RecruitingPhase 1NCT07012044

A Study to Find the Highest Dose of Cedazuridine and Decitabine Combination With Filgrastim as a Treatment Option After Hematopoietic Stem Cell Transplant in Children With High-Risk Acute Myeloid Leukemia

A Phase 1 Study of Oral Cedazuridine and Decitabine Combination (ASTX727, NSC# 820631) and Filgrastim as Maintenance Therapy Post-Hematopoietic Stem Cell Transplant in Children With High-Risk Acute Myeloid Leukemia


Sponsor

National Cancer Institute (NCI)

Enrollment

47 participants

Start Date

Jun 9, 2026

Study Type

INTERVENTIONAL

Conditions

Summary

This phase I trial tests the safety, side effects, and best dose of ASTX727 and filgrastim for the treatment of children with high risk acute myeloid leukemia that has come back after a period of improvement (recurrent) or that does not respond to treatment (refractory) who have undergone allogenic hematopoietic stem cell transplantation. ASTX727 is a combination of cedazuridine and decitabine. Cedazuridine is in a class of medications called cytidine deaminase inhibitors. It prevents the breakdown of decitabine, making it more available in the body so that decitabine will have a greater effect. Decitabine is in a class of medications called hypomethylation agents. It works by helping the bone marrow produce normal blood cells and by killing abnormal cells in the bone marrow. Filgrastim stimulates the production of neutrophils (a type of white blood cell) which can help to prevent infection. Giving ATSX727 and filgrastim may be safe and tolerable in treating children with high risk, recurrent or refractory acute myeloid leukemia who have undergone allogenic hematopoietic stem cell transplantation.


Eligibility

Max Age: 21 Years

Plain Language Summary

Simplified for easier understanding

This clinical trial is studying a procedure called Biospecimen Collection, a procedure called Bone Marrow Aspiration, and others for people with acute myeloid leukemia post cytotoxic therapy, recurrent acute myeloid leukemia, and other related conditions. The study is currently recruiting participants at 2 locations. People eligible for this study include up to age 21 Years.

This summary was AI-generated to explain the trial in plain language. It is not medical advice. Always discuss eligibility with your doctor before enrolling in a clinical trial.

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Interventions

PROCEDUREBiospecimen Collection

Undergo blood sample collection

PROCEDUREBone Marrow Aspiration

Undergo bone marrow aspiration

PROCEDUREBone Marrow Biopsy

Undergo bone marrow biopsy

DRUGDecitabine

Given PO

DRUGDecitabine and Cedazuridine

Given PO

BIOLOGICALFilgrastim

Given SC or IV

PROCEDUREImaging Procedure

Undergo diagnostic imaging

PROCEDURELumbar Puncture

Undergo lumbar puncture


Locations(7)

Children's Hospital of Alabama

Birmingham, Alabama, United States

Riley Hospital for Children

Indianapolis, Indiana, United States

University of Minnesota/Masonic Cancer Center

Minneapolis, Minnesota, United States

Memorial Sloan Kettering Cancer Center

New York, New York, United States

Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, United States

Children's Hospital of Pittsburgh of UPMC

Pittsburgh, Pennsylvania, United States

Saint Jude Children's Research Hospital

Memphis, Tennessee, United States

View Full Details on ClinicalTrials.gov

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NCT07012044


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