AZTOUND Study

• Research type

  Research Study

• Full title

  A Multiphase, Pharmacokinetics, Safety, and Efficacy Study of ASTX030 (Azacitidine and Cedazuridine) as Monotherapy in Subjects with Myeloid Neoplasm or in Combination with Venetoclax in Subjects with AML (AZTOUND Study)

• IRAS ID

  1012826

• Contact name

  Yuri Sano

• Contact email

  medicalinformation@taihooncology.com

• Sponsor organisation

  Taiho Oncology, Inc.

• Eudract number

  2024-515098-93

• Clinicaltrials.gov Identifier

  NCT04256317

• Research summary

  Myeloid neoplasms are blood cancers that begin in the bone marrow stem cells. These cancers include myelodysplastic syndromes (MDS, disorders caused by blood cells that do not work properly), chronic myelomonocytic leukaemia (CMML, a type of cancer that starts in white blood-forming cells of the bone marrow and invades the blood), and acute myeloid leukaemia (AML, a cancer of the blood and bone marrow). Azacitidine (via injections under the skin), is a drug used to treat certain types of cancers and is approved in the UK as a monotherapy for MDS and in combination with Venetoclax for AML. Azacitidine works by disrupting and killing rapidly dividing cells, including cancer cells. Cedazuridine, is a drug used in combination with other drugs such as azacitidine. Cedazuridine does not treat cancer directly but allows azacitidine to be given by mouth to block the way it is quickly broken down in the body and allowing it to help kill cancer cells. Venetoclax, is a drug given by mouth that works by stopping cancer cells from surviving beyond when they normally should.

  This multiphase study will evaluate the pharmacokinetics, safety and efficacy of ASTX030 (azacitidine + cedazuridine) as a monotherapy in patients with myeloid neoplasm or in combination with Venetoclax in patients with AML. The study is being sponsored by Taiho Oncology, Inc. The UK will participate in Phase 2 Combination Therapy (ASTX030 with venetoclax) and the Phase 3 Monotherapy (ASTX030).

  The study will involve:
  • Screening – within 21 days prior to treatment
  • Treatment phase – in 28-day cycles, for as long as patients benefit from treatment
  • Safety follow-up – 30 days after last treatment dose
  • Survival follow-up – every 3 months after safety follow up

  Approximately 280 participants will be enrolled.

  Study procedures: blood and urine tests, physical evaluations, height, weight, vital signs, ECOG, 12-lead ECG, ECHO, MUGAs, urine pregnancy tests and bone marrow aspirate/biopsy.

• REC name

  London - Chelsea Research Ethics Committee

• REC reference

  25/LO/0768

• Date of REC Opinion

  15 Dec 2025

• REC opinion

  Further Information Favourable Opinion