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

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.

Conditions

• Acute Myeloid Leukemia
• Pediatric AML

Interventions

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.

Sponsors & Collaborators

• Baylor College of Medicine

  collaborator OTHER

• Joanna Yi

  lead OTHER

Study Design

Allocation

NON_RANDOMIZED

Purpose

TREATMENT

Masking

NONE

Model

PARALLEL

Eligibility

Min Age

1 Month

Max Age

30 Years

Sex

ALL

Healthy Volunteers

No

Timeline & Regulatory

Start

2025-10-22

Primary Completion

2028-10-31

Completion

2031-08-31

FDA Drug

Yes

Countries

• United States

Study Locations