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Study on the Safety and Effectiveness of Azacitidine and Venetoclax for Untreated Higher-Risk Myelodysplastic Syndromes Not Eligible for Transplant

Verified siteInvestigationalNo placebo
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What is this trial about?

A plain-language summary of the goals, design and what participants do

This clinical trial is focused on studying a group of blood disorders known as higher-risk myelodysplastic syndromes (MDS). These are conditions where the bone marrow does not produce enough healthy blood cells. The study is specifically for patients who have not received previous treatment and are not eligible for a type of treatment called allogenic transplantation, which involves receiving stem cells from a donor. The trial will test the safety and initial effectiveness of a combination of two medications: Oral Azacitidine (also known by its code name CC-486 or ONUREG) and Venetoclax (also known as VENCLYXTO or by its code name ABT-199).

The purpose of the study is to find the best dose and treatment duration for these medications when used together. Participants will take these medications in tablet form by mouth. The study will monitor how the body responds to the treatment and any side effects that may occur. The trial will also look at how well the treatment works in improving the condition of the blood and bone marrow over time.

Throughout the study, participants will have regular check-ups, including blood tests and other assessments, to track their progress and ensure their safety. The study aims to provide valuable information on how these medications can be used to treat higher-risk myelodysplastic syndromes effectively.

The research process

The trial runs in 5 steps – from screening to follow-up. Each step says what happens and what the team monitors.

  1. Step 1

    Initial visit

    Upon joining the study, an initial visit is scheduled. During this visit, a detailed explanation of the study procedures and requirements is provided. An informed consent form is signed to confirm understanding and agreement to participate.

    A series of assessments are conducted, including blood tests and a bone marrow aspirate, to establish baseline health status and eligibility for the study.

  2. Step 2

    Medication administration

    The study involves taking two medications: azacitidine and venetoclax. Azacitidine is provided in tablet form, with dosages of 200 mg and 300 mg, taken orally. Venetoclax is also taken orally as a film-coated tablet.

    The specific dosage and frequency of administration are determined by the study team based on individual health assessments. The treatment duration is adjusted to find the optimal dose and treatment length.

  3. Step 3

    Regular monitoring

    Throughout the study, regular visits are required for monitoring health status and response to the medication. This includes blood tests and other assessments to track progress and any side effects.

    Participants are required to be available for these assessments and to communicate any changes in health or side effects experienced during the study.

  4. Step 4

    End of cycle evaluation

    At the end of each treatment cycle, an evaluation is conducted to assess the response to the medication. This includes measuring overall response and any dose-limiting toxicity.

    The results of these evaluations help determine the continuation or adjustment of the treatment plan.

  5. Step 5

    Completion of study

    Upon completion of the study, a final assessment is conducted to evaluate the overall health status and response to the treatment.

    Participants may be required to undergo additional follow-up assessments to monitor long-term effects and outcomes.

Who can join the trial?

12 criteria

  • Must understand and voluntarily sign an informed consent form before starting any study procedures.
  • Must be able to communicate with the study team and follow the study requirements, including regular blood tests and bone marrow tests.
  • Females of childbearing potential must agree to take pregnancy tests before and during the study and use effective birth control methods or abstain from sex during the study and for a period after the study ends.
  • Male participants must agree to use effective birth control methods or abstain from sex during the study and for 3 months after the study ends. They should also learn about sperm preservation before starting treatment.
  • Must be at least 18 years old.
  • Must have a diagnosis of myelodysplastic syndromes (MDS) with specific characteristics as confirmed by a doctor.
  • Must not have received prior treatment for MDS with certain types of chemotherapy or experimental drugs.
  • Must not be eligible for certain intensive treatments due to personal health factors.
  • Must have an ECOG performance status of 0, 1, or 2, which means being fully active or having some limitations but able to care for oneself.
  • Must have a white blood cell count of 10 G/L or less. Treatment to lower this count is allowed before starting the study.
  • Must have adequate liver function, as shown by specific blood test results.
  • Must have adequate kidney function, as shown by a specific calculation of kidney performance.

Who cannot join the trial?

5 criteria

  • Patients who have already received treatment for their condition.
  • Patients who are eligible for a type of treatment called allogenic transplantation, which is a procedure where a person receives blood-forming stem cells from a genetically similar, but not identical, donor.
  • Patients who are not within the specified age range for the study.
  • Patients who are not part of the specified clinical trial groups.
  • Patients who are not considered part of the vulnerable population selected for this study.
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Investigated drugs

  • Oral Azacitidine (ONUREG®)

    is a medication used in this clinical trial to help treat higher-risk myelodysplastic syndromes, which are a group of disorders caused by poorly formed or dysfunctional blood cells. This medication is taken by mouth and works by interfering with the growth of cancer cells, which may slow down or stop their spread. The goal of using Oral Azacitidine in this trial is to find the best dose and treatment duration when used together with another medication.

  • Venetoclax (VENCLYXTO®)

    is another medication used in this trial. It is designed to help treat certain types of blood cancers by targeting and blocking a specific protein that cancer cells need to survive. By doing this, Venetoclax can help kill cancer cells and reduce the number of abnormal blood cells. In this study, Venetoclax is combined with Oral Azacitidine to see how well they work together in treating higher-risk myelodysplastic syndromes.

What is already known about the treatment

  • ONUREG

    ONUREG is an oral medication administered in pill form, currently being studied in clinical trials for its use in treating higher-risk myelodysplastic syndromes that have not been treated before and are not suitable for a type of stem cell transplant. It is being researched to determine the best dose and treatment duration when used in combination with another medication, Venetoclax. ONUREG works by interfering with the growth of cancer cells at the molecular level, helping to slow down or stop their spread. It is classified as a type of chemotherapy drug.

  • VENCLYXTO

    VENCLYXTO, also known as Venetoclax, is an oral medication taken in pill form, and it is being tested in combination with ONUREG for treating higher-risk myelodysplastic syndromes in patients who have not received prior treatment and cannot undergo a specific type of stem cell transplant. This medication works by targeting and blocking a protein that helps cancer cells survive, leading to their death. VENCLYXTO is classified as a targeted therapy, which means it specifically attacks cancer cells while minimizing damage to normal cells.

Investigated diseases

Higher-risk myelodysplastic syndromes – Myelodysplastic syndromes (MDS) are a group of disorders caused by poorly formed or dysfunctional blood cells. In higher-risk MDS, the bone marrow produces immature blood cells that do not mature properly, leading to a shortage of healthy blood cells. This condition can cause symptoms such as fatigue, infections, and bleeding due to low levels of red blood cells, white blood cells, and platelets. Over time, higher-risk MDS can progress, with an increased likelihood of transforming into acute myeloid leukemia (AML). The progression involves worsening blood cell counts and increased bone marrow blasts. The disease is characterized by its impact on blood cell production and the potential for significant complications.
Trial detailsLast updated 7 Oct 2026
Age18+ yearsPhasePhase ITrial ID2024-514876-41-00Protocol codeGFM-ONUVEN-MDSEstimated enrolment36 patientsSponsorGroupe Francophone Des Myelodysplasies

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