Testing a new drug combination after stem cell transplant for childhood leukemia

Plain-English translation of NCT07012044 on ClinicalTrials.gov ↗ · Source last updated August 2026 · Translation generated July 2026 · How we translate trials

Phase 1 — Testing in a small group (usually 20–80 people) to find a safe dose and watch for side effects.

What is this trial?

This trial is testing a medication combination called ASTX727 (cedazuridine and ) along with (a growth-factor injection) to be given after children receive a stem cell transplant for high-risk acute myeloid leukemia. The study wants to find the safest and most effective dose of the medication and understand how well it works to prevent the leukemia from coming back. Researchers will monitor how your body handles the treatment and watch for any side effects.

After a stem cell transplant, children with high-risk leukemia have a significant chance the disease could return or not fully respond to previous treatment. This trial is exploring whether this medication combination, given as maintenance therapy after transplant, can improve long-term survival and reduce the risk of leukemia relapse.

Do you qualify?

You likely qualify if…

you are 21 years old or younger at the time of enrollment
you have newly diagnosed, relapsed, or treatment-resistant acute myeloid leukemia and are in complete remission before your transplant
you are planning to receive your first allogeneic (donor) stem cell transplant with a myeloablative conditioning regimen using busulfan, treosulfan, or total body irradiation
your leukemia status has been confirmed by bone marrow testing within 14 days before your conditioning regimen begins
if you have HIV, your CD4 count is above 500 cells and your viral load is undetectable

You likely don't qualify if…

you have active disease in your central nervous system or outside the bone marrow at the time of enrollment
you are taking HIV medications that may interact with or have similar toxicities to the study medication
you have a history of HIV complications beyond a low CD4 count, or resistant HIV
you have had a previous stem cell transplant

What participation looks like

You will receive the study treatment in 12 cycles, with each cycle lasting 28 days. During each cycle, you will receive injections for 6 days, and take the oral medication ASTX727 for 5 days. You will have regular blood draws and bone marrow biopsies throughout the study to monitor how the treatment is working and check for side effects. The study team will follow you for at least 1 year after your transplant to track your recovery and how well the treatment prevented leukemia from returning.

AI-generated summary from trial data · Jul 12, 2026 · Not medical advice

Testing a new drug combination after stem cell transplant for childhood leukemia

What is this trial?

Do you qualify?

What participation looks like

Trial locations(8 sites)