AZTOUND

A Multi-phase, Dose-Escalation Followed by an Open-label, Randomized, Crossover Study of Oral ASTX030 (Cedazuridine and Azacitidine Given in Combination) Versus Subcutaneous Azacitidine in Subjects With Myelodysplastic Syndromes (MDS), Chronic Myelomonocytic Leukemia (CMML), or Acute Myeloid Leukemia (AML)

What's the purpose of the trial?

Study ASTX030-01 is designed to move efficiently from Phase 1 to Phase 3. Phase 1 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) of ASTX030. Phase 2 is a randomized open-label crossover study to compare oral ASTX030 to subcutaneous (SC) azacitidine. Phase 3 is a randomized open-label crossover study comparing the final oral ASTX030 dose to SC azacitidine. The duration of the study is expected to be approximately 48 months.
Trial status

Accepting patients

Phase
Phase 2/3
Enrollment
316
Last Updated

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Participating Centers

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Experimental Treatments

Learn more about the experimental treatments being evaluated in this clinical trial.

  • ASTX030 is an oral DNMT inhibitor that is a combination of cedazuridine and azacitadine. It's being studied for use in several different indications.
  • Venetoclax is a BCL-2 inhibitor that may work by blocking the action of the BCL-2 protein on some cancer cells which can lead to cell death. 
  • Azacitidine is a type of chemotherapy called a hypomethylating agent that interferes with the growth and spread of cancer cells in the body. 

Published Results

Explore published results and other resources associated with this clinical trial (including press releases, news articles and videos).

Oral Azacitidine Yields Similar PK to Subcutaneous Azacitidine in MDS/CMML

The overall response rate (ORR) in patients with MDS (n = 11) was 50.0%, including 5 who achieved complete response (CR) and 2 who achieved marrow complete response. Additionally, 4 patients achieved hematologic improvement (HI), including 3 erythroid, 2 neutrophil, and 3 platelet HIs. Eight patients with MDS achieved stable disease, 2 had progressive disease, and 1 was not evaluable.

No patients with CMML responded to treatment. Five had stable disease, none had progressive disease, and 1 was not evaluable. Of 2 patients with AML, 1 achieved CR, and 1 was not evaluable.

Thirteen patients were red blood cell (RBC) transfusion-dependent at baseline; of those, 30.8% achieved 8 or more weeks of RBC transfusion independence. Median overall survival (OS) had not been reached as of most recent follow-up.

2025-12-11T00:00:00Z Read more

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