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No longer recruiting (closed or complete)Last updated: 8 August 2024

AAML1531: This phase III trial is evaluating different types of response-based chemotherapy in treating newly diagnosed Acute Myeloid Leukemia (AML) or Myelodysplastic syndrome in younger patients with Down syndromeRisk-Stratified Therapy for Acute Myeloid Leukemia in Down Syndrome

Trial purpose

Medical clipboardCancer treatment

Tumor type

Blood Cancers Haematological

Age

People<3

Trial acronym

AAML1531

Clinical summary

Summary

Drugs used in chemotherapy 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. Response-based chemotherapy separates patients into different risk groups and treats them according to how they respond to the first course of treatment (Induction I). Response-based treatment may be effective in treating acute myeloid leukemia or myelodysplastic syndrome in younger patients with Down syndrome while reducing the side effects. OUTLINE: INDUCTION I: Patients receive cytarabine intrathecally (IT) on day 1 and intravenously (IV) continuously over 96 hours, daunorubicin hydrochloride IV over 1-15 minutes, and thioguanine orally (PO) twice daily (BID) on days 1-4. Induction I continues for a minimum of 28 days. Patients are assigned to 1 of 2 treatment arms based on their MRD status after completion of Induction I. ARM A (STANDARD RISK): INDUCTION II: Patients receive cytarabine IV continuously over 96 hours, daunorubicin hydrochloride IV over 1-15 minutes, and thioguanine PO BID on days 1-4. Induction II continues for a minimum of 28 days. INDUCTION III: Patients receive cytarabine, daunorubicin hydrochloride, and thioguanine as in Induction II. Induction III continues for a minimum of 28 days. INTENSIFICATION I: Patients receive cytarabine IV continuously over 168 hours on days 1-7 and etoposide IV over 60-120 minutes on days 1-3. Intensification I continues for a minimum of 28 days. INTENSIFICATION II: Patients receive cytarabine and etoposide as in Intensification I. Intensification II continues for a minimum of 28 days. ARM B (HIGH RISK): INDUCTION II: Patients receive high dose cytarabine IV over 1-3 hours BID on days 1-4 and mitoxantrone hydrochloride IV over 15-30 minutes on days 3-6. Induction II continues for a minimum of 28 days. INTENSIFICATION I: Patients receive high dose cytarabine IV over 1-3 hours BID and etoposide IV over 60-120 minutes on days 1-5. Intensification I continues for a minimum of 28 days. INTENSIFICATION II: Patients receive high dose cytarabine IV over 3 hours BID on days 1, 2, 8, and 9. Patients also receive asparaginase or asparaginase Erwinia chrysanthemi (E. carotovora) intramuscularly (IM) or IV over 30 minutes on days 2 and 9. Intensification II continues for a minimum of 28 days. After completion of study treatment, patients are followed up at 1 month, monthly for 12 months, every 3 months for 12 months, every 6 months for 3 years, annually for 5 years, and then at relapse.

Conditions

This trial is treating people with Down syndrome who have been diagnosed with Acute Myeloid Leukaemia (AML) or Myelodysplastic syndrome

Eligibility

Inclusion

  • Patients must have constitutional trisomy 21 (Down syndrome) or trisomy 21 mosaicism (by karyotype or fluorescence in situ hybridization [FISH])

  • Patient has one of the following:

    • Patient has previously untreated de novo AML and meets the criteria for AML with >= 20% bone marrow blasts as set out in the World Health Organization (WHO) Myeloid Neoplasm classification

      • Attempts to obtain bone marrow either by aspirate or biopsy must be made unless clinically prohibitive; in cases where it is clinically prohibitive, peripheral blood with an excess of 20% blasts and in which adequate flow cytometric and cytogenetics/FISH testing is feasible can be substituted for the marrow exam at diagnosis
    • Patient has cytopenias and/or bone marrow blasts but does not meet the criteria for the diagnosis of AML (WHO Myeloid Neoplasm classification) because of < 20% marrow blasts and meets the criteria for a diagnosis of myelodysplastic syndrome (MDS)

    • For patients who do not meet criteria for AML or MDS as outlined above; patient has a history of transient myeloproliferative disorder (which may or may not have required chemotherapy intervention and:

      • Is > 8 weeks since resolution of transient myeloproliferative disease (TMD) with >= 5% blasts, OR
      • Has an increasing blast count (>= 5%) in serial bone marrow aspirates performed at least 4 weeks apart
  • Children who have previously received chemotherapy, radiation therapy or any anti-leukemic therapy are not eligible for this protocol, with the exception of cytarabine for the treatment of TMD

  • There are no minimal organ function requirements for enrollment on this study

    • Note: Previous cardiac repair with sufficient cardiac function is not an exclusion criteria
  • Each patient's 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 subjects research must be met

Exclusion

  • Patients with promyelocytic leukemia (French-American-British [FAB] M3)

  • Prior therapy

    • Patients =< 30 days from the last dose of cytarabine used for treatment of TMD

Exclusion

  • You have certain types of non-cancer medical conditions.
  • You have had certain treatments, surgical procedures or drugs.
Message

Clinical trials have complex eligibility criteria, and other criteria may apply for this trial. Ask your doctor about whether this trial could be right for you.

Participating hospitals

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More information

Trial Identifiers

Information on this page is partially produced from ClinicalTrials.gov *. View further details about this trial on the registry via the links below:

Trial sponsor

National Cancer Institute (NCI),Children's Oncology Group

Scientific Title

Risk-Stratified Therapy for Acute Myeloid Leukemia in Down Syndrome

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