Actively Recruiting
A Phase Ia/Ib Open-label, Multiple Dose, Study to Determine the Recommended Dose, Evaluate PKs, PDs, Safety, and Activity of Venetoclax in Combination With Oral Decitabine/Cedazuridine (ASTX727) in Pediatric Patients With Relapsed/Refractory Acute Myeloid Leukemia (AML)
Led by M.D. Anderson Cancer Center · Updated on 2026-04-29
40
Participants Needed
1
Research Sites
251 weeks
Total Duration
On this page
Sponsors
M
M.D. Anderson Cancer Center
Lead Sponsor
A
Astex Pharmaceuticals, Inc.
Collaborating Sponsor
AI-Summary
What this Trial Is About
To find a recommended dose of ASTX727 (cedazuridine/decitabine) in combination with venetoclax for pediatric patients with relapsed AML.
CONDITIONS
Official Title
A Phase Ia/Ib Open-label, Multiple Dose, Study to Determine the Recommended Dose, Evaluate PKs, PDs, Safety, and Activity of Venetoclax in Combination With Oral Decitabine/Cedazuridine (ASTX727) in Pediatric Patients With Relapsed/Refractory Acute Myeloid Leukemia (AML)
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Pediatric participants with relapsed or refractory AML by WHO criteria
- At least 5% blasts in bone marrow or peripheral blood or AML defining genetic abnormality
- Lansky performance status 6 for participants 6 years old, Karnofsky 50% for participants >16 years old
- Age between 2 and 18 years
- Able to swallow pills
- Baseline labs within specified limits for bilirubin, creatinine clearance, ALT/AST
- Females of childbearing potential must have negative pregnancy test within 14 days prior to dosing and agree to use effective contraception
- Males must use condom during study and for 3 months after last dose and report partner pregnancy
- Parent or legal representative must provide informed consent and assent as appropriate
You will not qualify if you...
- Receiving other anti-cancer therapy or investigational trials except hydroxyurea limited to first 2 cycles
- History of untreated invasive malignancy except certain skin cancers or carcinomas in situ
- Uncontrolled significant CNS conditions such as epilepsy, stroke, severe brain injuries, or psychosis
- Active cerebral or meningeal leukemia disease
- Uncontrolled active infections interfering with study drug assessment
- Active hepatitis B or C infection, or HIV infection with exceptions for stable disease or viral suppression
- Liver cirrhosis or serious active liver disease or suspected alcohol abuse
- Active graft versus host disease (GVHD) or on immunosuppressive drugs for GVHD; must be off calcineurin inhibitors for 4 weeks
- Recent chemotherapy/radiotherapy/investigational therapy within 2 weeks except specified agents
- Pregnant or lactating females
- Unwillingness to use effective contraception if of childbearing potential
- Severe uncontrolled medical or psychiatric conditions increasing risk or interfering with study
- Acute promyelocytic leukemia, juvenile myelomonocytic leukemia, or bone marrow failure syndromes
- Known or suspected allergy to decitabine, cedazuridine, or venetoclax
- Use of medications metabolized by CDA without proper review and dosing adjustment
- Cognitive impairment in adult participants (18 years or older)
AI-Screening
AI-Powered Screening
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Trial Site Locations
Total: 1 location
1
MD Anderson Cancer Center
Houston, Texas, United States, 77030
Actively Recruiting
Research Team
A
Amber Gibson, DO
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NON_RANDOMIZED
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
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