: Lactating women must agree not to breastfeed during treatment with gilteritinib and for 2 months after the last dose of gilteritinib
ARM D: Males of reproductive potential must agree to use effective contraception during treatment and for at least 4 months after the last dose of gilteritinib
NEUROPSYCHOLOGICAL TESTING: Patient must be enrolled on Arm A or Arm B. Patients who transfer to Arm C or Arm D are not eligible
NEUROPSYCHOLOGICAL TESTING: Patient must be 5 years or older at the time of enrollment
NEUROPSYCHOLOGICAL TESTING: English-, French- or Spanish-speaking
NEUROPSYCHOLOGICAL TESTING: No known history of neurodevelopmental disorder prior to diagnosis of AML (e.g., Down syndrome, fragile X, William syndrome, mental retardation)
NEUROPSYCHOLOGICAL TESTING: No significant visual or motor impairment that would prevent computer use or recognition of visual test stimuli
All patients and/or their parents or legal guardians must sign a written informed consent
All institutional, Food and Drug Administration (FDA), and National Cancer Institute (NCI) requirements for human studies must be met

Exclusion

Fanconi anemia
Shwachman Diamond syndrome
Patients with constitutional trisomy 21 or with constitutional mosaicism of trisomy 21
Telomere disorders
Germline predispositions known, or suspected by the treating physician to increase risk of toxicity with AML therapy
Any concurrent malignancy
Juvenile myelomonocytic leukemia (JMML)
Philadelphia chromosome positive AML
Mixed phenotype acute leukemia
Acute promyelocytic leukemia
Acute myeloid leukemia arising from myelodysplasia
Therapy-related myeloid neoplasms
Patients with persistent cardiac dysfunction prior to enrollment, defined as ejection fraction (EF) \< 50% (preferred method Biplane Simpson's EF) or if EF unavailable, shortening fraction (SF) \< 24%. \*Note: if clinically safe and feasible, repeat echocardiogram is strongly advised in order to confirm cardiac dysfunction following clinical stabilization, particularly if occurring in the setting of sepsis or other transient physiologic stressor. If the repeat echocardiogram demonstrates an EF \>= 50%, the patient is eligible to enroll and may receive an anthracycline-containing Induction regimen
Administration of prior anti-cancer therapy except as outlined below:
Hydroxyurea
All-trans retinoic acid (ATRA)
Corticosteroids (any route)
Intrathecal therapy given at diagnosis
In particular, strong inducers of CYP3A4 and/or P-glycoprotein (P-gp) should be avoided from the time of enrollment until it is determined whether the patient will receive gilteritinib. Patients receiving gilteritinib will be required to avoid strong CYP3A4 inducers and/or strong P-gp inducers for the duration of the study treatment
Patients \< 8.3 kg at study enrollment are not eligible
Female patients who are pregnant since fetal toxicities and teratogenic effects have been noted for several of the study drugs. A pregnancy test is required for female patients of childbearing potential
Lactating females who plan to breastfeed their infants
Sexually active patients of reproductive potential who have not agreed to use an effective contraceptive method for the duration of their study participation
ARM D: Patient does not have any congenital long QT syndrome or congenital heart block

What this trial measures

  • Event-free survival (EFS)Up to 3 years

    The Kaplan-Meier method will be used to estimate 3-year EFS, defined as the time from study entry until induction failure, relapse, or death.