A Multi-phase Study of ASTX030 (Azacitidine and Cedazuridine) in Myeloid Neoplasm Alone or in Combination With Venetoclax in AML (AZTOUND Study)
NCT ID: NCT04256317
Last Updated: 2025-12-29
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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RECRUITING
PHASE2/PHASE3
236 participants
INTERVENTIONAL
2020-05-21
2028-05-01
Brief Summary
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The duration of this multi-phase study is approximately 7 years.
Detailed Description
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Conditions
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Keywords
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Study Design
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RANDOMIZED
CROSSOVER
TREATMENT
NONE
Study Groups
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Phase 1 Monotherapy , Stage A (Dose Escalation)
In Cycle 1 (28 days per cycle), single dose oral azacitidine will be administered, followed by SC azacitidine, ASTX030 and oral cedazuridine on a specific dosing schedule; in Cycle 2, oral ASTX030 (cedazuridine + azacitidine) will be administered.
Azacitidine
Tablets/Capsules for oral administration and powder for reconstitution to aqueous suspension for SC administration.
ASTX030 (cedazuridine + azacitidine)
Tablets/Capsules for oral administration.
Cedazuridine
Tablets for oral administration.
Phase 1 Monotherapy, Stage B (Dose Expansion)
In Cycle 1 (28 days per cycle), single dose oral azacitidine will be administered, followed by SC azacitidine, ASTX030 and oral cedazuridine on a specific dosing schedule; in Cycle 2, oral ASTX030 (cedazuridine + azacitidine) will be administered. On Day 7 of Cycle 2 drug products will administered in a fed state and all other doses will be administered in fasted state.
Azacitidine
Tablets/Capsules for oral administration and powder for reconstitution to aqueous suspension for SC administration.
ASTX030 (cedazuridine + azacitidine)
Tablets/Capsules for oral administration.
Cedazuridine
Tablets for oral administration.
Phase 2 Monotherapy, Part B, Sequence A & B
In Sequence A: Oral ASTX030 (cedazuridine + azacitidine) will be administered in Cycle 1, followed by SC azacitidine in Cycle 2; all participants will receive ASTX030 in subsequent cycles (Cycles ≥3).
In Sequence B: SC azacitidine will be administered in Cycle 1, followed by oral cedazuridine + azacitidine tablets/capsules in Cycle 2; all participants will receive ASTX030 in subsequent cycles (Cycles ≥3).
ASTX030 (cedazuridine + azacitidine)
FDC Capsules for oral administration.
Azacitidine
Powder for reconstitution to aqueous suspension for SC administration.
Phase 3 Monotherapy, Sequence A & B
In Sequence A: Participants will receive ASTX030 in Cycle 1, followed by SC azacitidine in Cycle 2; all participants will receive ASTX030 in subsequent cycles (Cycles ≥3).
In Sequence B: Participants will receive SC azacitidine in Cycle 1 followed by ASTX030 in Cycle 2; all participants will receive ASTX030 in subsequent cycles (Cycles ≥3).
ASTX030 (cedazuridine + azacitidine)
FDC Capsules for oral administration.
Azacitidine
Powder for reconstitution to aqueous suspension for SC administration.
Phase 1 Combination Therapy
Treatment Arm 1: Participants will receive oral dose of ASTX030 along with ramp-up oral dosing of venetoclax in Cycle 1 (cycle length = 28 days); participants will receive ASTX030 along with venetoclax on a specific dosing schedule in subsequent cycles (Cycles ≥2).
Treatment Arm 2: Participants will receive SC azacitidine along with ramp-up oral dosing of venetoclax in Cycle 1 (cycle length = 28 days); participants will receive SC azacitidine along with oral dose of venetoclax on a specific dosing schedule in subsequent cycles (Cycles ≥2). At the beginning of Cycle 5, Arm 2 patients may be permitted to cross over to Arm 1.
ASTX030 (cedazuridine + azacitidine)
FDC Capsules for oral administration.
Azacitidine
Powder for reconstitution to aqueous suspension for SC administration.
Venetoclax
Oral tablets.
Interventions
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Azacitidine
Tablets/Capsules for oral administration and powder for reconstitution to aqueous suspension for SC administration.
ASTX030 (cedazuridine + azacitidine)
FDC Capsules for oral administration.
Azacitidine
Powder for reconstitution to aqueous suspension for SC administration.
ASTX030 (cedazuridine + azacitidine)
Tablets/Capsules for oral administration.
Cedazuridine
Tablets for oral administration.
Venetoclax
Oral tablets.
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
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 Combination Therapy:
1. Has histological confirmation of newly diagnosed AML by World Health Organization (WHO) 2022 criteria.
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
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 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.
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.
18 Years
ALL
No
Sponsors
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Taiho Oncology, Inc.
INDUSTRY
Responsible Party
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Locations
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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
University of Rochester - Wilmot Cancer Center
Rochester, 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
Fakultni Nemocnice Brno
Brno, South Moravian, 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
The Christie NHS Foundation Trust
Manchester, England, United Kingdom
University Hospital Southampton NHS Foundation Trust
Southampton, England, United Kingdom
Countries
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Central Contacts
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Other Identifiers
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2024-515098-93
Identifier Type: OTHER
Identifier Source: secondary_id
ASTX030-01
Identifier Type: -
Identifier Source: org_study_id