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A Study of Ivosidenib and Azacitidine for Patients Over 55 with Acute Myeloid Leukemia and IDH1 Mutation in Complete Remission

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 study involves patients over 55 years of age with Acute Myeloid Leukemia, also known as AML, which is a type of cancer that affects the blood and bone marrow where blood cells are made. The patients in this study have a specific change in their genes called an IDH1 mutation. All participants must be in complete remission, meaning their cancer is no longer detectable after receiving intensive chemotherapy, which is a strong treatment using powerful medicines to kill cancer cells. The study will use two medications given by mouth: azacitidine, which is also known by the brand name Onureg, and ivosidenib, which is also known as Tibsovo. Both medications are tablets that patients will take orally.

The purpose of the study is to evaluate how long patients remain free from their cancer coming back when they receive both azacitidine and ivosidenib together. The study will look at whether this combination of medications can help keep the cancer from returning for 24 months. During the study, patients will receive the combination treatment for up to 24 months. The study will monitor how well the treatment works and watch for any unwanted effects that might occur. Doctors will also check the blood and bone marrow regularly to see if there are any signs of the cancer returning.

Throughout the study, patients will have regular check-ups and tests to assess their health and the status of their disease. Blood tests will be performed to monitor organ function, including the kidneys and liver, and to check blood cell counts. The study will track various outcomes including how long patients survive without their cancer returning, overall survival rates, and whether any signs of disease can be detected in the blood or bone marrow using special laboratory tests. Any side effects or health problems that occur during treatment will be carefully recorded and evaluated according to standard medical classifications.

The research process

The trial runs in 7 steps – from screening to follow-up. Each step says what happens and what the team monitors.

  1. Step 1

    Beginning treatment with study medications

    Your treatment will begin after you have completed at least 2 consolidation treatments following your initial intensive chemotherapy. This must occur within 17 weeks after achieving complete remission.

    You will receive two medications in tablet form: azacitidine (brand name Onureg) and ivosidenib (brand name Tibsovo). Both medications are taken by mouth.

    The azacitidine tablets are available in two strengths: 200 mg and 300 mg. The ivosidenib tablets are 250 mg. Your doctor will determine the specific doses you will take.

  2. Step 2

    Taking the medications regularly

    You will need to take both medications as prescribed by your doctor throughout the study period.

    The medications are film-coated tablets that should be swallowed whole.

    It is important to follow the dosing schedule provided by your doctor and take the medications at the same time each day.

  3. Step 3

    Regular monitoring visits

    You will attend regular appointments to monitor your health and how you are responding to the treatment.

    During these visits, blood tests will be performed to check your blood cell counts and organ function.

    Your doctor will check that your bone marrow function remains adequate, with specific attention to your white blood cell count and platelet count.

    Tests will also monitor your kidney function (measured by estimated glomerular filtration rate) and liver function (measured by specific enzymes called AST and ALT, as well as bilirubin levels).

  4. Step 4

    Monitoring for disease status

    Your doctor will perform tests to check if your leukemia remains in remission.

    Some of these tests may include checking for minimal residual disease (MRD), which means looking for very small amounts of leukemia cells that standard tests might not detect.

    These tests may be done using flow cytometry (a laboratory technique that examines cells) or molecular tests if you have a specific genetic marker called NPM1.

  5. Step 5

    Safety monitoring

    Throughout the study, your doctor will monitor you for any side effects or adverse events related to the medications.

    Any health changes or symptoms you experience will be recorded and evaluated according to a standardized classification system.

    You should report any new symptoms or health concerns to your doctor promptly.

  6. Step 6

    Contraception requirements during treatment

    If you are a woman of childbearing potential, you must use effective contraception starting from the beginning of the study and continuing for 180 days (approximately 6 months) after your last dose of study medication.

    If you are a woman of childbearing potential, you will need to have pregnancy tests performed at scheduled times during the study.

    If you are a woman, you must not breastfeed during the study and for one month after your last dose of study medication.

    If you are a woman, you must not donate eggs during the study and for 180 days after your last dose of study medication.

    If you are a man with a partner of childbearing potential, you must use contraception during the study and for 90 days (approximately 3 months) after your last dose of study medication.

    If you are a man, you must not donate sperm during the study and for 90 days after your last dose of study medication.

  7. Step 7

    Follow-up assessments

    Your health will be monitored at specific time points, including assessments at 12 months and 24 months after starting treatment.

    These assessments will evaluate whether your leukemia remains in remission and your overall health status.

    Your doctor will continue to track your progress even after you complete the active treatment phase.

Who can join the trial?

22 criteria

  • You must be 55 years of age or older at the time of signing the consent form
  • You must not have any psychological, family, social, or geographical conditions that would make it difficult to follow the study plan and attend follow-up visits
  • You must be able to take the study medication by mouth
  • You must have French social security or health insurance
  • You must sign a written consent form to participate in the study
  • You must have a confirmed new diagnosis of AML, which stands for Acute Myeloid Leukemia, a type of blood cancer
  • You must have a specific genetic change called IDH1 R132 mutation found at the time of your AML diagnosis
  • You must have achieved complete remission or complete remission with incomplete blood count recovery after initial treatment with intensive chemotherapy, within 17 weeks before joining the study. Complete remission means your cancer is no longer detectable
  • You must have received at least 2 additional rounds of chemotherapy treatment called consolidations, either with intermediate dose of a drug called cytarabine or with standard dose cytarabine combined with another drug called idarubicin
  • You must not be a candidate for Allo-HSCT, which stands for allogeneic hematopoietic stem cell transplantation, a procedure where stem cells from a donor are used to replace your bone marrow
  • Your bone marrow, the soft tissue inside bones that makes blood cells, must be working adequately with an absolute neutrophil count of at least 1.0 and a platelet count of at least 50. Neutrophils are white blood cells that fight infection, and platelets help your blood clot
  • Your kidneys must be working adequately with an estimated glomerular filtration rate of at least 30, which measures how well your kidneys filter waste from your blood
  • Your liver must be working adequately with AST and ALT levels no more than 2.5 times the upper normal limit. These are enzymes that show how well your liver is functioning
  • Your bilirubin level must be no more than 1.5 times the upper normal limit. Bilirubin is a substance produced when red blood cells break down
  • Your ECOG performance status must be less than 3. This is a score that measures how well you can perform daily activities, with lower numbers meaning better function
  • You must not be pregnant
  • If you are a woman who can become pregnant, you must agree to use birth control starting at screening and continuing throughout the study and for at least 180 days after taking the last dose of study medication
  • If you are a woman who can become pregnant, you must agree to take pregnancy tests as planned during the study
  • You must agree not to breastfeed starting at screening and throughout the study and for one month after taking the last dose of study medication
  • You must agree not to donate eggs starting at screening and throughout the study and for 180 days after taking the last dose of study medication
  • If you have a partner who can become pregnant, you must agree to use birth control starting at screening and continuing throughout the study and for at least 90 days after taking the last dose of study medication
  • You must agree not to donate sperm starting at screening and throughout the study and for 90 days after taking the last dose of study medication

Who cannot join the trial?

3 criteria

  • The study information provided does not list specific reasons why patients cannot participate in this clinical trial.
  • Generally, exclusion criteria help doctors determine if the study is safe and appropriate for each patient, but these specific details were not included in the available study information.
  • If you are interested in this study, a doctor conducting the trial would need to review your complete medical history to determine if you meet all requirements for participation.
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Investigated drugs

  • Ivosidenib

    is a targeted cancer medication that works by blocking a specific abnormal protein (called IDH1 mutation) found in certain leukemia cells. This medication helps prevent cancer cells from growing and multiplying.

  • Oral azacitidine

    (also known as Onureg) is a cancer medication taken by mouth that helps control leukemia by affecting how cancer cells grow and develop. It works by interfering with the genetic material of cancer cells, which helps keep the disease in remission after initial treatment with chemotherapy.

What is already known about the treatment

  • Ivosidenib

    Ivosidenib is an oral medication in tablet form that is currently being studied in clinical trials for treating patients with Acute Myeloid Leukemia who have a specific genetic change called IDH1 mutation. This drug works by blocking an abnormal enzyme produced by the mutated IDH1 gene, which helps cancer cells grow, thereby allowing normal blood cells to develop properly. It belongs to a class of medications called targeted cancer therapies or IDH1 inhibitors, and in this study it is being combined with another medication called azacitidine to help prevent the cancer from coming back after initial treatment.

  • Azacitidine

    Azacitidine, also known by the brand name Onureg, is an oral medication in tablet form used to help maintain remission in patients with Acute Myeloid Leukemia after they have completed intensive chemotherapy. This medication works by interfering with the way cancer cells grow and multiply, specifically by affecting how genes are turned on and off in abnormal blood cells, which helps restore normal blood cell production. It is classified as a cancer chemotherapy drug, specifically a type called a hypomethylating agent, and is approved for use in maintaining remission in certain leukemia patients.

Investigated diseases

Acute Myeloid Leukemia – Acute Myeloid Leukemia is a type of cancer that affects the blood and bone marrow. It occurs when the bone marrow begins to make abnormal white blood cells called myeloblasts, which do not develop into healthy blood cells. These abnormal cells multiply rapidly and crowd out normal blood cells in the bone marrow and bloodstream. As the disease progresses, patients may experience symptoms such as fatigue, frequent infections, easy bruising, and bleeding due to low levels of healthy blood cells. The condition develops quickly and requires prompt medical attention. In some cases, the disease is associated with specific genetic mutations, such as changes in the IDH1 gene, which affect how cells grow and function.
Trial detailsLast updated 7 Oct 2026
Age18+ yearsPhasePhase IITrial ID2025-521322-14-00Protocol codeFILO-AML-01-MIVONUEstimated enrolment60 patientsSponsorFrench Innovative Leukemia Organization (F.I.L.O.).

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