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A Phase 1 Study of Oral Cedazuridine and Decitabine Combination (ASTX727) and Filgrastim as Maintenance Therapy After Stem Cell Transplant in Children With High-Risk Acute Myeloid Leukemia
Led by National Cancer Institute (NCI) · Updated on 2026-05-27
47
Participants Needed
2
Research Sites
N/A
Total Duration
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AI-Summary
What this Trial Is About
This trial focuses on children and young adults up to 21 years old with high-risk acute myeloid leukemia (AML) that has returned after treatment or does not respond to treatment. These patients have already undergone an allogeneic hematopoietic stem cell transplant. Researchers are evaluating the safety, side effects, and best dose of a combination drug called ASTX727, which includes cedazuridine and decitabine, along with filgrastim. The aim is to find a suitable maintenance therapy after transplant and to study how the drug affects the body and disease outcomes. Participants receive filgrastim either by injection under the skin or into a vein daily on days 1 to 6 of each cycle. ASTX727 is taken orally daily on days 2 to 6, with dosing adjusted as needed. Each treatment cycle lasts 28 days and may be repeated up to 12 times unless the disease worsens or side effects become unacceptable. During the study, patients undergo procedures such as bone marrow aspiration and biopsy, blood sampling, lumbar puncture, and diagnostic imaging to monitor their condition. Participants are closely monitored for adverse effects and the drug's activity in the body, including how it affects DNA methylation and immune response. Researchers will track graft versus host disease and graft rejection for up to two years, as well as event-free survival at one year. Follow-up occurs for up to one year after transplant or if the disease returns. This study provides detailed safety and dosing information to guide future therapies for high-risk AML post-transplant.
CONDITIONS
Brief 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...
- Patient must be 21 years of age or younger at enrollment
- Diagnosed with newly diagnosed high-risk de novo AML, therapy-related AML, relapsed, or refractory AML in complete remission at transplant
- No evidence of active central nervous system or extramedullary disease at enrollment and treatment start
- Plan to have bone marrow sample within 14 days before conditioning regimen for transplant
- Receiving 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
- Complete remission with no detectable minimal residual disease after transplant
- Performance status score of 50 or higher using Karnofsky or Lansky scales
- Fully recovered from transplant toxicities between 42 and 100 days post-transplant
- Platelet count of at least 50,000/μL without transfusion in last 7 days
- Hemoglobin of at least 8.0 g/dL
- Absolute neutrophil count of at least 1,000/μL without growth factor support in last 3 days
- Adequate kidney function with GFR of at least 60 mL/min/1.73 m² by approved measurement
- Bilirubin level no greater than 1.5 times upper limit of normal
- ALT and AST levels no greater than three times upper limit of normal
- Albumin level of at least 2 g/dL
You will not qualify if you...
- Known inherited marrow failure syndromes such as Fanconi Anemia, Dyskeratosis congenita, or Shwachman-Diamond syndrome
- Known or suspected allergy to filgrastim, decitabine, or cedazuridine (ASTX727)
- Prior solid organ transplantation
- Pregnancy or breastfeeding; use of effective contraception required for reproductive potential
- Receiving other investigational drugs or anti-cancer agents during the study
- Use of drugs metabolized by cytidine deaminase on ASTX727 dosing days and 24 hours after
- Unable to swallow intact tablets; no nasogastric or feeding tube use allowed
- Starting ASTX727 and filgrastim more than 100 days after transplant
- Graft loss or steroid refractory/dependent acute graft versus host disease
- Uncontrolled infection or active transplant-related complications needing treatment
- Inability to comply with study safety monitoring requirements
- Patients receiving planned radiation or immunotherapy during the study period (except certain prophylactic treatments)
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 screening and enrollment visit
Duration - Up to 12 cycles of 28 days each
Participants receive filgrastim once daily on days 1-6 and oral ASTX727 once daily on days 2-6. Treatment cycles repeat every 28 days for up to 12 cycles unless there is disease progression or unacceptable toxicity. Bone marrow aspiration and biopsy, blood sample collections, and possibly lumbar puncture and diagnostic imaging occur throughout this period.
Multiple visits every cycle during treatment
Duration - Up to 1 year post treatment
After completing treatment, participants are followed up for safety and disease status at 1 year post-transplant and/or at relapse.
Follow-up visits as scheduled up to 1 year
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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