A Multi-phase Study of ASTX030 (Azacitidine and Cedazuridine) in Myeloid Neoplasm Alone or in Combination With Venetoclax in AML (AZTOUND Study)
A Multi-phase, Pharmacokinetics, Safety, and Efficacy Study of ASTX030 (Azacitidine and Cedazuridine) as Monotherapy in Subjects With Myeloid Neoplasm or in Combination With Venetoclax in Subjects With AML (AZTOUND Study)
Who can join
As published by the study on ClinicalTrials.gov. "Not stated" means the study hasn't said — ask the study team.
- Age
- 18 Years and older
- Sex
- All sexes
- Healthy volunteers
- Not accepted
- Compensation
- Not stated
Other requirements are listed under Eligibility below. Only the study team can confirm whether you can join.
What to expect
No one can promise a trial will help you personally — that is what the research is trying to find out.
What participation involves
Not stated in the study description — ask the study team about visits and procedures.
- Compensation
- Not stated
- Study-related exams and procedures are typically provided at no cost. This is how many trials work in general — confirm the details for this study with the study team.
- Participants are monitored closely by the research team throughout the study.
- Some participants may receive access to an investigational treatment, which is still being studied.
- Your participation helps researchers learn whether this approach could help future patients.
About this study
In plain language
This study tests ASTX030, a pill containing azacitidine and cedazuridine, for people with certain blood and bone marrow cancers. Some parts compare it with azacitidine shots under the skin. Other parts test it with venetoclax for people with newly diagnosed acute myeloid leukemia (AML) who cannot have strong starting chemotherapy.
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.
Compensation is read from the study's own description and may be missing — always confirm with the study team.
Eligibility
Who can join
- Phase 2, ASTX030 alone: Have MDS, CMML, or another listed MDS/MPN cancer that affects how blood cells are made. Azacitidine alone must be a suitable treatment.
- Phase 3, ASTX030 alone: Have one of the listed types or risk groups of MDS or CMML. Azacitidine alone must be a suitable treatment.
- Phase 3, ASTX030 alone: Be fully active or have limits only with hard physical activity, and have organs that work well enough.
- Phase 3, ASTX030 alone: If you had a donor stem cell transplant, have no signs that the donor cells are attacking your body. Have no major surgery within 3 weeks or cell-killing chemotherapy within 4 weeks before treatment, except hydroxyurea.
- Phase 3, ASTX030 alone: Be able to swallow the required pills within 10 minutes and go without food for 4 hours. Be expected to live at least 12 weeks.
- Combination treatment: Have newly diagnosed AML confirmed by testing, and be expected to live at least 12 weeks.
- Combination treatment: Be unable to have strong starting chemotherapy because you are 75 or older, or age 18–74 with a listed heart, lung, kidney, or liver problem or limits in daily activity.
- Combination treatment: Meet the study’s daily activity limits, which differ for people age 75 or older and those age 18–74.
Who can't join
- All parts: Have HIV, hepatitis B or C, an allergy to the assigned study drugs or their ingredients, or other health problems that make joining unsafe. Some past cancers also rule you out, but there are exceptions.
- All parts: Cannot stop medicines that slow the stomach from emptying during the first 2 treatment cycles.
- ASTX030 alone: Have an uncontrolled stomach or upper bowel ulcer, a life-threatening illness, or MDS/MPN disease outside the bone marrow, including a liver or spleen enlarged enough to feel during an exam.
- ASTX030 alone: Had a recent research treatment or still have major side effects from it. In Phases 2 and 3, had more than 1 cycle of decitabine, azacitidine, or guadecitabine.
- Combination treatment: Have certain past blood cancers, certain changes in cancer-cell chromosomes, or AML in the brain or spinal cord.
- Combination treatment: Have severe liver or kidney problems, certain serious heart problems, an uncontrolled infection needing treatment, trouble absorbing medicines through the gut, or a white blood cell count above 25,000 per microliter. Hydroxyurea may be used to lower this count.
- Combination treatment: Previously received azacitidine, decitabine, venetoclax, CAR-T cell therapy, or research treatments for MDS or AML. Also excluded if taking part in another study that requires treatment or procedures.
- Combination treatment: Cannot stop or avoid certain medicines that interact with study treatment; have significant mental illness or substance abuse problems; or ate grapefruit, Seville oranges, starfruit, or products made from them within 7 days before treatment.
Simplified for readability. The official criteria below are what the study team uses.
Official eligibility criteria
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 status of 0 to 1. 3. Participants with adequate organ function. 4. For participants with prior allogeneic stem cell transplant, no evidence of graft-versus-host disease (GVHD). 5. Participants with no major surgery within 3 weeks before first study treatment. 6. Participants with no cytotoxic chemotherapy (excluding hydroxyurea) within 4 weeks before first study treatment. 7. Is able to swallow the number of tablets/capsules required for the treatment assignment within a 10-minute period and tolerate 4 hours of fasting. 8. Participants with projected life expectancy of at least 12 weeks. * Phase 1 and Phase 2 Combination Therapy: 1. Has histological confirmation of newly diagnosed AML by World Health Organization (WHO) 2022 criteria (Phase 1) or 2016 criteria (Phase 2). 2. Participants with projected life expectancy of at least 12 weeks. 3. Must be considered ineligible for intensive induction chemotherapy defined by the following: a. Aged 75 years or older, or b. Aged 18 to 74 years with at least one of the following comorbidities: i. Severe cardiac disorder (e.g., congestive heart failure requiring treatment, ejection fraction ≤50%, or chronic stable angina). ii. Severe pulmonary disorder (e.g., diffusing capacity of the lung for carbon monoxide (DLCO) ≤65% or forced expiratory volume in 1 second \[FEV1\] ≤65%). iii. Creatinine clearance ≥30 mL/min to \<45 mL/min. iv. Moderate hepatic impairment with total bilirubin \>1.5 to ≤3.0 × upper limit of normal (ULN). v. ECOG Performance Status of 2 or 3. 4. Has an ECOG Performance Status of 0-2 for participants ≥75 years of age or 0-3 for participants 18 to 74 years of age. Exclusion Criteria: * All Monotherapy Phases: 1. Has an active uncontrolled gastric or duodenal ulcer. 2. Has poor medical risk because of other conditions. 3. Has known human immunodeficiency virus (HIV) infection. 4. Is known to be positive for Hepatitis B or C infection. 5. Has a life-threatening illness. 6. Has a history of other malignancies prior to study entry, with the exception of adequately treated in situ carcinoma of the breast or cervix uteri; localized basal cell carcinoma or squamous cell carcinoma of the skin; previous malignancy confined and surgically resected or adequately treated and controlled with other modalities; and any early stage malignancy for which no definitive therapy is required. 7. Participants with MDS/MPN including CMML who have clinical extramedullary disease including clinically palpable hepatomegaly or splenomegaly. 8. Has previous treatment with more than 1 cycle of decitabine, azacitidine, or guadecitabine (Phases 2 and 3 only). 9. 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. 10. Has a known or suspected hypersensitivity to cedazuridine or azacitidine or any of their excipients. 11. Cannot discontinue treatment with any drugs that delay gastric emptying such as glucagon-like peptide-1 (GLP-1) and/or gastric inhibitory polypeptide (GIP) agonists in Cycles 1 and 2 of the study. 12. Has a known or suspected hypersensitivity to cedazuridine or azacitidine or any of their excipients. * Phase 1 and Phase 2 Combination Therapy: 1. Has a history of MPN including myelofibrosis, essential thrombocythemia, polycythemia vera, chronic myeloid leukemia with or without BCR-ABL1 translocation, or AML with BCR-ABL1 translocation. 2. Has the following karyotype abnormalities: t(15;17) or other acute promyelocytic leukemia variants that remain sensitive to all-trans retinoic acid (ATRA) therapy \[t(8;21) and inv(16) are excluded in Phase 2 only\]. 3. Has known active central nervous system involvement from AML. 4. Has known human immunodeficiency virus (HIV) infection. 5. Is known to be positive for Hepatitis B or C infection. 6. Has severe hepatic impairment 7. Has severe renal impairment 8. Has a malabsorption syndrome or other condition that precludes enteral route of administration. 9. Has a cardiovascular disability status of New York Heart Association Class \>2. 10. Has significant history of renal, neurologic, psychiatric, endocrinologic, metabolic, immunologic, hepatic, cardiovascular, or pulmonary disease; or any other medical condition that in the opinion of the investigator would adversely affect his/her participation in this study. 11. Has clinically significant uncontrolled systemic infection requiring therapy (viral, bacterial, or fungal). 12. Has a history of other malignancies prior to study entry with the exception of adequately treated in situ carcinoma of the breast or cervix uteri; localized basal cell carcinoma or squamous cell carcinoma of the skin; previous malignancy confined and surgically resected (or adequately treated and controlled with other modalities); and any early stage malignancy for which no definitive therapy is required. 13. Has a WBC count \>25,000/ microliters (μL) (hydroxyurea treatment is permitted to meet this criterion). 14. Has received treatment with any of the following: 1. A hypomethylating agent (azacitidine or decitabine) or venetoclax, including prior treatment for MDS. 2. Chimeric Antigen Receptor (CAR)-T cell therapy. 3. Investigational therapies for MDS or AML. 15. Cannot discontinue treatment with any of the following: 1. Prophylactic antifungal therapy with CYP3A inhibitor activity or other concomitant medications with moderate or strong CYP3A inhibitor activity ≥7 days or 5 halflives, whichever is greater, prior to Cycle 1 Day 1 (C1D1). 2. Drugs that are strong CYP3A or P-gp inhibitors ≥7 days or 5 half-lives, whichever is greater, prior to C1D1. 16. Cannot avoid concomitant drugs known as moderate or strong CYP3A inducers. 17. Cannot discontinue treatment with any drugs that delay gastric emptying such as GLP-1 and/or GIP agonists in Cycles 1 and 2 of the study. 18. Is participating in another research study requiring interventions such as drug therapy or study procedures. 19. Has a known or suspected hypersensitivity to cedazuridine, azacitidine, venetoclax, or any of their excipients. 20. Has known significant mental illness or other conditions such as alcohol or other substance abuse or addictions 21. Consumes grapefruit, grapefruit products, Seville oranges (including marmalade containing Seville oranges) or starfruit ≤7 days prior to C1D1.
Study sites (24)
CA
- Keck School of Medicine of USC · Los Angeles, CA 90089More trials in Los Angeles
- UC Irvine Health - Chao Family Comprehensive Cancer Center · Orange, CA 92868More trials in Orange
CT
- Yale University · New Haven, CT 06510More trials in New Haven
FL
- University of Miami - Sylvester Comprehensive Cancer Center · Miami, FL 33136More trials in Miami
GA
- University of Emory - Winship Cancer Institute · Atlanta, GA 30322More trials in Atlanta
MA
- Dana-Farber Cancer Institute · Boston, MA 02114More trials in Boston
NC
- Duke University · Durham, NC 27705More trials in Durham
NJ
- John Theurer Cancer Center / Hackensack University · Hackensack, NJ 07601More trials in Hackensack
NY
- Roswell Park Comprehensive Cancer Center · Buffalo, NY 14263More trials in Buffalo
- New York University Langone Hospital - Long Island · Mineola, NY 11501More trials in Mineola
- Perlmutter Cancer Center - 34th Street · New York, NY 10016More trials in New York
- Icahn School of Medicine at Mount Sinai · New York, NY 10029More trials in New York
- Weill Cornell Medical Center · New York, NY 10065More trials in New York
- Montefiore Medical Center · The Bronx, NY 10467More trials in The Bronx
OH
- Ohio State University Comprehensive Cancer Center (OSUCCC) - The James Cancer Hospital and Solove Research Institute · Columbus, OH 43210More trials in Columbus
OR
- Oregon Health and Science University · Portland, OR 97239More trials in Portland
- Oregon Oncology Specialists · Salem, OR 97301More trials in Salem
SC
- Hollings Cancer Center · Charleston, SC 29425More trials in Charleston
TN
- Vanderbilt University Medical Center · Nashville, TN 37232More trials in Nashville
TX
- Baylor Research Institute dba Baylor Scott & White Research Institute · Dallas, TX 75204More trials in Dallas
- University of Texas Southwestern Medical Center · Dallas, TX 75390More trials in Dallas
- MD Anderson Cancer Center · Houston, TX 77030More trials in Houston
WA
- Seattle Cancer Care Alliance · Seattle, WA 98109More trials in Seattle
WI
- Froedtert & Medical College of Wisconsin · Milwaukee, WI 53226More trials in Milwaukee