Actively Recruiting
A Multi-phase, Pharmacokinetics, Safety, and Efficacy Study of ASTX030 (Azacitidine and Cedazuridine) Alone or with Venetoclax in Myeloid Neoplasm and AML (AZTOUND Study)
Led by Taiho Oncology, Inc. · Updated on 2026-04-30
316
Participants Needed
71
Research Sites
52 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
This research investigates ASTX030, a combination of azacitidine and cedazuridine, given alone or with venetoclax for people with myeloid neoplasms and acute myeloid leukemia (AML). The study includes multiple phases, from early dose-finding stages to larger randomized trials, evaluating the safety, pharmacokinetics, and effectiveness of ASTX030 compared to standard subcutaneous azacitidine. It aims to understand how these treatments work in participants with myelodysplastic syndromes (MDS), chronic myelomonocytic leukemia (CMML), and AML over approximately eight years. The study involves several treatment arms: early phases focus on dose escalation and expansion of oral ASTX030; later phases compare oral ASTX030 to subcutaneous azacitidine in randomized crossover designs. Combination therapy arms test ASTX030 plus venetoclax against azacitidine plus venetoclax with specific dosing schedules over 28-day cycles. Participants may switch treatments after initial cycles. Treatments include oral tablets, capsules, and injections, with some doses taken in a fasted or fed state. Participants will undergo regular cycles of treatment with detailed monitoring including blood tests to measure drug levels and responses, assessments of side effects, and evaluations of disease status. Researchers will track outcomes like drug exposure, response rates, survival, and safety over months to years. This includes measuring DNA methylation changes, transfusion independence, and adverse events. The study is open to adults and includes ongoing safety and effectiveness follow-up.
CONDITIONS
Brief 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
Eligibility Criteria
You may qualify if you...
- Adults with confirmed diagnosis of myelodysplastic syndromes (MDS), chronic myelomonocytic leukemia (CMML), or related myelodysplastic/myeloproliferative neoplasms eligible for azacitidine treatment.
- Participants with confirmed MDS or CMML with specific subtypes or risk levels, good performance status (ECOG 0-1), adequate organ function, and no graft-versus-host disease if prior transplant.
- Able to swallow tablets or capsules within 10 minutes and tolerate fasting for 4 hours.
- Projected life expectancy of at least 12 weeks.
- Participants with newly diagnosed AML in combination therapy arms, ineligible for intensive chemotherapy due to age or comorbidities, with ECOG status 0-2 (older adults) or 0-3 (younger adults).
You will not qualify if you...
- Active uncontrolled gastric or duodenal ulcer.
- Poor medical risk due to other conditions.
- Known HIV infection or positive Hepatitis B or C infection.
- Life-threatening illness or history of certain other malignancies unless adequately treated.
- Presence of clinical extramedullary disease such as palpable hepatomegaly or splenomegaly in MDS/MPN including CMML.
- Previous treatment with more than one cycle of decitabine, azacitidine, or guadecitabine (Phases 2 and 3).
- Recent use of investigational drugs or ongoing adverse events from such treatments.
- Known hypersensitivity to cedazuridine, azacitidine, venetoclax, or their excipients.
- Inability to discontinue drugs affecting gastric emptying or certain CYP3A inhibitors before and during early cycles.
- History of other specific leukemias or karyotype abnormalities excluding AML variants sensitive to retinoic acid.
- Active CNS involvement from AML.
- Severe hepatic or renal impairment.
- Malabsorption syndrome or conditions preventing oral drug intake.
- Significant uncontrolled infections or serious medical conditions that may affect participation.
- White blood cell count over 25,000/µL (hydroxyurea allowed).
- Prior treatment with hypomethylating agents, venetoclax, CAR-T therapy, or investigational therapies for myeloid diseases.
- Current participation in other research with drug interventions.
- Significant mental illness or substance abuse.
- Consumption of grapefruit or certain citrus fruits within 7 days before treatment start.
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - Repeated 28-day cycles until disease progression or discontinuation
Participants receive oral and/or subcutaneous administration of azacitidine, cedazuridine, and venetoclax in 28-day cycles. Treatment includes dose escalation, dose expansion, and combination therapy depending on the trial phase and arm. Participants follow specific dosing schedules each cycle and may crossover between treatment arms as applicable.
Visits each cycle according to dosing schedule (approximately monthly visits)
Duration - Up to 36 months
Participants are monitored for safety, treatment response, and survival outcomes after completing treatment. Follow-up includes assessments up to 36 months to evaluate adverse events, overall survival, and duration of response.
Periodic visits for monitoring during follow-up
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
Research Team
T
Taiho Oncology, Inc.
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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