RECRUITING
Phase 2 / Phase 3
ClinicalTrials.gov
A Multi-phase Study of ASTX030 (Azacitidine and Cedazuridine) in Myeloid Neoplasm Alone or in Combination With Venetoclax in AML (AZTOUND Study)
Sponsor: Taiho Oncology, Inc. • ClinicalTrials.gov ID: NCT04256317
Registry updated: 2026-10-06
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Check Eligibility →Description
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.
Interventions
-
Azacitidine
(Drug)
Tablets/Capsules for oral administration and powder for reconstitution to aqueous suspension for SC administration.
-
ASTX030 (cedazuridine + azacitidine)
(Drug)
FDC Capsules for oral administration.
-
Azacitidine
(Drug)
Powder for reconstitution to aqueous suspension for SC administration.
-
ASTX030 (cedazuridine + azacitidine)
(Drug)
Tablets/Capsules for oral administration.
-
Cedazuridine
(Drug)
Tablets for oral administration.
-
Venetoclax
(Drug)
Oral tablets.
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.
Conditions
Myelodysplastic Syndromes
Acute Myeloid Leukemia
Myelodysplastic Syndrome/Neoplasm
Chronic Myelomonocytic Leukemia
Locations
- Keck School of Medicine of USC — Los Angeles, California, United States
- UC Irvine Health - Chao Family Comprehensive Cancer Center — Orange, California, United States
- Yale University — New Haven, Connecticut, United States
- University of Miami - Sylvester Comprehensive Cancer Center — Miami, Florida, United States
- University of Emory - Winship Cancer Institute — Atlanta, Georgia, United States
- Dana-Farber Cancer Institute — Boston, Massachusetts, United States
- John Theurer Cancer Center / Hackensack University — Hackensack, New Jersey, United States
- Roswell Park Comprehensive Cancer Center — Buffalo, New York, United States
- New York University Langone Hospital - Long Island — Mineola, New York, United States
- Perlmutter Cancer Center - 34th Street — New York, New York, United States
- Icahn School of Medicine at Mount Sinai — New York, New York, United States
- Weill Cornell Medical Center — New York, New York, United States
- Montefiore Medical Center — The Bronx, New York, United States
- Duke University — Durham, North Carolina, United States
- Ohio State University Comprehensive Cancer Center (OSUCCC) - The James Cancer Hospital and Solove Research Institute — Columbus, Ohio, United States
- Oregon Health and Science University — Portland, Oregon, United States
- Oregon Oncology Specialists — Salem, Oregon, United States
- Hollings Cancer Center — Charleston, South Carolina, United States
- Vanderbilt University Medical Center — Nashville, Tennessee, United States
- Baylor Research Institute dba Baylor Scott & White Research Institute — Dallas, Texas, United States
- University of Texas Southwestern Medical Center — Dallas, Texas, United States
- MD Anderson Cancer Center — Houston, Texas, United States
- Seattle Cancer Care Alliance — Seattle, Washington, United States
- Froedtert & Medical College of Wisconsin — Milwaukee, Wisconsin, United States
- Eastern Health - Health Sciences Centre — St. John's, Newfoundland and Labrador, Canada
- Princess Margaret Cancer Centre — Toronto, Ontario, Canada
- Fakultni Nemocnice Ostrava — Ostrava, Moravian-Silesian, Czechia
- Fakultní Nemocnice Královské Vinohrady — Prague, Prague, Czechia
- Vseobecna Fakultni Nemocnice v Praze — Prague, Prague, Czechia
- Institut Universitaire du Cancer de Toulouse (IUCT) Oncopole — Toulouse, Haute-Garonne, France
- Hôpital l'Archet — Nice, Provence-Alpes-Côte d'Azur Region, France
- Hôpital Saint-Louis — Paris, Île-de-France Region, France
- Universitätsklinikum Freiburg — Freiburg im Breisgau, Baden-Wurttemberg, Germany
- Universitätsklinikum Heidelberg — Heidelberg, Baden-Wurttemberg, Germany
- Städtisches Klinikum Braunschweig — Braunschweig, Lower Saxony, Germany
- Universitätsklinikum Halle — Halle, Saxony-Anhalt, Germany
- Szent-Györgyi Albert Klinikai Központ, II. sz. Belgyógyászati Klinika és Kardiológiai Központ — Szeged, Csongrád megye, Hungary
- Petz Aladár Győr-Moson-Sopron Vármegyei Egyetemi Oktató Kórház — Győr, Győr-Moson-Sopron, Hungary
- Debreceni Egyetem Klinikai Központ — Debrecen, Hajdú-Bihar, Hungary
- Semmelweis Egyetem Belgyógyászati és Hematológiai Klinika — Budapest, Hungary
- Ospedale Santa Maria delle Croci di Ravenna — Ravenna, Emilia-Romagna, Italy
- Azienda Ospedaliero - Universitaria Careggi — Florence, Florence, Italy
- Azienda Ospedaliera Ordine Mauriziano di Torino — Torino, Turin, Italy
- Azienda Ospedaliero-Universitaria di Bologna - Policlinico Sant Orsola-Malpighi — Bologna, Italy
- Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico — Milan, Italy
- Azienda Ospedaliero-Universitaria Maggiore della Carità di Novara — Novara, Italy
- Fondazione PTV - Policlinico Tor Vergata — Roma, Italy
- Umberto I - Policlinico di Roma — Roma, Italy
- Azienda Ospedaliero-Universitaria Citta della Salute e della Scienza di Torino — Torino, Italy
- Samodzielny Publiczny Szpital Kliniczny nr 1 w Lublinie — Lublin, Lublin Voivodeship, Poland
- Wojewódzkie Wielospecjalistyczne Centrum — Lodz, Lódzkie, Poland
- Szpitale Pomorskie Spółka Z Ograniczoną Odpowiedzialnością — Gdynia, Pomeranian Voivodeship, Poland
- Institut Català d'Oncologia Badalona — Badalona, Barcelona, Spain
- Institut Català d'Oncologia - Hospital Duran i Reynals (ICO L'Hospitalet) — L'Hospitalet de Llobregat, Barcelona, Spain
- Clinica Universidad de Navarra - Pamplona — Pamplona, Navarre, Spain
- Hospital Universitario Central de Asturias — Oviedo, Principality of Asturias, Spain
- Hospital Universitari Vall d'Hebrón — Barcelona, Spain
- Hospital Clinic de Barcelona — Barcelona, Spain
- Hospital San Pedro de Alcantara — Cáceres, Spain
- Institut Català d'Oncologia Girona (ICO Girona) — Girona, Spain
- Hospital Universitario Virgen de las Nieves — Granada, Spain
- Hospital General Universitario Gregorio Marañón — Madrid, Spain
- Clínica Universidad de Navarra - Madrid — Madrid, Spain
- MD Anderson Cancer Center Madrid — Madrid, Spain
- Hospital Universitario La Paz — Madrid, Spain
- Hospital Quirónsalud Málaga — Málaga, Spain
- Complejo Asistencial Universitario de Salamanca - Hospital Clínico — Salamanca, Spain
- Hospital Universitari i Politècnic La Fe — Valencia, Spain
- King's College Hospital NHS Foundation Trust — London, England, United Kingdom
- Maidstone and Tunbridge Wells NHS Trust - Maidstone Hospital — Maidstone, England, United Kingdom
- The Christie NHS Foundation Trust — Manchester, England, United Kingdom
- Oxford University Hospitals NHS Foundation Trust — Oxford, England, United Kingdom
- University Hospital Southampton NHS Foundation Trust — Southampton, England, United Kingdom
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Data sourced from ClinicalTrials.gov, a service of the U.S. National Institutes of Health.
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