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A Multi-phase, Dose-Escalation followed by an Open-label, Randomized, Crossover Study of Oral ASTX030 (Cedazuridine/Azacitidine Together) vs Subcutaneous Azacitidine in Myelodysplastic Syndromes, Chronic Myelomonocytic Leukemia or Acute Myeloid Leukemia

Clinical Trial Details

This clinical trial is for men and women with myelodysplastic syndromes (MDS), chronic myelomonocytic leukemia (CMML), other myelodysplastic syndrome/myeloproliferative neoplasms (MDS/MPN), or acute myeloid leukemia (AML).
   
The purpose of this study is to test the levels of the investigational drug ASTX030 in participants’ blood, the safety and tolerability of the drug, and how participants respond to the drug, including if food has an effect.

ASTX030 is a combination of two medicines, azacitidine and cedazuridine, given by mouth. Azacitidine is a drug used to treat certain types of cancers. Cedazuridine does not treat cancer directly, but may allow azacitidine to be given by mouth to block the way it is broken down in the body and allow it to help kill cancer cells.
   
Investigational means that ASTX030 is not approved by the U.S. Food and Drug Administration (FDA), but is undergoing testing to find out if it is safe and effective.

This is the first time these study drugs have been given together in humans, however, azacytidine alone given by injection under the skin has been approved in the United States for the treatment of myelodysplastic syndromes (MDS) and chronic myelomonocytic leukemia (CMML) and in the European Union for the treatment of MDS, CMML, and acute myeloid leukemia (AML). The medication INQOVI® contains cedazuridine together with decitabine, a drug similar to azacitidine, and is approved for the treatment of patients with MDS or CMML in the US, Canada, and Australia. 
   
The study drugs may be given to participants in multiple forms and doses as determined by the study team. The type of pills (tablets and/or capsules) given may change, but the medicine will be the same. To understand how ASTX030 works, participants will also be given an injectable form of azacitidine under the skin for a few of the treatments.
   
The study is expected to last about 3 years. The amount of time participants will be treated or be followed up for health information as part of the study depends on how they respond to and tolerate the study drug(s). If participants benefit from treatment, they may receive the study drug(s) for as long as they continue to benefit.

Key Eligibility: 
  1. Men and women 18 years of age or older
  2. Confirmed MDS, CMML, and other MDS/MPN or AML

Detailed eligibility reviewed when you contact the study team

 

Study contact by location

Upper East Side - Manhattan

Contact(s)

Victoria Mendez
212-746-4829
vib9049@med.cornell.edu

Primary Investigator(s)

Protocol ID(s)

Weill Cornell Medicine IRB #:

2106023688

ClinicalTrials.gov:

NCT04256317

Sponsor:

ASTX030-01

Status

Open to Enrollment

Age Group

Adult

Sponsor