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Study of Tamibarotene and Azacitidine Treatment in Newly Diagnosed Adults with RARA-positive Higher-risk Myelodysplastic Syndrome (MDS)

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What is this trial about?

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

This study focuses on patients with Higher-risk Myelodysplastic Syndrome, a condition where bone marrow does not produce enough healthy blood cells. The study will test a combination of two medications: tamibarotene (also known as SY-1425) and azacitidine, comparing them to treatment with azacitidine and placebo. The purpose is to determine how well the combination therapy works in newly diagnosed adult patients whose disease tests positive for a specific marker called RARA.

The treatment involves taking tamibarotene tablets by mouth and receiving azacitidine through injections under the skin or into a vein. Azacitidine is given at a dose of 75 mg per square meter of body surface for 7 days, while tamibarotene is given as a daily tablet. Some patients will receive a placebo tablet instead of tamibarotene, but all patients will receive azacitidine.

The study will track how many patients achieve complete remission (when signs of cancer disappear) and will also monitor how long patients survive, whether they need fewer blood transfusions, and how the treatment affects their quality of life. Doctors will regularly check patients' blood counts and bone marrow to assess how well the treatment is working and watch for any side effects.

The research process

The trial runs in 4 steps – from screening to follow-up. Each step says what happens and what the team monitors.

  1. Step 1

    Initial assessment

    Your eligibility for the study will be confirmed through medical tests and examinations

    A bone marrow test will be performed to verify that bone marrow blasts are greater than 5%

    Laboratory tests will check your liver and kidney function

    If you are a woman who can become pregnant, a pregnancy test will be conducted

  2. Step 2

    Treatment assignment

    You will be randomly assigned to one of two treatment groups

    Group 1: Tamibarotene tablets plus Azacitidine injections

    Group 2: Placebo tablets plus Azacitidine injections

    Neither you nor your doctor will know which tablet treatment group you are in

  3. Step 3

    Treatment administration

    Azacitidine will be given as an injection under the skin or into a vein

    You will take either Tamibarotene or placebo tablets by mouth

    Regular blood tests and medical examinations will monitor your response to treatment

    Your quality of life will be assessed through questionnaires

  4. Step 4

    Monitoring period

    Your condition will be regularly monitored for any changes in disease status

    Blood cell counts will be checked regularly

    Side effects will be tracked through laboratory tests and physical examinations

    The study will continue until February 2031

Who can join the trial?

15 criteria

  • Must be at least 18 years old when signing the consent form
  • Must test positive for RARA (a specific protein marker) using a special test
  • Must be newly diagnosed with higher-risk myelodysplastic syndrome (HR-MDS) with specific risk scores:
    • Very High (score greater than 6)
    • High (score 4.5 to 6)
    • Intermediate (score 3 to 4.5)
    • Must have more than 5% blast cells (immature blood cells) in bone marrow
    • Must have an ECOG Performance Status of 2 or less (ability to carry out daily activities)
    • Must have adequate organ function, including:
      • Acceptable liver function tests
      • Kidney function showing filtration rate of at least 30 mL/min
      • Women who can become pregnant must have a negative pregnancy test
      • Must be willing to:
        • Attend all scheduled study visits
        • Follow treatment plans
        • Complete required laboratory tests
        • Use two methods of birth control, including a barrier method
        • Must be able to understand and sign an informed consent document

Who cannot join the trial?

15 criteria

  • Prior treatment with hypomethylating agents (drugs that affect DNA)
  • Active or prior acute myeloid leukemia (a type of blood cancer)
  • History of other cancers within the past 3 years, except for successfully treated skin cancer or early-stage cancer
  • Severe heart conditions, including:
    • Uncontrolled high blood pressure
    • Recent heart attack
    • Unstable heart disease
    • Severe liver problems or active liver disease
    • Severe kidney problems requiring dialysis
    • Active, uncontrolled infections
    • Known HIV infection or active hepatitis B or C
    • Pregnancy or breastfeeding
    • Mental conditions that could interfere with following study procedures
    • Participation in other clinical trials within 30 days before this study
    • Known allergic reactions to study medications
    • Life expectancy less than 3 months
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Investigated drugs

  • Tamibarotene

    A medication that belongs to a class of drugs called retinoids. It works by targeting specific genes and proteins in cancer cells. In this trial, it is being studied for treating patients with higher-risk myelodysplastic syndrome, a condition where the bone marrow doesn't produce enough healthy blood cells.

  • Azacitidine

    A chemotherapy medication that helps bone marrow produce normal blood cells and kills abnormal cells in the bone marrow. It is commonly used to treat myelodysplastic syndrome. This medication works by interfering with the DNA of cancer cells, which helps prevent them from growing and dividing.

  • Placebo

    An inactive substance that contains no medication. It is used as a control to help determine if the experimental treatment is effective. Some patients in this study receive a placebo along with azacitidine to compare results with those who receive tamibarotene plus azacitidine.

What is already known about the treatment

  • Tamibarotene

    An oral retinoid medication that specifically targets the retinoic acid receptor alpha (RARA) in blood cells. It is being investigated in clinical trials for the treatment of higher-risk myelodysplastic syndrome (MDS) in combination with azacitidine, particularly in patients who are RARA-positive. The drug works by binding to and activating RARA receptors, which helps regulate blood cell development and differentiation. This medication belongs to the class of retinoids and is administered in tablet form.

  • Azacitidine

    A chemotherapy medication administered by injection or intravenous infusion that is used to treat myelodysplastic syndrome (MDS) and other blood disorders. It works by preventing the production of DNA in cancer cells and helps bone marrow produce normal blood cells, while also killing abnormal cells in the blood and bone marrow. This drug belongs to the class of medications called hypomethylating agents and is considered a standard treatment option for higher-risk MDS patients.

Investigated diseases

Myelodysplastic Syndrome – A group of blood disorders where the bone marrow does not produce enough healthy blood cells. The condition occurs when blood-forming cells in the bone marrow become abnormal and create defective cells. This leads to low levels of one or more types of blood cells: red blood cells, white blood cells, or platelets. The condition primarily affects older adults and develops gradually over time. People with this condition may experience fatigue, frequent infections, and easy bruising or bleeding due to the lack of proper blood cell production.
Trial detailsLast updated 7 Oct 2026
Age18+ yearsPhasePhase IIITrial ID2023-510361-97-00Protocol codeSY-1425-301Estimated enrolment550 patientsSponsorSyros Pharmaceuticals Inc.

sourced from the EU Clinical Trials Register and site verification

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