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 (May 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
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 →
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