Cedazuridine + Decitabine + Filgrastim for Acute Myeloid Leukemia

This trial tests a new treatment option for children with high-risk acute myeloid leukemia (AML) that has returned or resisted previous treatments. It combines two medicines, cedazuridine and decitabine, to target leukemia cells, along with filgrastim to boost white blood cells and prevent infections. The trial aims to determine the safest and most effective dose of this treatment. Children who have undergone a stem cell transplant and have their AML in remission may qualify for this trial. As a Phase 1 trial, the research focuses on understanding how the treatment works in people, offering participants the chance to be among the first to receive this new treatment.

The trial requires that you do not take certain medications that are metabolized by cytidine deaminase (like cytarabine and gemcitabine) on days when ASTX727 is given and for 24 hours after. If you're on other anti-cancer drugs or investigational drugs, you cannot participate. Always discuss with your doctor before making any changes to your medication.

Research has shown that the combination of cedazuridine and decitabine, known as ASTX727, has a safety profile similar to other treatments for acute myeloid leukemia (AML). Studies indicate that patients generally tolerate this combination well, with most side effects being manageable and not severe.

Additionally, researchers have combined ASTX727 with venetoclax for older or less fit patients. These studies found it safe for most participants, suggesting the treatment might suit different groups of people with AML.

Filgrastim, another component of this trial, helps increase white blood cell production. It is widely used and has a well-known safety record, with most people experiencing only mild side effects like bone pain.

Researchers are excited about this treatment for Acute Myeloid Leukemia because it combines cedazuridine and decitabine in a novel way. This combination allows for the oral administration of decitabine, which is typically given intravenously, making it more convenient for patients. Cedazuridine acts to enhance the effectiveness of decitabine by preventing its breakdown in the body, potentially improving treatment outcomes. Additionally, the inclusion of filgrastim helps stimulate white blood cell production, which can aid in recovery and reduce infection risk. This approach represents a promising advance in making leukemia treatment more patient-friendly while possibly increasing its efficacy.

Research has shown that the combination of two drugs, cedazuridine and decitabine, known as ASTX727, yields promising results for treating acute myeloid leukemia (AML). In one study, 67% of patients responded well to this treatment, with 40% achieving complete remission, meaning no signs of cancer were found. Another study found that ASTX727 improved survival rates for patients with AML and myelodysplastic syndromes (MDS) after a stem cell transplant. Cedazuridine extends the effectiveness of decitabine in the body. In this trial, participants will receive ASTX727 along with Filgrastim, which may enhance the treatment's effectiveness. These findings suggest that ASTX727 could be a strong option for patients with high-risk, recurring, or hard-to-treat AML.¹²³⁴⁵