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NCT07059975
UPDATE AML: UPdated Disease Monitoring And Treatment for Enhanced Outcomes for Pediatric AML
Conditions: Acute Myeloid Leukemia, Pediatric AML
Sex: All
Ages: 1 Month – 30 Years
Healthy volunteers: No
Phase: EARLY_PHASE1
Enrollment: 36
Sponsor: Baylor College of Medicine
Location: Texas Children's Cancer and Hematology Center Houston Texas
Summary
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.
Eligibility Criteria
Inclusion Criteria:
* 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:
\- Diagnosis
1. ≥ 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.
2. \< 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
* CEBPA bZIP mutation
* t(1;22)(p13.3;q13.1) RBM15(OTT) fusion
* t(7;12)(q36.3;p13.2) MNX1::ETV6
* t(8;16)(p11.2;p13.3) KAT6A::CREBBP - 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
* 11q23.3 partial tandem duplication (PTD) KMT2A PTD
3. 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).
4. 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 \7 days old, then the following laboratory evaluations must be re-checked within 48 hours prior to initiating therapy: bilirubin, ALT (SGPT) and serum creatinine. If the recheck is outside the limits of eligibility, the patient should be followed with periodic labs but may not receive protocol therapy until the bilirubin, ALT, and/or serum creatinine meet eligibility criteria. If \>14 days have passed from the planned start of protocol therapy and the bilirubin, ALT, and/or serum creatinine are still outside the limits of eligibility, the patient may not receive protocol therapy and rather will be considered a screen failure.
Adequate renal function defined as:
• An estimated creatinine clearance or GFR ≥ 60 ml/min/1.73m2. Any calculation method is acceptable, including the automatic creatinine clearance provided within the Epic EMR.
Adequate liver function defined as:
* A direct bilirubin \< 2 mg/dL
* ALT \
Source: ClinicalTrials.gov (NCT07059975). StuddyBuddy aggregates publicly available trial information.