NCT07059975

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

UPdated Disease Monitoring And Treatment for Enhanced Outcomes for Pediatric AML: A Pilot Trial

• Status: Recruiting
• Phase: EARLY_Phase 1
• Study type: Interventional
• Enrollment: 36 (estimated)
• Sponsor: Joanna Yi · Academic / Other
• Sex: All
• Age: 1 Month – 30 Years
• Healthy volunteers: Not accepted

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.

Detailed description

UPDATE AML is a research study that investigates the tolerability of substituting two cycles of chemotherapy into the standard pediatric acute myeloid leukemia (AML) chemotherapy treatment regimen for patients at intermediate-risk (IR) and high-risk (HR) of the leukemia coming back ("relapse"). Newly diagnosed patients will receive standard Induction 1 treatment off study. If they do not have a FLT3-ITD mutation, they will be eligible to complete treatment on the UPDATE AML study. Patients considered Low Risk will receive the standard treatments outside of the UPDATE AML trial but will be eligible for the non-treatment parts of the trial. IR and HR patients will receive a combination of idarubicin, fludarabine, cytarabine (Ida-FLA) as their 2nd cycle of chemotherapy ("Induction 2"). This combination has shown desirable anti-leukemic activity in children whose leukemia has relapsed but has not yet been studied extensively in children newly diagnosed with AML. Secondly the trial will administer the new drug venetoclax (FDA approved for adults with AML but not for pediatric patients) combined with idarubicin and cytarabine as the 3rd or 4th cycle (of the HR or IR regimens respectively). Texas Children's Hospital will be providing the venetoclax. The other major goal of UPDATE AML is to improve to detection of residual leukemia. Currently, the intensity of treatment for pediatric patients with AML depends on the genetic changes in the leukemia cells and their response to the first month of therapy. Of patients who have no detectable residual disease at the end of the first month, 30% of them will still relapse, indicating residual leukemia was still present, just not detected. Our study will develop individualized tests for each patient's genetic change which should increase detection of tiny amounts of residual leukemia. If our trial can successfully develop these individualized tests, future pediatric AML trials will incorporate these tests into use for all patients. The research study procedures include screening for eligibility study treatment, evaluation, administration of the substituted chemotherapy cycles, and follow-up visits. Participants will be offered the possibility to provide additional blood and bone marrow samples (to be collected only at times of regular medical examination). There are additional surveys patients and parents can participate in, to understand the challenges facing families undergoing pediatric AML treatment. Besides the 2 substituted chemotherapy cycles, patients will otherwise receive standard chemotherapy as their relapse risk indicates. Patients will be followed for approximately 3 years from enrollment. It is expected that about 36 people will take part in this research study.

Conditions

• Acute Myeloid Leukemia
• Pediatric AML

Interventions

Type	Name	Description
DRUG	Idarubicin Hydrochloride	Idarubicin is given in combination with fludarabine and cytarabine for Ida-FLA, and in combination with venetoclax and cytarabine for VIA.
DRUG	Fludarabine	Fludarabine is given in combination with idarubicin and cytarabine for Ida-FLA.
DRUG	Cytarabine (Ara-C)	Cytarabine is given in combination with other chemotherapy agents in every cycle and both arms.
DRUG	Venetoclax	Venetoclax is given in combination with idarubicin and cytarabine for VIA.
DRUG	Etoposide	Etoposide is given in combination with cytarabine for AE, as Intensification 1 for IR patients.
DRUG	Asparaginase Erwinia Chrysanthemi (recombinant)	Rylaze is given in combination with cytarabine for Intensification 3 for IR patients.
DRUG	Intrathecal triple	Methotrexate, hydrocortisone and cytarabine are combined into one preparation for intrathecal administration at multiple time points during treatment.
OTHER	SOC	Low-Risk Patients will receive Texas childern's Hospital practice standard for de novo AML.

Timeline

• Start date: 2025-10-22
• Primary completion: 2028-10-01
• Completion: 2031-08-01
• First posted: 2025-07-11
• Last updated: 2025-11-04

Locations

1 site across 1 country: United States

Regulatory

• FDA-regulated drug study