Actively Recruiting

Phase 2
Phase 3
Age: 18Years +
All Genders
NCT04256317

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

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

316

Participants Needed

71

Research Sites

440 weeks

Total Duration

On this page

AI-Summary

What this Trial Is About

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.

CONDITIONS

Official 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

Eligible

You may qualify if you...

  • Phase 2 Monotherapy: Confirmed diagnosis of MDS, CMML, or other MDS/MPN and candidates for single agent azacitidine
  • Phase 3 Monotherapy: Confirmed MDS or CMML with specific subtypes or risk levels, ECOG performance status 0 to 1
  • Adequate organ function
  • No evidence of graft-versus-host disease if prior allogeneic stem cell transplant
  • No major surgery within 3 weeks before first study treatment
  • No cytotoxic chemotherapy (excluding hydroxyurea) within 4 weeks before first study treatment
  • Able to swallow required tablets/capsules within 10 minutes and tolerate 4 hours fasting
  • Projected life expectancy of at least 12 weeks
  • Phase 1 and Phase 2 Combination Therapy: Newly diagnosed AML confirmed by WHO criteria
  • Projected life expectancy of at least 12 weeks
  • Considered ineligible for intensive induction chemotherapy based on age or comorbidities
  • ECOG Performance Status 0-2 for participants 75 years or older or 0-3 for participants 18 to 74 years old
Not Eligible

You will not qualify if you...

  • Active uncontrolled gastric or duodenal ulcer
  • Poor medical risk from other conditions
  • Known HIV infection
  • Positive for Hepatitis B or C infection
  • Life-threatening illness
  • History of other malignancies except certain treated and controlled cancers
  • Clinical extramedullary disease such as palpable hepatomegaly or splenomegaly in MDS/MPN including CMML
  • Previous treatment with more than 1 cycle of decitabine, azacitidine, or guadecitabine (Phases 2 and 3 only)
  • Recent investigational drug or therapy treatment within 2 weeks or ongoing significant adverse events from prior investigational treatments
  • Known or suspected hypersensitivity to cedazuridine or azacitidine or their excipients
  • Unable to discontinue drugs that delay gastric emptying such as GLP-1 or GIP agonists in Cycles 1 and 2
  • History of certain myeloproliferative neoplasms or AML with BCR-ABL1 translocation (Combination Therapy)
  • Specific karyotype abnormalities including t(15;17) or other acute promyelocytic leukemia variants (Combination Therapy)
  • Active central nervous system involvement from AML (Combination Therapy)
  • Severe hepatic or renal impairment (Combination Therapy)
  • Malabsorption syndrome or other conditions preventing oral administration (Combination Therapy)
  • Cardiovascular disability NYHA Class >2 (Combination Therapy)
  • Significant history of various serious diseases affecting participation (Combination Therapy)
  • Clinically significant uncontrolled infections (Combination Therapy)
  • White blood cell count >25,000/μL (Combination Therapy)
  • Prior treatment with hypomethylating agents, venetoclax, CAR-T therapy, or investigational therapies for MDS or AML (Combination Therapy)
  • Cannot discontinue certain medications affecting CYP3A or P-gp enzymes prior to study start (Combination Therapy)
  • Participation in another research study involving drug therapy or procedures (Combination Therapy)
  • Known hypersensitivity to cedazuridine, azacitidine, venetoclax, or their excipients (Combination Therapy)
  • Significant mental illness or substance abuse
  • Consumption of grapefruit, Seville oranges, or starfruit within 7 days prior to study start

AI-Screening

AI-Powered Screening

Complete this quick 3-step screening to check your eligibility

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Trial Site Locations

Total: 71 locations

1

Keck School of Medicine of USC

Los Angeles, California, United States, 90089

Actively Recruiting

2

UC Irvine Health - Chao Family Comprehensive Cancer Center

Orange, California, United States, 92868

Actively Recruiting

3

Yale University

New Haven, Connecticut, United States, 06510

Actively Recruiting

4

University of Miami - Sylvester Comprehensive Cancer Center

Miami, Florida, United States, 33136

Actively Recruiting

5

University of Emory - Winship Cancer Institute

Atlanta, Georgia, United States, 30322

Actively Recruiting

6

Dana-Farber Cancer Institute

Boston, Massachusetts, United States, 02114

Actively Recruiting

7

John Theurer Cancer Center / Hackensack University

Hackensack, New Jersey, United States, 07601

Actively Recruiting

8

Roswell Park Comprehensive Cancer Center

Buffalo, New York, United States, 14263

Actively Recruiting

9

New York University Langone Hospital - Long Island

Mineola, New York, United States, 11501

Actively Recruiting

10

Perlmutter Cancer Center - 34th Street

New York, New York, United States, 10016

Actively Recruiting

11

Icahn School of Medicine at Mount Sinai

New York, New York, United States, 10029

Actively Recruiting

12

Weill Cornell Medical Center

New York, New York, United States, 10065

Withdrawn

13

Montefiore Medical Center

The Bronx, New York, United States, 10467

Actively Recruiting

14

Duke University

Durham, North Carolina, United States, 27705

Actively Recruiting

15

Ohio State University Comprehensive Cancer Center (OSUCCC) - The James Cancer Hospital and Solove Research Institute

Columbus, Ohio, United States, 43210

Actively Recruiting

16

Oregon Health and Science University

Portland, Oregon, United States, 97239

Actively Recruiting

17

Oregon Oncology Specialists

Salem, Oregon, United States, 97301

Actively Recruiting

18

Hollings Cancer Center

Charleston, South Carolina, United States, 29425

Actively Recruiting

19

Vanderbilt University Medical Center

Nashville, Tennessee, United States, 37232

Actively Recruiting

20

Baylor Research Institute dba Baylor Scott & White Research Institute

Dallas, Texas, United States, 75204

Withdrawn

21

University of Texas Southwestern Medical Center

Dallas, Texas, United States, 75390

Actively Recruiting

22

MD Anderson Cancer Center

Houston, Texas, United States, 77030

Actively Recruiting

23

Seattle Cancer Care Alliance

Seattle, Washington, United States, 98109

Actively Recruiting

24

Froedtert & Medical College of Wisconsin

Milwaukee, Wisconsin, United States, 53226

Actively Recruiting

25

Eastern Health - Health Sciences Centre

St. John's, Newfoundland and Labrador, Canada, A1B 3V6

Actively Recruiting

26

Princess Margaret Cancer Centre

Toronto, Ontario, Canada, M5G 2C1

Actively Recruiting

27

Fakultni Nemocnice Ostrava

Ostrava, Moravian-Silesian, Czechia, 708 52

Actively Recruiting

28

Fakultní Nemocnice Královské Vinohrady

Prague, Prague, Czechia, 100 34

Actively Recruiting

29

Vseobecna Fakultni Nemocnice v Praze

Prague, Prague, Czechia, 128 08

Actively Recruiting

30

Institut Universitaire du Cancer de Toulouse (IUCT) Oncopole

Toulouse, Haute-Garonne, France, 31059

Actively Recruiting

31

Hôpital l'Archet

Nice, Provence-Alpes-Côte d'Azur Region, France, 06202

Actively Recruiting

32

Hôpital Saint-Louis

Paris, Île-de-France Region, France, 75010

Actively Recruiting

33

Universitätsklinikum Freiburg

Freiburg im Breisgau, Baden-Wurttemberg, Germany, 79106

Actively Recruiting

34

Universitätsklinikum Heidelberg

Heidelberg, Baden-Wurttemberg, Germany, 69120

Not Yet Recruiting

35

Städtisches Klinikum Braunschweig

Braunschweig, Lower Saxony, Germany, 38114

Actively Recruiting

36

Universitätsklinikum Halle

Halle, Saxony-Anhalt, Germany, 06120

Actively Recruiting

37

Szent-Györgyi Albert Klinikai Központ, II. sz. Belgyógyászati Klinika és Kardiológiai Központ

Szeged, Csongrád megye, Hungary, 6725

Not Yet Recruiting

38

Petz Aladár Győr-Moson-Sopron Vármegyei Egyetemi Oktató Kórház

Győr, Győr-Moson-Sopron, Hungary, 9023

Actively Recruiting

39

Debreceni Egyetem Klinikai Központ

Debrecen, Hajdú-Bihar, Hungary, 4032

Actively Recruiting

40

Semmelweis Egyetem Belgyógyászati és Hematológiai Klinika

Budapest, Hungary, 1088

Actively Recruiting

41

Ospedale Santa Maria delle Croci di Ravenna

Ravenna, Emilia-Romagna, Italy, 48121

Actively Recruiting

42

Azienda Ospedaliero - Universitaria Careggi

Florence, Florence, Italy, 50134

Actively Recruiting

43

Azienda Ospedaliera Ordine Mauriziano di Torino

Torino, Turin, Italy, 10128

Actively Recruiting

44

Azienda Ospedaliero-Universitaria di Bologna - Policlinico Sant Orsola-Malpighi

Bologna, Italy, 40138

Actively Recruiting

45

Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico

Milan, Italy, 20122

Actively Recruiting

46

Azienda Ospedaliero-Universitaria Maggiore della Carità di Novara

Novara, Italy, 28100

Actively Recruiting

47

Fondazione PTV - Policlinico Tor Vergata

Roma, Italy, 00133

Actively Recruiting

48

Umberto I - Policlinico di Roma

Roma, Italy, 00161

Actively Recruiting

49

Azienda Ospedaliero-Universitaria Citta della Salute e della Scienza di Torino

Torino, Italy, 10126

Actively Recruiting

50

Samodzielny Publiczny Szpital Kliniczny nr 1 w Lublinie

Lublin, Lublin Voivodeship, Poland, 20-081

Actively Recruiting

51

Wojewódzkie Wielospecjalistyczne Centrum

Lodz, Lódzkie, Poland, 93-513

Actively Recruiting

52

Szpitale Pomorskie Spółka Z Ograniczoną Odpowiedzialnością

Gdynia, Pomeranian Voivodeship, Poland, 81-519

Actively Recruiting

53

Institut Català d'Oncologia Badalona

Badalona, Barcelona, Spain, 08916

Actively Recruiting

54

Institut Català d'Oncologia - Hospital Duran i Reynals (ICO L'Hospitalet)

L'Hospitalet de Llobregat, Barcelona, Spain, 08908

Actively Recruiting

55

Clinica Universidad de Navarra - Pamplona

Pamplona, Navarre, Spain, 31008

Actively Recruiting

56

Hospital Universitario Central de Asturias

Oviedo, Principality of Asturias, Spain, 33011

Not Yet Recruiting

57

Hospital Universitari Vall d'Hebrón

Barcelona, Spain, 08035

Actively Recruiting

58

Hospital Clinic de Barcelona

Barcelona, Spain, 08036

Actively Recruiting

59

Hospital San Pedro de Alcantara

Cáceres, Spain, 10003

Actively Recruiting

60

Institut Català d'Oncologia Girona (ICO Girona)

Girona, Spain, 17007

Actively Recruiting

61

Hospital Universitario Virgen de las Nieves

Granada, Spain, 18014

Actively Recruiting

62

Hospital General Universitario Gregorio Marañón

Madrid, Spain, 28007

Actively Recruiting

63

Clínica Universidad de Navarra - Madrid

Madrid, Spain, 28027

Actively Recruiting

64

MD Anderson Cancer Center Madrid

Madrid, Spain, 28033

Actively Recruiting

65

Hospital Universitario La Paz

Madrid, Spain, 28046

Actively Recruiting

66

Hospital Quirónsalud Málaga

Málaga, Spain, 29004

Actively Recruiting

67

Complejo Asistencial Universitario de Salamanca - Hospital Clínico

Salamanca, Spain, 37007

Actively Recruiting

68

Hospital Universitari i Politècnic La Fe

Valencia, Spain, 46026

Actively Recruiting

69

King's College Hospital NHS Foundation Trust

London, England, United Kingdom, SE5 9RS

Actively Recruiting

70

The Christie NHS Foundation Trust

Manchester, England, United Kingdom, M20 4GJ

Actively Recruiting

71

University Hospital Southampton NHS Foundation Trust

Southampton, England, United Kingdom, SO16 6YD

Not Yet Recruiting

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Research Team

T

Taiho Oncology, Inc.

CONTACT

How is the study designed?

Study Type

INTERVENTIONAL

Masking

NONE

Allocation

RANDOMIZED

Model

CROSSOVER

Primary Purpose

TREATMENT

Number of Arms

6

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