UPDATE AML: UPdated Disease Monitoring And Treatment for Enhanced Outcomes for Pediatric AML

Recruiting now · Early Phase 1

Conditions studied: Acute Myeloid Leukemia, Pediatric AML

In brief

This research study investigates the tolerability of substituting two cycles of chemotherapy into the standard pediatric acute myeloid leukemia (AML) chemotherapy treatment regimen for patients with newly diagnosed AML at intermediate-risk (IR) and high-risk (HR) of relapse. The goal is to achieve similar or better survival with chemotherapy cycles that are intensive but less likely to cause long-term complications. Patients will enroll on this trial at the end of their first induction cycle. The two cycles to be substituted are: * "Ida-FLA" (idarubicin+fludarabine/cytarabine) as Induction 2 * "VIA" (venetoclax+idarubicin+cytarabine) as Intensification 1 of the HR treatment regimen, and Intensification 2 of the IR treatment backbone. Researchers will evaluate side effects and outcomes for up to three years after enrollment. Participants will also have the opportunity to participate in optional research studies including patient surveys and blood and bone marrow sample testing.

Key facts

Study ID

NCT07059975

Run by

Joanna Yi

People needed

36

Starts

2025-10-22

Expected to finish

2031-08-01

Last updated by the study team

2025-11-04

Who can join

Age: 0 and older, up to 30. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• Age Patients ≥ 1 months old to ≤ 30 years old are eligible
• Patients must be diagnosed with AML or myeloid sarcoma according to the 2022 WHO classification with or without extramedullary disease. Patients with AML must have 1 of the following at initial diagnosis:
• ≥ 20% bone marrow blasts
• 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 below:
• t(8;21)(q22;q22.1) RUNX1::RUNX1T1
• inv(16)(p13.1q22) or t(16;16)(p13.1;q22) CBFB::MYH11
• Translocation involving 11q23.3 KMT2A rearrangement
• t(6;9)(p23;q34.1) DEK::NUP214
• inv(3)(q21.3q26.2) or t(3;3)(q21.3;q26.2) MECOM rearrangement
• Megakaryoblastic with t(1;22)(p13.3;q13.3) RBM15::MRTFA
• Mutated NPM1
• t(5;11)(q35.3;p15.5) NUP98::NSD1
• inv(16)(p13.3q24.3) CBFA2T3::GLIS2
• t(11;12)(p15.5;p13.5) NUP98::KDM5A
• A complete blood count (CBC) documenting the presence of at least 1,000/µL circulating leukemic cells (blasts) if a bone marrow aspirate or biopsy cannot be performed (i.e., a WBC count ≥ 10,000/μL with ≥ 10% blasts or a WBC count of ≥ 5,000/μL with ≥ 20% blasts).
• Biopsy-proven myeloid sarcoma with or without bone marrow involvement.
• Note: patients with newly diagnosed AML, myelodysplasia-related (that are not from conditions listed in protocol section 4.2.1) ARE eligible while patients with therapy-related AML are excluded.
• Prior Therapy Patients must receive DA10+GO (Cytarabine days 1-10 + Daunorubicin days 1,3,5 [DA10] + Gemtuzumab ozogamcin [GO]) as prescribed in AAML1831 or the TXCH practice standard for Induction 1. Patients may have received any number of intrathecal treatments and have any CNS status at the time of enrollment.
• Performance Status Patients must have a performance status corresponding to Karnofsky/Lansky score >40. (Use Karnofsky for patients ≥16 years of age and Lanksy for patients <16 years of age.)
• Organ Function Requirements All laboratory studies to determine eligibility must be performed within 7 days prior to enrollment unless otherwise indicated. Laboratory values used to assess eligibility must be no older than seven (7) days at the start of therapy. Laboratory tests need not be repeated if therapy starts within seven (7) days of obtaining labs to assess eligibility. If a post-enrollment lab value is outside the limits of eligibility, or laboratory values are > seven (7) days old, then the following laboratory evaluations must be re-checked within 48 hours prior to initiating therapy: CBC with differential, bilirubin, ALT (SGPT) and serum creatinine. If the recheck is outside the limits of eligibility, the patient may not receive protocol therapy and will be considered off protocol therapy.
• Adequate renal function defined as:
• A creatinine clearance or GFR ≥ 60 ml/min/1.73m2
• Adequate liver function defined as:
• A direct bilirubin < 2 mg/dL

You may not qualify if…

• Patients with the following constitutional conditions are not eligible:
• Fanconi anemia
• Schwachman Diamond Syndrome
• Telomere Disorders
• Patients with constitutional trisomy 21 or with constitutional mosaicism of trisomy 21
• Germline predispositions known, or suspected by the treating physician, to increase risk of toxicity with AML therapy
• Therapy-related AML
• Patients with any of the following oncologic diagnoses are not eligible:
• Any concurrent malignancy
• Juvenile myelomonocytic leukemia
• Philadelphia chromosome positive AML
• Mixed phenotype acute leukemia
• Acute promyelocytic leukemia
• AML with FLT3 internal tandem duplication (FLT3-ITD)
• Pregnancy and Breastfeeding
• Female patients who are pregnant may not participate. A pregnancy test is required for female patients of childbearing potential.
• Lactating females who plan to breastfeed their infants are not eligible.
• Sexually active patients of reproductive potential who have not agreed to use an effective contraceptive method for the duration of their study participation are not eligible.

Where it is running

• Texas Children's Cancer and Hematology Center — Houston, Texas, United States (enrolling)

Full record on ClinicalTrials.gov

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