This study involves people with newly diagnosed acute myeloid leukemia (a type of blood cancer where abnormal white blood cells grow rapidly in the bone marrow) who have a specific change in their genes called an NPM1 mutation. The treatment being tested includes Revumenib (also known by its code name SNDX-5613), which is an experimental medicine given as tablets by mouth, combined with standard intensive chemotherapy medicines. The chemotherapy medicines used in this study include daunorubicin hydrochloride, idarubicin hydrochloride, and cytarabine, which are given through a vein. Some participants will receive Revumenib along with chemotherapy, while others will receive placebo along with chemotherapy.
The purpose of this study is to find out if adding Revumenib to intensive chemotherapy helps people with this type of leukemia live longer without their disease getting worse compared to chemotherapy alone, and to see if this combination helps more people achieve complete remission with no detectable signs of cancer cells remaining in their bone marrow. During the study, participants will receive their assigned treatment and will be monitored regularly to check how well the treatment is working and to watch for any side effects. The study will track various outcomes including how long participants live, how long they remain in remission, and how many participants achieve complete remission with no measurable remaining disease.
The study will also carefully monitor the safety of the treatment by recording any unwanted effects that occur, checking blood test results regularly, and performing heart function tests and other examinations. Doctors will measure treatment success by looking at whether the cancer goes away completely, whether cancer cells can still be detected in the bone marrow or blood using special tests, and how long participants remain free of cancer. The study is expected to continue for several years to gather enough information about the long-term effects of this treatment combination.
1Screening and randomization
Before starting treatment, your medical history will be reviewed and tests will be performed to confirm eligibility for the study.
Blood tests will be performed to check kidney and liver function, as well as blood cell counts.
A heart function test will be performed using an echocardiogram or MUGA scan to ensure your heart is functioning adequately.
A pregnancy test will be required if applicable.
The presence of an NPM1 mutation in your leukemia cells will be confirmed through laboratory testing.
Your white blood cell count must be reduced to acceptable levels before starting the study treatment. Medications such as hydroxyurea or a single dose of cytarabine may be used to achieve this.
Once all tests are completed and eligibility is confirmed, you will be randomly assigned to receive either revumenib or placebo in combination with intensive chemotherapy. Neither you nor your doctor will know which group you are assigned to.
2Induction treatment cycle 1
This phase begins on Day 1 of the first cycle and is designed to reduce leukemia cells in your body.
You will receive intensive chemotherapy consisting of daunorubicin hydrochloride or idarubicin hydrochloride combined with cytarabine. These medications will be given through a vein.
Starting on Day 8 of the first cycle, you will begin taking either revumenib tablets or placebo tablets by mouth. The specific dose and frequency will be determined by your doctor.
You will continue taking the tablets daily as instructed throughout this cycle.
Regular blood tests will be performed to monitor your blood cell counts, kidney function, liver function, and other important health markers.
Your bone marrow will be tested to assess the response to treatment and check for measurable residual disease, which refers to small amounts of leukemia cells that may remain.
3Additional induction or consolidation cycles
Depending on your response to the first induction cycle, you may receive additional cycles of intensive chemotherapy.
Each cycle will include chemotherapy given through a vein, similar to the first cycle.
You will continue taking the assigned tablets (either revumenib or placebo) by mouth daily throughout these cycles.
Bone marrow tests will be repeated to assess whether you have achieved complete remission, which means that leukemia cells are no longer detectable in your bone marrow and blood counts have returned to normal levels.
Blood tests will continue regularly to monitor your overall health and detect any side effects from the treatment.
4Ongoing monitoring and follow-up
Throughout the study, you will have regular appointments to monitor your condition and assess the effectiveness of the treatment.
Blood samples will be collected periodically to check for measurable residual disease using molecular testing methods.
Your doctor will monitor you for any side effects or complications from the treatment, including changes in blood cell counts, kidney function, liver function, heart function, and overall well-being.
Your performance status will be assessed regularly to determine how well you are able to carry out daily activities.
The duration of your participation in the study will depend on your response to treatment and whether the leukemia returns.
If you achieve complete remission, you will continue to be monitored for signs of relapse, which is when leukemia cells return after treatment.
5Long-term follow-up
After completing the active treatment phase, you will enter a long-term follow-up period.
During this time, your doctor will continue to monitor your health status and check for any signs that the leukemia has returned.
The study will track important outcomes such as event-free survival, which measures the time from the start of treatment until treatment failure, relapse, or death, and overall survival, which measures the time from the start of treatment until death from any cause.
Information about any side effects that occur during or after treatment will be collected and recorded.
Your participation in the follow-up phase will help determine the long-term effectiveness and safety of the treatment approach used in this study.
Who Can Join the Study?
You must be at least 12 years old and weigh at least 40 kilograms at the time of signing the consent form
You must have newly diagnosed AML (a type of blood cancer called acute myeloid leukemia) that has not been treated before, and you must be suitable for receiving strong chemotherapy
Your cancer must have a specific genetic change called an NPM1 mutation, which will be confirmed through testing
Your white blood cell count (cells that fight infection) must be 25 or below on a specific measurement scale by day 8 of the first treatment cycle
Your life expectancy must be at least 3 months as determined by your doctor
Your ability to carry out daily activities must meet certain standards based on your age
Your kidneys must work well enough, with a measurement called creatinine clearance of at least 30, which shows how well your kidneys filter waste from your blood
Your liver must work adequately, with certain blood test results within acceptable ranges
Your heart must pump blood effectively, with an ejection fraction (a measurement of how well your heart pumps) of at least 50 percent
If you are a woman who can become pregnant, you must have a negative pregnancy test before starting treatment
If you are a woman who can become pregnant, you must agree to use highly effective birth control methods from the start of treatment through the required time period, and you must not donate eggs or undergo fertility treatments during this time
If you are a man, you must either be surgically sterile or agree to use barrier protection (condoms) from the start of treatment through the required time period, and you must not donate sperm during this time
You or your legal representative must be able and willing to provide written consent and follow study instructions
Who Cannot Join the Study?
The study does not list specific exclusion criteria in the provided information, which means the research team will determine if you are suitable to participate based on a detailed medical evaluation during the screening process.
Generally, clinical trials exclude patients who have other serious medical conditions that could interfere with the study treatment or make it unsafe for them to participate.
You may not be able to join if you are pregnant or breastfeeding, as the study medication could potentially harm an unborn baby or nursing infant.
If you have severe problems with your liver (the organ that cleans your blood and helps digest food) or kidneys (the organs that filter waste from your blood), you might not be eligible.
You may be excluded if you have an active infection that is not controlled with treatment.
If you have previously received treatment for this type of leukemia (a type of blood cancer), you might not qualify for this study which is specifically for newly diagnosed patients.
You cannot participate if you are allergic to any of the study medications or their ingredients.
If you are taking certain other medications that could interfere with the study drug, you may need to stop them or you may not be able to join the study.
You may be excluded if you have other types of cancer or a history of certain cancers.
If you have heart problems or abnormal heart rhythms that could be worsened by the study treatment, you might not be eligible.
Revumenib is an investigational medication being studied for the treatment of acute myeloid leukemia (AML) in patients who have a specific genetic change called an NPM1 mutation. This medication is being tested to see if it can help improve outcomes when given together with standard chemotherapy treatments.
Intensive Chemotherapy refers to strong cancer-fighting medications that are given to destroy leukemia cells. In this study, intensive chemotherapy is the standard treatment that all participants will receive, either alone or in combination with revumenib.
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 produce abnormal white blood cells that do not function properly. These abnormal cells multiply rapidly and crowd out healthy blood cells. The disease progresses quickly, with abnormal cells accumulating in the bone marrow and blood. As the condition advances, patients may experience fatigue, frequent infections, and easy bruising or bleeding due to the lack of healthy blood cells. The NPM1 mutation is a specific genetic change found in some cases of this disease.
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