Study on Venetoclax and ASTX727 for Treating Chronic Myelomonocytic Leukemia and Other Related Blood Disorders

Sponsor: National Cancer Institute (NCI)

Sponsor score: 0
Inactive

ClinicalTrials ID:NCT05600894

Provider View

This clinical trial is testing whether a combination of the drugs Venetoclax and ASTX727 is more effective than ASTX727 alone in reducing symptoms of certain bone marrow cancers, including chronic myelomonocytic leukemia and related disorders.

Patient Parameters

Program Overview

This study is exploring if combining two drugs, Venetoclax and ASTX727, can better reduce symptoms of certain bone marrow cancers compared to using ASTX727 alone. These cancers include chronic myelomonocytic leukemia (CMML) and myelodysplastic syndrome/myeloproliferative neoplasm (MDS/MPN) with excess immature blood cells (blasts). ASTX727 helps the bone marrow produce normal blood cells and kills abnormal ones, while Venetoclax targets a protein that cancer cells need to survive. The study aims to see if this combination can more effectively manage these conditions.

Description

The main goal of this study is to see how well the combination of ASTX727 and Venetoclax works in achieving complete remission in patients with chronic myelomonocytic leukemia (CMML) and related blood disorders with excess immature cells. Researchers will also look at the overall response rate, survival rates, and other health outcomes.

Participants will be randomly assigned to one of two groups:

  • Combination Therapy Group: Participants will take ASTX727 by mouth for 5 days starting on the third day of the first treatment cycle, and then on the first day of each following cycle. They will also take Venetoclax by mouth for 14 days of each cycle. Each cycle lasts 28 days, and treatment continues unless the disease worsens or side effects become unacceptable. Participants will have regular bone marrow biopsies and provide blood and cheek swab samples.

  • Monotherapy Group: Participants will take ASTX727 by mouth for 5 days starting on the third day of the first treatment cycle, and then on the first day of each following cycle. Each cycle lasts 28 days, and treatment continues unless the disease worsens or side effects become unacceptable. If participants do not respond to this treatment, they may switch to the combination therapy group. They will also have regular bone marrow biopsies and provide blood and cheek swab samples.

After finishing the study treatment, participants will be followed up every 6 months for up to 5 years or until they pass away, whichever comes first.

Eligibility Criteria

Inclusion Criteria

  • Diagnosed with a type of blood disorder called MDS/MPN with at least 5% immature blood cells (blasts).
  • White blood cell count must be below 10,000/mm³, and treatment with hydroxyurea is allowed to meet this requirement.
  • Must be 18 years or older.
  • Must have a performance status score of 0 to 2, indicating good overall health.
  • Liver and kidney function must be within specified limits.
  • HIV-positive patients can participate if their viral load is undetectable with treatment.
  • Patients with hepatitis B or C must have the virus under control or cured.
  • Patients with other cancers can participate if it does not interfere with the study treatment.
  • Must be able to understand and sign a consent form.
  • Must be able to swallow pills.

Exclusion Criteria

  • Need for urgent treatment, except for hydroxyurea.
  • More than one cycle of previous treatment for MDS/MPN, except for certain medications.
  • Participation in another clinical trial within 4 weeks before starting this study.
  • Uncontrolled central nervous system disease.
  • Consumption of grapefruit, Seville oranges, or starfruit within 3 days before starting the study.
  • Allergic reactions to similar drugs.
  • Uncontrolled illness requiring intravenous treatment.
  • Pregnant or breastfeeding women, and women of childbearing potential must use effective contraception.
  • Men must also use effective contraception during and after the study.
  • Any medical condition with an expected survival of less than 12 months.
  • Active infection at the time of study entry.

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