Could a pill replace chemo shots for some blood cancers?

NCT ID NCT04256317

Disease control Sponsor: Taiho Oncology, Inc. Source: ClinicalTrials.gov ↗

First seen Jun 24, 2026 · Last updated Jun 27, 2026 · Updated 1 time

Summary

This study is testing an experimental oral drug called ASTX030 (a combination of azacitidine and cedazuridine) for people with certain blood cancers like myelodysplastic syndromes (MDS), acute myeloid leukemia (AML), and chronic myelomonocytic leukemia (CMML). The drug is given alone or with another drug called venetoclax. The goal is to see if the pill form works as well as the standard injected version and to check its safety. About 316 participants will be enrolled across multiple phases over roughly 8 years.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance: ASTX030 (a combination of azacitidine and cedazuridine), given as a pill, sometimes with venetoclax
What this could lead to: If successful, this could offer a more convenient oral alternative to injected chemotherapy for certain blood cancers, potentially improving quality of life.
What could go wrong: This is an early-to-mid-stage trial (Phase 2/3) with a small number of participants. The drug may not work as well as the injected version, and side effects are still being studied.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 2/3

Runs two stages together: whether the treatment works, then large-scale confirmation.

Participants

About 316 people

The number the study aims to enrol. It can still change while the study runs.

Started

May 2020

Expected to finish

Nov 2028

An estimate. End dates often move.

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

18 years and older

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

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.

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Conditions

The condition(s) this trial relates to.

acute myeloid leukemia chronic myelomonocytic leukemia hematologic disorder leukemia myelodysplastic syndrome myelodysplastic syndrome with excess blasts Myelodysplastic Syndromes myelodysplastic/myeloproliferative neoplasm

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Acute myeloid leukemia Chronic myelomonocytic leukemia Myelodysplastic syndrome/neoplasm Myelodysplastic syndromes

How to take part

Only the study team decides who joins. These are the ways to reach them.

The study's own enquiry address

This study publishes an address for enquiries. See it below .
The places running it

69 sites in 10 countries. The list below names each one and where it is.
The official record

ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.

Open the record ↗
A doctor treating you

A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.

Contacts and locations

Show contact details

Study contacts

Contact

Email: •••••@•••••

Locations

Azienda Ospedaliera Ordine Mauriziano di Torino
RECRUITING

Torino, Turin, 10128, Italy
Azienda Ospedaliero - Universitaria Careggi
RECRUITING

Florence, Florence, 50134, Italy
Azienda Ospedaliero-Universitaria Citta della Salute e della Scienza di Torino
RECRUITING

Torino, 10126, Italy
Azienda Ospedaliero-Universitaria Maggiore della Carità di Novara
RECRUITING

Novara, 28100, Italy
Azienda Ospedaliero-Universitaria di Bologna - Policlinico Sant Orsola-Malpighi
RECRUITING

Bologna, 40138, Italy
Baylor Research Institute dba Baylor Scott & White Research Institute
WITHDRAWN

Dallas, Texas, 75204, United States
Clinica Universidad de Navarra - Pamplona
RECRUITING

Pamplona, Navarre, 31008, Spain
Clínica Universidad de Navarra - Madrid
RECRUITING

Madrid, 28027, Spain
Complejo Asistencial Universitario de Salamanca - Hospital Clínico
RECRUITING

Salamanca, 37007, Spain
Dana-Farber Cancer Institute
RECRUITING

Boston, Massachusetts, 02114, United States
Debreceni Egyetem Klinikai Központ
RECRUITING

Debrecen, Hajdú-Bihar, 4032, Hungary
Duke University
RECRUITING

Durham, North Carolina, 27705, United States
Eastern Health - Health Sciences Centre
RECRUITING

St. John's, Newfoundland and Labrador, A1B 3V6, Canada
Fakultni Nemocnice Ostrava
RECRUITING

Ostrava, Moravian-Silesian, 708 52, Czechia
Fakultní Nemocnice Královské Vinohrady
RECRUITING

Prague, Prague, 100 34, Czechia
Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico
RECRUITING

Milan, 20122, Italy
Fondazione PTV - Policlinico Tor Vergata
RECRUITING

Roma, 00133, Italy
Froedtert & Medical College of Wisconsin
RECRUITING

Milwaukee, Wisconsin, 53226, United States
Hollings Cancer Center
RECRUITING

Charleston, South Carolina, 29425, United States
Hospital Clinic de Barcelona
RECRUITING

Barcelona, 08036, Spain
Hospital General Universitario Gregorio Marañón
RECRUITING

Madrid, 28007, Spain
Hospital Quirónsalud Málaga
RECRUITING

Málaga, 29004, Spain
Hospital San Pedro de Alcantara
RECRUITING

Cáceres, 10003, Spain
Hospital Universitari Vall d'Hebrón
RECRUITING

Barcelona, 08035, Spain
Hospital Universitari i Politècnic La Fe
RECRUITING

Valencia, 46026, Spain
Hospital Universitario Central de Asturias
NOT_YET_RECRUITING

Oviedo, Principality of Asturias, 33011, Spain
Hospital Universitario La Paz
RECRUITING

Madrid, 28046, Spain
Hospital Universitario Virgen de las Nieves
RECRUITING

Granada, 18014, Spain
Hôpital Saint-Louis
RECRUITING

Paris, Île-de-France Region, 75010, France
Hôpital l'Archet
RECRUITING

Nice, Provence-Alpes-Côte d'Azur Region, 06202, France
Icahn School of Medicine at Mount Sinai
RECRUITING

New York, New York, 10029, United States
Institut Català d'Oncologia - Hospital Duran i Reynals (ICO L'Hospitalet)
RECRUITING

L'Hospitalet de Llobregat, Barcelona, 08908, Spain
Institut Català d'Oncologia Badalona
RECRUITING

Badalona, Barcelona, 08916, Spain
Institut Català d'Oncologia Girona (ICO Girona)
RECRUITING

Girona, 17007, Spain
Institut Universitaire du Cancer de Toulouse (IUCT) Oncopole
RECRUITING

Toulouse, Haute-Garonne, 31059, France
John Theurer Cancer Center / Hackensack University
RECRUITING

Hackensack, New Jersey, 07601, United States
Keck School of Medicine of USC
RECRUITING

Los Angeles, California, 90089, United States
King's College Hospital NHS Foundation Trust
RECRUITING

London, England, SE5 9RS, United Kingdom
MD Anderson Cancer Center
RECRUITING

Houston, Texas, 77030, United States
MD Anderson Cancer Center Madrid
RECRUITING

Madrid, 28033, Spain
Montefiore Medical Center
RECRUITING

The Bronx, New York, 10467, United States
New York University Langone Hospital - Long Island
RECRUITING

Mineola, New York, 11501, United States
Ohio State University Comprehensive Cancer Center (OSUCCC) - The James Cancer Hospital and Solove Research Institute
RECRUITING

Columbus, Ohio, 43210, United States
Oregon Health and Science University
RECRUITING

Portland, Oregon, 97239, United States
Oregon Oncology Specialists
RECRUITING

Salem, Oregon, 97301, United States
Ospedale Santa Maria delle Croci di Ravenna
RECRUITING

Ravenna, Emilia-Romagna, 48121, Italy
Perlmutter Cancer Center - 34th Street
RECRUITING

New York, New York, 10016, United States
Petz Aladár Győr-Moson-Sopron Vármegyei Egyetemi Oktató Kórház
RECRUITING

Győr, Győr-Moson-Sopron, 9023, Hungary
Princess Margaret Cancer Centre
RECRUITING

Toronto, Ontario, M5G 2C1, Canada
Roswell Park Comprehensive Cancer Center
RECRUITING

Buffalo, New York, 14263, United States
Samodzielny Publiczny Szpital Kliniczny nr 1 w Lublinie
RECRUITING

Lublin, Lublin Voivodeship, 20-081, Poland
Seattle Cancer Care Alliance
RECRUITING

Seattle, Washington, 98109, United States
Semmelweis Egyetem Belgyógyászati és Hematológiai Klinika
RECRUITING

Budapest, 1088, Hungary
Städtisches Klinikum Braunschweig
RECRUITING

Braunschweig, Lower Saxony, 38114, Germany
Szent-Györgyi Albert Klinikai Központ, II. sz. Belgyógyászati Klinika és Kardiológiai Központ
NOT_YET_RECRUITING

Szeged, Csongrád megye, 6725, Hungary
Szpitale Pomorskie Spółka Z Ograniczoną Odpowiedzialnością
RECRUITING

Gdynia, Pomeranian Voivodeship, 81-519, Poland
The Christie NHS Foundation Trust
RECRUITING

Manchester, England, M20 4GJ, United Kingdom
UC Irvine Health - Chao Family Comprehensive Cancer Center
RECRUITING

Orange, California, 92868, United States
Umberto I - Policlinico di Roma
RECRUITING

Roma, 00161, Italy
University Hospital Southampton NHS Foundation Trust
NOT_YET_RECRUITING

Southampton, England, SO16 6YD, United Kingdom
University of Emory - Winship Cancer Institute
RECRUITING

Atlanta, Georgia, 30322, United States
University of Miami - Sylvester Comprehensive Cancer Center
RECRUITING

Miami, Florida, 33136, United States
University of Texas Southwestern Medical Center
RECRUITING

Dallas, Texas, 75390, United States
Universitätsklinikum Freiburg
RECRUITING

Freiburg im Breisgau, Baden-Wurttemberg, 79106, Germany
Universitätsklinikum Halle
RECRUITING

Halle, Saxony-Anhalt, 06120, Germany
Universitätsklinikum Heidelberg
NOT_YET_RECRUITING

Heidelberg, Baden-Wurttemberg, 69120, Germany
Vanderbilt University Medical Center
RECRUITING

Nashville, Tennessee, 37232, United States
Vseobecna Fakultni Nemocnice v Praze
RECRUITING

Prague, Prague, 128 08, Czechia
Weill Cornell Medical Center
WITHDRAWN

New York, New York, 10065, United States
Wojewódzkie Wielospecjalistyczne Centrum
RECRUITING

Lodz, Lódzkie, 93-513, Poland
Yale University
RECRUITING

New Haven, Connecticut, 06510, United States

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