Study on Oral Decitabine and Cedazuridine (ASTX727) with Venetoclax for Higher-Risk Acute Myeloid Leukemia Patients

Sponsor: National Cancer Institute (NCI)

Sponsor score: 0

ClinicalTrials ID:NCT04817241

This clinical trial is testing a new combination of oral medications, ASTX727 and venetoclax, to treat patients with higher-risk acute myeloid leukemia (AML) who do not have a specific gene change called FLT3. The study aims to see if this combination is safe and effective in controlling the disease.

Patient Parameters

Parameter	Options

Program Overview

This study is exploring the effects of a new oral treatment, ASTX727 (a combination of decitabine and cedazuridine), together with venetoclax, for patients with higher-risk acute myeloid leukemia (AML) who do not have a change in the FLT3 gene. Decitabine helps the bone marrow produce normal blood cells and kills abnormal cells, while cedazuridine increases decitabine's effectiveness. Venetoclax works by blocking a protein that cancer cells need to survive. The study will compare this new combination to standard chemotherapy to see if it is safe and effective.

Description

The study has two main parts: Phase Ib and Phase II.

In Phase Ib, researchers will test different doses of ASTX727 and venetoclax to find the safest and most effective dose. Patients will take ASTX727 by mouth once a day for 4 or 5 days and venetoclax once a day for 21 or 28 days in each 28-day cycle, for up to 12 cycles. Bone marrow samples and blood tests will be collected throughout the trial.

In Phase II, patients will be randomly assigned to one of two groups:

Group 1: Patients will take ASTX727 and venetoclax as determined in Phase Ib, for up to 12 cycles.
Group 2: Patients will receive standard chemotherapy with cytarabine and daunorubicin for up to 2 cycles.

After treatment, patients will be monitored every 3 months for up to 2 years to assess their health and response to the treatment.

Eligibility Criteria

Inclusion Criteria

Aged 18 to 65 years old.
Diagnosed with acute myeloid leukemia (AML) based on specific medical criteria.
Considered higher-risk AML according to specific guidelines.
Have not received more than one cycle of certain previous treatments for AML.
Must provide a bone marrow sample before starting the trial.
In good overall health with a performance status score of 0 to 2.
Have specific blood counts and organ function within acceptable limits.
Women of childbearing potential must use effective contraception during and after the study.
Men must use effective contraception during and after the study.
Able to understand and sign a consent form.
Have adequate heart function.

Exclusion Criteria

Have a lower-risk type of AML.
Have specific gene mutations (FLT3 TKD or FLT-ITD).
Cannot take oral medication or keep a medication diary.
Have active leukemia in the central nervous system.
Are receiving other cancer treatments or have had major surgery recently.
Have another cancer that requires treatment soon.
Have allergies to similar medications used in the study.
Are taking certain medications that interact with the study drugs.
Have uncontrolled illnesses or infections.
Have mental health or social issues that affect study participation.
Are pregnant or breastfeeding.
Have previously taken venetoclax or more than one cycle of certain other treatments.

Locations

FACILITY	ZIP	CITY	STATE
University of Alabama at Birmingham Cancer Center	35233	Birmingham	Alabama
Yale University	06520	New Haven	Connecticut
Roswell Park Cancer Institute	14263	Buffalo	New York
Montefiore Medical Center-Einstein Campus	10461	The Bronx	New York
Montefiore Medical Center - Moses Campus	10467	The Bronx	New York
Vanderbilt University/Ingram Cancer Center	37232	Nashville	Tennessee
University of Virginia Cancer Center	22908	Charlottesville	Virginia

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