NCT04256317 · Taiho Oncology, Inc.
A Multi-phase Study of ASTX030 (Azacitidine and Cedazuridine) in Myeloid Neoplasm Alone or in Combination With Venetoclax in AML (AZTOUND Study)
(AZTOUND)
What this study is about
Study ASTX030-01 is a multi-phase study comprising of Phases 1-3 treatment given alone treatment group$1 and a Phase 1 two or more treatments used together treatment group$1 Phase 1 treatment given alone consists of an where both patients and doctors know the treatment given gradually increasing doses Stage (Stage A) using multiple cohorts at escalating dose levels of taken by mouth cedazuridine and azacitidine (only one study drug will be escalated at a time) followed by a Dose Expansion Stage (Stage B). Phase 2 treatment given alone is a randomly assigned, where both patients and doctors know the treatment given, crossover study to compare taken by mouth ASTX030 to injected under the skin (SC) azacitidin
View original scientific description
Study ASTX030-01 is a multi-phase study comprising of Phases 1-3 Monotherapy arms and a Phase 1 Combination Therapy arm 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).
Interventions
DRUG
Azacitidine
Tablets/Capsules for oral administration and powder for reconstitution to aqueous suspension for SC administration.
DRUG
ASTX030 (cedazuridine + azacitidine)
FDC Capsules for oral administration.
DRUG
Azacitidine
Powder for reconstitution to aqueous suspension for SC administration.
DRUG
ASTX030 (cedazuridine + azacitidine)
Tablets/Capsules for oral administration.
DRUG
Cedazuridine
Tablets for oral administration.
DRUG
Venetoclax
Oral tablets.
Primary outcome measures
Phase 1, 2 and 3 Monotherapy: Total Cycle Area Under the Curve (AUC) From 0 to 24 Hours (AUC0-24) Exposures
Time frame: Predose and at multiple timepoints post-dose up to 24 hours
Ratio of azacitidine total cycle AUC0-24 exposures after oral ASTX030 over SC azacitidine.
Phase 1 Combination Therapy: Number of Participants with Treatment-emergent Adverse Events (TEAEs)
Time frame: Up to 24 months
Phase 1 and 2 Combination Therapy: Complete Response (CR) Rate as Assessed by the Investigator
Time frame: Up to 36 months
Phase 1 Combination Therapy: AUC0-24 of Venetoclax With ASTX030
Time frame: Pre-dose and at multiple timepoints post-dose up to 24 hours on Day 7 (with ASTX030] of Cycle 1
Phase 1 Combination Therapy: AUC0-24 of Venetoclax Without ASTX030
Time frame: Pre-dose and at multiple timepoints post-dose up to 24 hours on Day 14 (without ASTX030) of Cycle 1
Phase 1 Combination Therapy: Maximum Plasma Concentration (Cmax) of Venetoclax With ASTX030
Time frame: Pre-dose and at multiple timepoints post-dose up to 24 hours on Day 7 (with ASTX030] of Cycle 1
Phase 1 Combination Therapy: Cmax of Venetoclax Without ASTX030
Time frame: Pre-dose and at multiple timepoints post-dose up to 24 hours on Day 14 (without ASTX030) of Cycle 1
Who can participate
This study lists these criteria on ClinicalTrials.gov. A study coordinator reviews eligibility during screening — this page does not determine whether you qualify.
Inclusion criteria
- Phase 2 Monotherapy: 1\. Has Confirmed MDS, CMML, or other MDS/MPN diagnosis who are candidates to receive and benefit from single agent azacitidine and as applicable according to local country approvals and/or local institution standard practice.
- Phase 3 Monotherapy: 1. Has confirmed MDS or CMML and is a candidate to receive and benefit from single agent azacitidine as applicable according to local country approvals and/or local institution standard practice: a) French-American-British myelodysplastic syndrome subtypes: refractory anemia (RA) or refractory anemia with ringed sideroblasts (if accompanied by neutropenia or thrombocytopenia or requiring transfusions), refractory anemia with excess blasts (RAEB), refractory anemia with excess blasts in transformation (RAEB-T), and CMML or MDS with intermediate-2 or high risk MDS according to the International Prognostic Scoring System (IPSS). 2. Eastern Cooperative Oncology Group (ECOG) performance s
Where
- Los Angeles, California
- Orange, California
- New Haven, Connecticut
- Miami, Florida
- Atlanta, Georgia
- Boston, Massachusetts
- Hackensack, New Jersey
- Buffalo, New York
- Mineola, New York
- New York, New York
- The Bronx, New York
- Durham, North Carolina
And 9 more locations — see the full list below.
Related conditions & keywords
Frequently asked questions
What is a clinical trial?
A clinical trial is a research study that tests new medical treatments, drugs, devices, or procedures to determine their safety and effectiveness. Trials are carefully designed and monitored to protect participants while advancing medical knowledge.
Is it safe to participate?
Clinical trials follow strict safety guidelines and ethical standards. Trials must be reviewed and approved, and participants are closely monitored by medical professionals throughout the study. You can withdraw at any time if you choose.
Will I be compensated?
Many clinical trials offer compensation for your time, travel expenses, and inconvenience. The specific compensation varies by study and will be discussed during the screening process. All study-related medical care is typically provided at no cost to participants.
Will I receive a placebo instead of treatment?
When effective treatment exists, participants typically receive either the standard treatment plus the study intervention, or the standard treatment plus placebo. You would not be denied effective care. Placebos are primarily used when no proven treatment is available, or in addition to standard care. Your trial consent form will clearly explain what treatments you may receive.
Can I leave a trial if I change my mind?
Absolutely. Participation in clinical trials is completely voluntary. You have the right to withdraw from the study at any time, for any reason, without penalty or loss of benefits to which you are otherwise entitled.
How long does a clinical trial last?
Trial duration varies widely depending on the study design and purpose. Some trials last just a few weeks, while others may continue for months or years. The study coordinator will provide specific timeline information during your screening call.
Data: ClinicalTrials.gov · synced Apr 30, 2026 · Source of record for eligibility and locations