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Study on the Effectiveness and Safety of Oral Azacitidine for Patients with Low or Intermediate-Risk Myelodysplastic Syndrome

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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 condition known as Myelodysplastic Syndrome (MDS), which affects the blood and bone marrow. Specifically, the study targets individuals with low- or intermediate-risk MDS, as classified by a system called the International Prognostic Scoring System-Revised (IPSS-R). The treatment being tested is an oral medication called Azacitidine, available in 200 mg and 300 mg tablets. This medication is being compared to a placebo, with both groups receiving the best supportive care available.

The purpose of the study is to evaluate the safety and effectiveness of Azacitidine in treating MDS. The study is divided into two parts. In the first part, different doses of the medication will be tested to find the most effective and safe dose. In the second part, the chosen dose will be compared to a placebo to see how well it works in improving the condition. Participants will take the medication orally, and the study will monitor their response over several cycles of treatment.

Throughout the study, participants will be closely observed to assess any changes in their condition and to ensure their safety. The study aims to determine if Azacitidine can help achieve a complete response, which means a significant improvement in blood cell counts and overall health. The results will help in understanding the potential benefits of this treatment for people with low- or intermediate-risk Myelodysplastic Syndrome.

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

    Joining the study

    Upon joining the study, you will be randomly assigned to one of two groups. One group will receive the medication oral azacitidine, and the other group will receive a placebo. A placebo is a substance with no active medication, used to compare the effects of the actual drug.

  2. Step 2

    Medication administration

    If you are in the group receiving oral azacitidine, you will take either a 200 mg or 300 mg tablet. The exact dosage will be determined during the study. The medication is taken by mouth, and you will follow the specific instructions provided by the study team regarding how often and for how long you need to take the medication.

  3. Step 3

    Monitoring and assessments

    Throughout the study, you will undergo regular monitoring and assessments. This includes checking your vital signs, such as blood pressure and heart rate, and performing laboratory tests to evaluate your health and the effects of the medication. These assessments help ensure your safety and measure the effectiveness of the treatment.

  4. Step 4

    Evaluation of results

    The study aims to evaluate the complete response (CR) within 6 cycles of treatment. A complete response means that there are no signs of the disease after treatment. The study will also assess other outcomes, such as the duration of response and any side effects you may experience.

  5. Step 5

    Completion of the study

    At the end of the study, you will have a final evaluation to assess your overall health and the outcomes of the treatment. The study team will provide you with information about your participation and any next steps, if necessary.

Who can join the trial?

4 criteria

  • Participants must be 18 years of age or older at the time of signing the consent form.
  • Participants must have a documented diagnosis of Myelodysplastic Syndrome (MDS) according to the 2016 World Health Organization (WHO) classification. This should meet the International Prognostic Scoring System-Revised (IPSS-R) classification of low- or intermediate-risk disease.
  • Participants must have at least one cytopenia, which means a low count of blood cells. This can include anemia (low red blood cells), thrombocytopenia (low platelets), or neutropenia (low white blood cells).
  • Participants must have an Eastern Cooperative Oncology Group (ECOG) performance status of 0, 1, or 2. This is a scale used to assess how a patient's disease is progressing, including their level of functioning and ability to care for themselves.

Who cannot join the trial?

12 criteria

  • Patients with a different type of blood disorder than the one being studied cannot participate.
  • Patients who have had another type of cancer in the past, unless it was treated and has not come back for at least 5 years, cannot participate.
  • Patients who are currently receiving treatment for another type of cancer cannot participate.
  • Patients who have had a heart attack or severe heart problems in the past 6 months cannot participate.
  • Patients with uncontrolled high blood pressure cannot participate.
  • Patients with severe liver disease cannot participate.
  • Patients with severe kidney disease cannot participate.
  • Patients who are pregnant or breastfeeding cannot participate.
  • Patients who are not willing to use effective birth control during the study cannot participate.
  • Patients who have participated in another clinical trial within the last 30 days cannot participate.
  • Patients who have an allergy to the study medication or similar drugs cannot participate.
  • Patients with any other medical condition that the study doctors think would make it unsafe for them to participate cannot participate.
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Investigated drugs

Oral Azacitidine (Oral-Aza, ONUREG®) is a medication used in this clinical trial to treat a condition called myelodysplastic syndrome (MDS), which affects the blood and bone marrow. This medication is taken by mouth and works by helping to slow down or stop the growth of abnormal blood cells. The goal of using Oral Azacitidine in this study is to see if it can improve the health of patients with low- or intermediate-risk MDS by reducing the number of abnormal cells and improving blood cell counts. The study aims to find the best dose that balances effectiveness and safety for patients.

What is already known about the treatment

Oral Azacitidine (ONUREG®) – This medication is taken by mouth and is being studied for its effectiveness and safety in treating a blood disorder called Myelodysplastic Syndrome (MDS), specifically in patients with low or intermediate risk according to a specific scoring system. Currently, it is in the clinical trial phase, where researchers are determining the best dose and evaluating its benefits and risks. Oral Azacitidine works by altering the way certain genes are expressed in cells, which can help control the abnormal growth of blood cells. It belongs to a class of drugs known as hypomethylating agents, which are used to treat certain types of blood cancers.

Investigated diseases

Myelodysplastic Syndrome (MDS) – Myelodysplastic Syndrome is a group of disorders caused by poorly formed or dysfunctional blood cells. It occurs when something goes wrong in the bone marrow, where blood cells are produced. Over time, the bone marrow becomes less effective at producing healthy blood cells, leading to a shortage of red blood cells, white blood cells, and platelets. This can result in symptoms such as fatigue, infections, and easy bruising or bleeding. The disease can progress slowly or quickly, and in some cases, it may transform into acute myeloid leukemia. The progression of MDS varies greatly among individuals, depending on the specific type and severity of the syndrome.
Trial detailsLast updated 7 Oct 2026
Age18+ yearsPhasePhase IVTrial ID2022-500479-29-00Protocol codeCA055-026Estimated enrolment306 patientsSponsorCelgene Corp.

sourced from the EU Clinical Trials Register and site verification

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