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

Trial statusRecruiting
Trial phasePhase 3
Trial typeInterventional
Biological sexAll
AgeUp to 21
SponsorChildren's Oncology Group

About this trial

This phase III trial compares standard chemotherapy to therapy with liposome-encapsulated daunorubicin-cytarabine (CPX-351) and/or gilteritinib for patients with newly diagnosed acute myeloid leukemia with or without FLT3 mutations. Drugs used in chemotherapy, such as daunorubicin, cytarabine, and gemtuzumab ozogamicin, 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. CPX-351 is made up of daunorubicin and cytarabine and is made in a way that makes the drugs stay in the bone marrow longer and could be less likely to cause heart problems than traditional anthracycline drugs, a common class of chemotherapy drug. Some acute myeloid leukemia patients have an abnormality in the structure of a gene called FLT3. Genes are pieces of DNA (molecules that carry instructions for development, functioning, growth and reproduction) inside each cell that tell the cell what to do and when to grow and divide. FLT3 plays an important role in the normal making of blood cells. This gene can have permanent changes that cause it to function abnormally by making cancer cells grow. Gilteritinib may block the abnormal function of the FLT3 gene that makes cancer cells grow. The overall goals of this study are, 1) to compare the effects, good and/or bad, of CPX-351 with daunorubicin and cytarabine on people with newly diagnosed AML to find out which is better, 2) to study the effects, good and/or bad, of adding gilteritinib to AML therapy for patients with high amounts of FLT3/ITD or other FLT3 mutations and 3) to study changes in heart function during and after treatment for AML. Giving CPX-351 and/or gilteritinib with standard chemotherapy may work better in treating patients with acute myeloid leukemia compared to standard chemotherapy alone.

Eligibility criteria

Qualifiers

All patients must be enrolled on APEC14B1 and consented to Eligibility Screening (Part A) prior to enrollment and treatment on AAML1831

Patients must be 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

>= 20% bone marrow blasts (obtained within 14 days prior to enrollment)

Disqualifiers

Fanconi anemia

Shwachman Diamond syndrome

Patients with constitutional trisomy 21 or with constitutional mosaicism of trisomy 21

Telomere disorders

Trial design

Treatments tested in this trial

  • Allogeneic Hematopoietic Stem Cell Transplantation
  • Asparaginase Erwinia chrysanthemi
  • Biospecimen Collection
  • Bone Marrow Aspiration
  • Bone Marrow Biopsy
  • Computed Tomography
  • Cytarabine
  • Daunorubicin Hydrochloride
  • Dexrazoxane Hydrochloride
  • Etoposide
  • Fludeoxyglucose F-18
  • Gemtuzumab Ozogamicin
  • Gilteritinib Fumarate
  • Liposome-encapsulated Daunorubicin-Cytarabine
  • Magnetic Resonance Imaging
  • Methotrexate
  • Mitoxantrone Hydrochloride
  • Positron Emission Tomography
  • Questionnaire Administration
  • Therapeutic Hydrocortisone

Treatment groups

1,186 Participants
are divided into 14 treatment groups

14

Treatment groups

See each treatment group below.

Sponsors and collaborators

Children's Oncology Group

Lead sponsor

National Cancer Institute (NCI)

Collaborator