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

Conditions

• Acute Myeloid Leukemia Post Cytotoxic Therapy
• Recurrent Acute Myeloid Leukemia
• Refractory Acute Myeloid Leukemia

Eligibility Criteria

Sex

ALL

Age

N/A - 21 Years

Healthy Volunteers

Not accepted

Interventions

• Biospecimen Collection — PROCEDURE
  Undergo blood sample collection
• Bone Marrow Aspiration — PROCEDURE
  Undergo bone marrow aspiration
• Bone Marrow Biopsy — PROCEDURE
  Undergo bone marrow biopsy
• Decitabine — DRUG
  Given PO
• Decitabine and Cedazuridine — DRUG
  Given PO
• Filgrastim — BIOLOGICAL
  Given SC or IV
• Imaging Procedure — PROCEDURE
  Undergo diagnostic imaging
• Lumbar Puncture — PROCEDURE
  Undergo lumbar puncture

Study Details

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.

Key Dates

Start date

Jul 26, 2026

Status verified

May 2026

Primary completion

Mar 31, 2027

Completion

Mar 31, 2027

Study Design

Enrollment

47 participants (estimated)

Allocation

NA

Intervention model

SINGLE_GROUP

Primary purpose

TREATMENT

Arms

• Experimental: Treatment (ASTX727, diecitabine, filgrastim)
  Patients receive filgrastim SC or IV QD on days 1-6 and ASTX727 PO QD on days 2-6. Patients may receive decitabine PO QD on days 2-6 to achieve the appropriate dose. Cycles repeat every 28 days for 12 cycles in the absence of disease progression or unacceptable toxicity. Patients undergo bone marrow aspiration and biopsy and blood sample collection throughout the study and may undergo lumbar puncture and diagnostic imaging throughout the study.

Primary Outcome Measure

Maximum tolerated dose of ASTX727 [ Time Frame: Up to completion of cycle 2 (cycle length=28 days) ]

Locations (5)

Find similar trials in Minneapolis, MN

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