Actively Recruiting
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
Led by National Cancer Institute (NCI) · Updated on 2026-05-13
47
Participants Needed
2
Research Sites
22 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
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.
CONDITIONS
Official Title
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
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Patients must be 21 years of age or younger at enrollment
- Diagnosed with high-risk, relapsed, or refractory acute myeloid leukemia in complete remission before transplant
- Plan to submit bone marrow sample within 14 days before transplant conditioning
- Eligible HIV-infected patients must meet specific criteria including controlled viral load and CD4 count >500
- Undergoing first allogeneic marrow, peripheral blood, or cord blood transplant
- Conditioning regimen must be myeloablative including high dose busulfan, treosulfan, or total body irradiation
- No prior exposure to ASTX727; prior decitabine exposure allowed
- Body surface area of at least 1 m² at treatment enrollment
- AML must be in complete remission with or without minimal residual disease before transplant conditioning
- Post-transplant AML must be in complete remission with no detectable minimal residual disease
- Performance status score of 50 or higher (Karnofsky or Lansky scale)
- Fully recovered from transplant and conditioning toxicities between 42 to 100 days post-transplant
- Platelet count of at least 50,000/µL without recent transfusion
- Hemoglobin level of at least 8.0 g/dL
- Absolute neutrophil count of at least 1,000/µL without recent growth factor support
- Kidney function (GFR) of at least 60 mL/min/1.73 m² by approved methods
- Liver tests within specified normal limits
- Albumin level of at least 2 g/dL
You will not qualify if you...
- Known inherited marrow failure syndromes such as Fanconi Anemia or Dyskeratosis congenita
- Known or suspected allergy to filgrastim, decitabine, or cedazuridine
- Prior solid organ transplant
- Pregnancy or breastfeeding; requirement for effective contraception during and after treatment
- Currently receiving other investigational or anti-cancer drugs (except intrathecal chemotherapy within 14 days)
- Receiving or planned to receive other anti-cancer therapies, radiation, or immunotherapy during study (except prophylactic GVHD treatments)
- Use of drugs metabolized by cytidine deaminase on ASTX727 dosing days and 24 hours after
- Inability to swallow intact tablets; no feeding tube administration
- Unable to start study drugs between 42 and 100 days post-transplant
- Graft loss or steroid-refractory/dependent acute graft versus host disease
- Uncontrolled infections or active transplant complications needing treatment
- Inability to comply with safety monitoring requirements
AI-Screening
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Trial Site Locations
Total: 2 locations
1
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States, 19104
Actively Recruiting
2
Saint Jude Children's Research Hospital
Memphis, Tennessee, United States, 38105
Actively Recruiting
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
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