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Study on the Effectiveness of Ivosidenib in Patients with IDH1 Mutated Myelodysplastic Syndrome

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 Syndrome (MDS), which is a group of disorders caused by poorly formed or dysfunctional blood cells. The study is specifically looking at patients with a mutation in the IDH1 gene. The treatment being tested is a medication called AG-120, also known by its code name S95031, which is taken as a film-coated tablet. This medication is being evaluated for its effectiveness in treating MDS with the IDH1 mutation.

The purpose of the study is to determine how well patients respond to the treatment with AG-120. The study will also assess the safety and tolerability of the medication. Participants will receive the treatment over a period of time, and their health will be monitored to see how the disease responds to the medication. Some participants may receive a placebo, which is a substance with no active medication, to compare the effects of the treatment.

Throughout the study, participants will have regular check-ups to monitor their condition and any side effects they may experience. The study aims to gather information on the overall response to the treatment, including how long the response lasts and any changes in the disease. The study will also look at factors that might predict how well a patient responds to the treatment. This research is important for understanding how to better treat Myelodysplastic Syndrome with the IDH1 mutation and improve patient outcomes.

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 assessment

    Upon joining the study, an initial assessment is conducted to confirm eligibility. This includes reviewing medical history and performing necessary tests to ensure the presence of the IDH1 mutation in blood or marrow.

    Eligibility criteria include being 18 years or older, having a specific performance status, and meeting certain health conditions related to kidney, liver, and heart function.

  2. Step 2

    Treatment initiation

    The treatment involves the administration of AG-120 (also known as ivosidenib), which is a 250 mg film-coated tablet taken orally.

    The dosage and frequency of administration are determined by the study protocol and are aimed at evaluating the response rate and safety of the medication.

  3. Step 3

    Monitoring and follow-up

    Regular follow-up visits are scheduled to monitor the patient's response to the treatment. This includes assessing overall hematological response at 3 and 6 months.

    Safety and tolerability are evaluated, particularly for patients in cohort C, using established criteria for non-hematological toxicities.

  4. Step 4

    Response evaluation

    The primary goal is to determine the overall response, including complete response (CR), partial response (PR), and stable disease with hematological improvement (HI).

    Secondary evaluations include response duration, time to progression, overall survival, and any adverse events or toxicities.

  5. Step 5

    Study completion

    The study is estimated to conclude by August 31, 2026. Upon completion, final assessments are conducted to evaluate the long-term effects and outcomes of the treatment.

Who can join the trial?

13 criteria

  • Must be **18 years or older**.
  • Must have an **ECOG performance status** of 0 to 2. This is a scale that measures how well you can perform daily activities.
  • Women who can have children must have a **negative pregnancy test** within 7 days before starting the treatment.
  • Women and men who can have children must agree to use **two effective forms of birth control** or not have sexual intercourse during the study and for 3 months after the last dose of the study drug.
  • Men must agree to use a **condom** during sexual activity with women who can have children during the study and for 3 months after the last dose. They should also learn about **sperm preservation** before starting treatment.
  • Must have a **myelodysplastic syndrome** as defined by specific medical criteria, including certain risk levels and previous treatment responses.
  • Must have an **IDH1 mutation** in blood or bone marrow before starting the treatment.
  • Must have **normal kidney function**, which means certain blood test results are within a specific range.
  • Must have **normal liver function**, which means certain liver test results are within a specific range.
  • Must have an **adequate heart function**, with a measurement called cardiac ejection fraction greater than 40%.
  • Must not be **resistant to platelet transfusions**.
  • Must understand and voluntarily **sign a consent form** to participate in the study.
  • Must be able to **attend all study visits** and follow the study requirements.

Who cannot join the trial?

5 criteria

  • Patients who do not have a **myelodysplastic syndrome** with an **IDH1 mutation**. **Myelodysplastic syndrome** is a condition where the bone marrow does not produce enough healthy blood cells. An **IDH1 mutation** is a specific change in a gene that can affect cell growth.
  • Patients who are not within the specified age range for the study. The study is open to certain age groups only.
  • Patients who are not part of the specified clinical trial groups. The study is designed for specific groups of patients.
  • Patients who are not male or female, as the study includes both genders.
  • Patients who are not considered part of a vulnerable population. A **vulnerable population** includes groups of people who may have additional risks or needs.
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Investigated drugs

AG-120: This medication is an IDH1 inhibitor used in the study to treat patients with Myelodysplastic Syndrome who have a specific mutation in the IDH1 gene. The goal of using AG-120 is to assess how well patients respond to the treatment, including achieving complete or partial remission or stable disease. Additionally, the study aims to evaluate the safety and tolerability of AG-120 in these patients.

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 myelodysplastic syndrome with IDH1 mutations. It is not yet widely used in standard medical practice, as it is still under investigation. The main therapeutic indication for AG-120 is to target and inhibit the mutated IDH1 enzyme, which is involved in the abnormal production of blood cells. By blocking this enzyme, AG-120 aims to restore normal cell function and growth. It belongs to the pharmacological class of enzyme inhibitors, specifically targeting the IDH1 mutation.

Investigated diseases

Myelodysplastic Syndrome with IDH1 Mutated – This is a type of blood disorder where the bone marrow does not produce enough healthy blood cells. It is characterized by the presence of mutations in the IDH1 gene, which can affect the normal development of blood cells. Over time, this condition can lead to a decrease in red blood cells, white blood cells, and platelets, causing symptoms like fatigue, infections, and easy bruising. The disease progresses as the bone marrow becomes less effective at producing healthy cells, potentially leading to more severe blood-related issues. The mutation in the IDH1 gene can influence the behavior of the disease, making it distinct from other forms of myelodysplastic syndromes.
Trial detailsLast updated 7 Oct 2026
Age18+ yearsPhasePhase IITrial ID2024-515352-20-00Protocol codeIDIOME StudyEstimated enrolment48 patientsSponsorGroupe Francophone Des Myelodysplasies

sourced from the EU Clinical Trials Register and site verification

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