What is ASTX030 studying for myeloid neoplasms and AML?

ASTX030 is being studied to assess its safety, how the body processes it, and how it works alone or with venetoclax in people with myeloid neoplasms and acute myeloid leukemia. The research compares oral ASTX030 to standard subcutaneous azacitidine and looks at treatment effects in different phases. Taiho Oncology, Inc. is leading this study to better understand these treatment options.

Who may be eligible for this myeloid neoplasm clinical trial?

Adults aged 18 and older with diagnosed myelodysplastic syndromes, chronic myelomonocytic leukemia, or newly diagnosed AML may be eligible. Participants must be able to swallow tablets, tolerate fasting, and have adequate organ function without active severe infections or certain other health risks. Please review eligibility criteria for more details.

Is there any cost to participate in this myeloid neoplasm clinical trial?

Study-related care, including study drugs and all trial visits, is provided at no cost. However, personal travel expenses and other related costs may vary and are generally not covered by the study.

Actively Recruiting

Phase 2

Phase 3

Age: 18Years +

All Genders

ID04256317

A Multi-phase, Pharmacokinetics, Safety, and Efficacy Study of ASTX030 Azacitidine and Cedazuridine Alone or with Venetoclax in Myeloid Neoplasm and AML AZTOUND Study

Led by Taiho Oncology, Inc. · Updated on 2026-04-30

316

Participants Needed

Research Sites

52 weeks

Total Duration

AI-Summary

What this Trial Is About

This research investigates ASTX030, a combination of azacitidine and cedazuridine, given alone or with venetoclax for people with myeloid neoplasms and acute myeloid leukemia AML. The study includes multiple phases, from early dose-finding stages to larger randomized trials, evaluating the safety, pharmacokinetics, and effectiveness of ASTX030 compared to standard subcutaneous azacitidine. It aims to understand how these treatments work in participants with myelodysplastic syndromes MDS, chronic myelomonocytic leukemia CMML, and AML over approximately eight years. The study involves several treatment arms early phases focus on dose escalation and expansion of oral ASTX030 later phases compare oral ASTX030 to subcutaneous azacitidine in randomized crossover designs. Combination therapy arms test ASTX030 plus venetoclax against azacitidine plus venetoclax with specific dosing schedules over 28-day cycles. Participants may switch treatments after initial cycles. Treatments include oral tablets, capsules, and injections, with some doses taken in a fasted or fed state. Participants will undergo regular cycles of treatment with detailed monitoring including blood tests to measure drug levels and responses, assessments of side effects, and evaluations of disease status. Researchers will track outcomes like drug exposure, response rates, survival, and safety over months to years. This includes measuring DNA methylation changes, transfusion independence, and adverse events. The study is open to adults and includes ongoing safety and effectiveness follow-up.

CONDITIONS

Brief Title

A Multi-phase Study of ASTX030 (Azacitidine and Cedazuridine) in Myeloid Neoplasm Alone or in Combination With Venetoclax in AML (AZTOUND Study)

Who Can Participate

Age: 18Years +

All Genders

Eligibility Criteria

You may qualify if you...

Adults with confirmed diagnosis of myelodysplastic syndromes (MDS), chronic myelomonocytic leukemia (CMML), or related myelodysplastic/myeloproliferative neoplasms eligible for azacitidine treatment.
Participants with confirmed MDS or CMML with specific subtypes or risk levels, good performance status (ECOG 0-1), adequate organ function, and no graft-versus-host disease if prior transplant.
Able to swallow tablets or capsules within 10 minutes and tolerate fasting for 4 hours.
Projected life expectancy of at least 12 weeks.
Participants with newly diagnosed AML in combination therapy arms, ineligible for intensive chemotherapy due to age or comorbidities, with ECOG status 0-2 (older adults) or 0-3 (younger adults).

You will not qualify if you...

Active uncontrolled gastric or duodenal ulcer.
Poor medical risk due to other conditions.
Known HIV infection or positive Hepatitis B or C infection.
Life-threatening illness or history of certain other malignancies unless adequately treated.
Presence of clinical extramedullary disease such as palpable hepatomegaly or splenomegaly in MDS/MPN including CMML.
Previous treatment with more than one cycle of decitabine, azacitidine, or guadecitabine (Phases 2 and 3).
Recent use of investigational drugs or ongoing adverse events from such treatments.
Known hypersensitivity to cedazuridine, azacitidine, venetoclax, or their excipients.
Inability to discontinue drugs affecting gastric emptying or certain CYP3A inhibitors before and during early cycles.
History of other specific leukemias or karyotype abnormalities excluding AML variants sensitive to retinoic acid.
Active CNS involvement from AML.
Severe hepatic or renal impairment.
Malabsorption syndrome or conditions preventing oral drug intake.
Significant uncontrolled infections or serious medical conditions that may affect participation.
White blood cell count over 25,000/µL (hydroxyurea allowed).
Prior treatment with hypomethylating agents, venetoclax, CAR-T therapy, or investigational therapies for myeloid diseases.
Current participation in other research with drug interventions.
Significant mental illness or substance abuse.
Consumption of grapefruit or certain citrus fruits within 7 days before treatment start.

Research Team

Taiho Oncology, Inc.

Not the Right Trial for You?

Explore thousands of other clinical trials that might be a better match.
Sign up to get personalized trial recommendations delivered to your inbox.

Already have an account? Log in here