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Study on Ivosidenib and Azacitidine for Adults with Myelodysplastic Syndromes and IDH1 Mutation

Verified siteRegistered drugNo 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 condition known as myelodysplastic syndromes (MDS), which affects the blood and bone marrow. In this study, two treatments are being tested: ivosidenib, also known by its code name AG-120/S95031, and azacitidine, marketed as Vidaza. Both treatments are being used as monotherapies, meaning they are given alone without combining with other treatments. The purpose of the study is to evaluate the effectiveness of these treatments in patients who have not previously received a type of medication called hypomethylating agents and have a specific genetic change known as an IDH1 mutation.

Participants in the study will be randomly assigned to receive either ivosidenib or azacitidine. Ivosidenib is taken orally as a film-coated tablet, while azacitidine is administered as a powder mixed into a liquid for injection. The study will last for a period of up to 48 weeks, during which the effects of the treatments will be monitored. Some participants may receive a placebo, which is a substance with no active medication, to help compare the effects of the actual treatments.

The study aims to observe how well the treatments work in achieving remission, which means reducing or eliminating signs of the disease. It will also look at other outcomes such as overall survival, quality of life, and the rate at which the disease progresses to a more severe form called acute myeloid leukemia (AML). Participants' health and response to the treatments will be closely monitored throughout the study to gather comprehensive data on the safety and effectiveness of ivosidenib and azacitidine in treating myelodysplastic syndromes 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

    Joining the study

    Upon joining the study, you will be randomly assigned to one of two groups. One group will receive ivosidenib and the other will receive azacitidine. This assignment is random, meaning it is like flipping a coin to decide which group you will be in.

  2. Step 2

    Receiving ivosidenib

    If you are assigned to the ivosidenib group, you will take a 250 mg film-coated tablet by mouth. This medication is taken once daily. The duration of this treatment will be determined by the study team based on your response to the medication.

  3. Step 3

    Receiving azacitidine

    If you are assigned to the azacitidine group, you will receive the medication as an injection. This can be administered either through a vein (intravenous) or under the skin (subcutaneous). The dosage is 25 mg/ml, and the frequency and duration will be determined by the study team based on your response to the medication.

  4. Step 4

    Regular check-ups

    Throughout the study, you will have regular check-ups. These visits are important to monitor your health and the effects of the medication. The study team will conduct various tests and assessments during these visits.

  5. Step 5

    End of study participation

    Your participation in the study will continue until the study ends or until the study team decides it is best for you to stop. The estimated end date for the study is December 1, 2028. At the end of your participation, you will have a final visit to assess your health and discuss any further steps.

Who can join the trial?

12 criteria

  • Must have a diagnosis of HMA naive IDH1 R132 mutated MDS. This means the patient has not been treated with hypomethylating agents (HMA) and has a specific genetic mutation called IDH1 R132 in their myelodysplastic syndrome (MDS).
  • The diagnosis must be confirmed according to the World Health Organization (WHO) criteria.
  • For patients with moderate high, high, and very high-risk MDS, they are eligible regardless of blood counts, with blast counts (immature blood cells) between 0-19%.
  • For patients with low and moderate low-risk MDS, they must have low blood cell counts related to MDS, such as:
    • Platelets less than 100,000 per microliter (a measure of blood clotting cells).
    • Absolute neutrophil count (ANC) less than 1,000 per cubic millimeter (a measure of a type of white blood cell).
    • Hemoglobin (Hgb) less than 10 grams per deciliter (a measure of red blood cells).
    • For low and moderate low-risk MDS, they must have a blast count between 5-19%.
    • For low and moderate low-risk MDS, they must be eligible for HMA therapy. Very low-risk participants are not eligible.
    • The IDH1 R132 mutation must be confirmed either locally or centrally, with specific types of mutations (C/G/H/L/S).
    • Participants can be of any gender.
    • Participants must be within the age range of adults and older adults.
    • Participants from vulnerable populations are eligible.

Who cannot join the trial?

4 criteria

  • Patients who have previously been treated with a type of medication called a hypomethylating agent (HMA) cannot participate. These are drugs used to treat certain blood disorders.
  • Patients with a different type of blood disorder than the one being studied cannot participate.
  • Patients who are not within the specified age range for the study cannot participate.
  • Patients who are not able to give their own consent or are considered part of a vulnerable population cannot participate.
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Investigated drugs

  • Ivosidenib

    is a medication used in this clinical trial to treat patients with a specific type of blood disorder called myelodysplastic syndromes (MDS). This disorder is characterized by the bone marrow not producing enough healthy blood cells. Ivosidenib works by targeting and blocking a specific enzyme that is mutated in some patients with MDS. By blocking this enzyme, the medication helps to stop the growth of abnormal cells and allows the bone marrow to produce more healthy blood cells.

  • Azacitidine

    is another medication used in this trial for treating myelodysplastic syndromes (MDS). It is a type of chemotherapy that works by interfering with the growth of cancer cells, which are abnormal cells that grow uncontrollably. Azacitidine helps to slow down or stop the growth of these cells, allowing the bone marrow to produce more normal blood cells. This can help improve symptoms and quality of life for patients with MDS.

What is already known about the treatment

Ivosidenib – Ivosidenib is administered orally in the form of tablets and is currently being studied in clinical trials for its effectiveness in treating myelodysplastic syndromes (MDS) in patients who have not previously received hypomethylating agents. It is primarily indicated for MDS with a specific genetic mutation known as IDH1. At the molecular level, ivosidenib works by inhibiting the mutated IDH1 enzyme, which is involved in the production of an oncometabolite that can lead to abnormal cell growth. This medication is classified as a targeted therapy, specifically an IDH1 inhibitor, and is being explored for its potential to improve outcomes in patients with this genetic mutation.

Investigated diseases

Myelodysplastic Syndromes (MDS) – Myelodysplastic Syndromes are a group of disorders caused by poorly formed or dysfunctional blood cells. These disorders occur 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 anemia, infections, or bleeding. The progression of MDS can vary, with some cases remaining stable for years, while others may progress to more severe forms of blood disorders. The condition is characterized by a shortage of one or more types of blood cells, which can lead to symptoms like fatigue, shortness of breath, and increased risk of infections. MDS can sometimes transform into acute myeloid leukemia, a more aggressive type of blood cancer.
Trial detailsLast updated 2 Oct 2026
Age18+ yearsPhasePhase IIITrial ID2023-510155-37-00Protocol codeS095031-178Estimated enrolment48 patientsSponsorInstitut De Recherches Internationales Servier IRIS

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