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Study of Ivosidenib and Azacitidine for Patients Aged 18 and Older with Untreated Acute Myeloid Leukemia with IDH1 Mutation

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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 type of blood cancer called Acute Myeloid Leukemia (AML) in patients who have not received treatment before and have a specific genetic change known as an IDH1 mutation. The study is testing a new treatment approach that combines two medications: AG-120 (also known as Ivosidenib) and Azacitidine. AG-120 is taken as a tablet, while Azacitidine is given as an injection. The trial will compare the effects of this combination with the effects of Azacitidine alone.

The purpose of the study is to see if the combination of AG-120 and Azacitidine can improve the time patients live without their disease getting worse, compared to those receiving Azacitidine with a placebo. A placebo is a substance with no active medication, used to help understand the real effects of the treatment. Participants in the study will be randomly assigned to receive either the combination of AG-120 and Azacitidine or Azacitidine with a placebo. The study will be conducted in a way that neither the participants nor the researchers know who is receiving which treatment, to ensure unbiased results.

Throughout the study, participants will receive regular check-ups and monitoring to assess their health and the effects of the treatment. The study aims to provide valuable information on whether the combination of AG-120 and Azacitidine can offer a better treatment option for patients with previously untreated Acute Myeloid Leukemia with an IDH1 mutation.

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

    Randomization

    Upon joining the study, you will be randomly assigned to one of two groups. One group will receive the combination of AG-120 (ivosidenib) and azacitidine, while the other group will receive a placebo and azacitidine. This process is called randomization and ensures that the study results are unbiased.

  2. Step 2

    Medication administration

    If assigned to the AG-120 group, you will take a 250 mg film-coated tablet of AG-120 orally. This medication is taken daily.

    All participants will receive azacitidine, which is administered as a suspension for injection. The dosage is 25 mg/ml, and it is given intravenously. The frequency and duration of azacitidine administration will be determined by the study protocol and your healthcare provider.

  3. Step 3

    Monitoring and assessments

    Throughout the study, regular monitoring will occur to assess your health and the effectiveness of the treatment. This includes blood tests and bone marrow sampling to evaluate your response to the medication.

    Quality of life assessments will be conducted at various points during the study to understand the impact of the treatment on your daily life.

  4. Step 4

    End of treatment evaluation

    At the end of the treatment period, a comprehensive evaluation will be conducted to determine the outcome of the therapy. This includes assessing event-free survival, which measures the time until treatment failure, relapse, or death from any cause.

  5. Step 5

    Follow-up

    After completing the treatment, follow-up visits will be scheduled to monitor your health and any long-term effects of the treatment. These visits are crucial for ensuring your well-being and gathering data for the study.

Who can join the trial?

16 criteria

  • Must be at least 18 years old and meet at least one of the following conditions that make intensive chemotherapy unsuitable:
    • 75 years or older
    • ECOG PS of 2, which is a measure of daily living abilities
    • Severe heart problems, like heart failure needing treatment or certain heart conditions
    • Severe lung problems, like reduced lung function
    • Poor kidney function, with creatinine clearance less than 45 mL/minute
    • High bilirubin levels, more than 1.5 times the normal limit
    • Any other health issue that the study doctor thinks makes intensive chemotherapy unsuitable
    • If female and able to have children, must have a negative pregnancy test before starting the study. Must use two effective forms of birth control during the study and for 90 days after the last dose.
    • Must have Acute Myeloid Leukemia (AML) that has not been treated before, according to specific criteria. Cannot have AML only outside the bone marrow or blood.
    • Must have a specific IDH1 mutation in the bone marrow or blood, confirmed by a central lab test.
    • Must have an ECOG PS score between 0 and 2, indicating the ability to perform daily activities.
    • Must have adequate liver function, with certain liver enzyme levels not exceeding specific limits, unless due to certain conditions.
    • Must have adequate kidney function, with creatinine levels not exceeding specific limits or clearance above 30 mL/min.
    • Must agree to have regular blood and bone marrow samples taken.
    • Must be able to understand and willing to sign a consent form to participate in the study.
    • Must be willing to complete quality of life assessments during and after the study treatment.

Who cannot join the trial?

4 criteria

  • Patients who have already received treatment for their **Acute Myeloid Leukemia**. This means if you have been treated for this type of blood cancer before, you cannot participate.
  • Patients without an **IDH1 Mutation**. This is a specific change in the genes that is required for the study.
  • Patients who are not within the specified age range. The study is only for certain age groups.
  • Patients who belong to a vulnerable population. This means people who might need special protection or care cannot join the study.
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Investigated drugs

  • AG-120

    This is an investigational medication being studied for its potential to treat acute myeloid leukemia (AML) in patients who have a specific genetic mutation known as IDH1. The medication is designed to target and inhibit the activity of the mutated IDH1 enzyme, which may help to slow down or stop the growth of cancer cells.

  • Azacitidine

    This is a medication used in the treatment of certain types of blood cancers, including acute myeloid leukemia (AML). It works by interfering with the growth of cancer cells, which can help to reduce the number of abnormal blood cells and improve blood cell counts. In this study, azacitidine is used in combination with AG-120 to evaluate their effectiveness together in treating AML.

What is already known about the treatment

AG-120 – This medication is administered orally and is currently being studied in clinical trials for its effectiveness in treating acute myeloid leukemia with an IDH1 mutation. It is not yet widely used in standard medical practice but shows promise in ongoing research. AG-120 works by inhibiting the mutated IDH1 enzyme, which is involved in the production of an oncometabolite that contributes to cancer cell growth. It is classified as an enzyme inhibitor, specifically targeting the IDH1 mutation in cancer cells.

Investigated diseases

Acute Myeloid Leukemia with an IDH1 Mutation – This is a type of cancer that affects the blood and bone marrow, characterized by the rapid growth of abnormal white blood cells. It specifically involves a mutation in the IDH1 gene, which plays a role in cell metabolism. The disease typically progresses quickly, leading to symptoms such as fatigue, frequent infections, and easy bruising or bleeding. As the abnormal cells accumulate, they interfere with the production of normal blood cells. This can result in anemia, increased risk of infections, and impaired blood clotting. The condition is considered rare and requires specialized medical attention.
Trial detailsLast updated 7 Oct 2026
Age18+ yearsPhasePhase IIITrial ID2024-514309-73-00Protocol codeAG120-C-009Estimated enrolment141 patientsSponsorInstitut De Recherches Internationales Servier IRIS

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