OncoMatch/Clinical Trials/NCT04256317
A Multi-phase Study of ASTX030 (Azacitidine and Cedazuridine) in Myeloid Neoplasm Alone or in Combination With Venetoclax in AML (AZTOUND Study)
Is NCT04256317 recruiting? Yes, currently enrolling (Jun 2026). This Phase 2/3 trial studies multiple treatments for myelodysplastic syndromes.
Treatment: Azacitidine · ASTX030 (cedazuridine + azacitidine) · Azacitidine · ASTX030 (cedazuridine + azacitidine) · Cedazuridine · Venetoclax — Study ASTX030-01 is a multi-phase study comprising of Phases 1-3 Monotherapy arms, and Phase 1 and Phase 2 Combination Therapy arms. Phase 1 Monotherapy consists of an open-label Dose Escalation Stage (Stage A) using multiple cohorts at escalating dose levels of oral cedazuridine and azacitidine (only one study drug will be escalated at a time) followed by a Dose Expansion Stage (Stage B). Phase 2 Monotherapy is a randomized, open-label, crossover study to compare oral ASTX030 to subcutaneous (SC) azacitidine. Phase 3 Monotherapy is a randomized open-label crossover study comparing the final fixed dose of oral ASTX030 to SC azacitidine. Phase 1 Combination Therapy is an open-label, multicenter, randomized, exploratory study comparing ASTX030 and SC azacitidine in combination with venetoclax in participants with treatment-naïve AML. Phase 2 Combination Therapy is an open-label, single arm, study evaluating the efficacy, safety, pharmacokinetics (PK), and drug interactions of ASTX030 in combination with venetoclax in participants with treatment-naïve AML. The duration of this multi-phase study is approximately 8 years.
Check if I qualifyExtracted eligibility criteria
Treatments studied
Targeted therapy
Chemotherapy
Other
Cancer type
Myelodysplastic Syndrome
Acute Myeloid Leukemia
Performance status
ECOG 0–1(Restricted strenuous activity)
Prior therapy
Cannot have received: decitabine, azacitidine, or guadecitabine (decitabine, azacitidine, guadecitabine)
Exception: no more than 1 cycle allowed (Phases 2 and 3 only)
Has previous treatment with more than 1 cycle of decitabine, azacitidine, or guadecitabine (Phases 2 and 3 only)
Cannot have received: investigational drug or therapy
Exception: within 2 weeks, or 5 half-lives, whichever is longer, before the protocol-defined first dose of study treatment
Has been treated with any investigational drug or therapy within 2 weeks, or 5 half-lives, whichever is longer, before the protocol-defined first dose of study treatment, or ongoing clinically significant adverse events from previous treatment with investigational drug or therapy
Cannot have received: hypomethylating agent (azacitidine or decitabine) or venetoclax (azacitidine, decitabine, venetoclax)
A hypomethylating agent (azacitidine or decitabine) or venetoclax, including prior treatment for MDS
Cannot have received: CAR-T cell therapy
Chimeric Antigen Receptor (CAR)-T cell therapy
Cannot have received: investigational therapies for MDS or AML
Investigational therapies for MDS or AML
Lab requirements
Kidney function
creatinine clearance ≥30 mL/min to <45 mL/min allowed for combination therapy; severe renal impairment excluded
Liver function
moderate hepatic impairment with total bilirubin >1.5 to ≤3.0 × ULN allowed for combination therapy; severe hepatic impairment excluded
Participants with adequate organ function. Combination therapy: creatinine clearance ≥30 mL/min to <45 mL/min, moderate hepatic impairment with total bilirubin >1.5 to ≤3.0 × ULN allowed; severe hepatic/renal impairment excluded
Structured fields extracted by AI. May contain errors — verify against the official protocol.
US trial sites
- Keck School of Medicine of USC · Los Angeles, California
- UC Irvine Health - Chao Family Comprehensive Cancer Center · Orange, California
- Yale University · New Haven, Connecticut
- University of Miami - Sylvester Comprehensive Cancer Center · Miami, Florida
- University of Emory - Winship Cancer Institute · Atlanta, Georgia
Showing up to 5 US sites.
See all sites on ClinicalTrials.gov →Frequently asked questions
Is NCT04256317 currently recruiting?
Yes, this trial is currently recruiting patients.
Are there prior therapy exclusions?
Yes. Prior decitabine, azacitidine, or guadecitabine, investigational drug or therapy, hypomethylating agent (azacitidine or decitabine) or venetoclax disqualifies patients from enrollment.
Could you qualify for this trial?
Enter your biomarker results to see how this trial's eligibility criteria match your specific cancer profile.
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