This study focuses on Acute Myeloid Leukemia (AML), a type of cancer that affects the blood and bone marrow. The study specifically targets patients with newly diagnosed AML who have certain genetic changes called KMT2A rearrangements or NPM1 mutations and who cannot receive intensive chemotherapy. The purpose of this study is to determine if adding a medication called bleximenib to a combination of venetoclax and azacitidine (VEN+AZA) works better than using venetoclax and azacitidine alone.
The treatment involves taking these medications in cycles. Some participants will receive bleximenib along with venetoclax and azacitidine, while others will receive venetoclax and azacitidine with a placebo. This is a double-blind study, which means neither the participants nor the researchers will know which treatment group a participant is in until the study is completed.
The study will monitor how participants respond to treatment by tracking various health indicators and will assess whether the addition of bleximenib improves complete remission (when there are no detectable cancer cells) and overall survival in patients with this type of leukemia.
1Start of Trial
After joining the study, you will be randomly assigned to one of two treatment groups. One group will receive bleximenib, venetoclax, and azacitidine, while the other group will receive venetoclax, azacitidine, and a placebo (an inactive substance that looks like bleximenib).
This is a double-blind study, which means neither you nor your doctor will know which treatment group you are in.
2Treatment Administration
Your treatment will consist of 28-day cycles of medication:
Venetoclax (Venclyxto): You will take this medication in tablet form. Venetoclax comes as 50 mg and 100 mg film-coated tablets.
Azacitidine: This medication will be given as an injection under your skin or through an intravenous (IV) line. The formulation used is Azacitidine betapharm 25 mg/mL powder for suspension for injection.
Bleximenib or matching placebo: Depending on your assigned group, you will receive either bleximenib (30 mg, 50 mg, or 100 mg) or a matching placebo in the form of film-coated tablets.
3Monitoring and Assessments
Throughout the trial, you will have regular visits to monitor your health and the effectiveness of the treatment.
These visits will include blood tests to check your blood cell counts and assess how well your kidneys and liver are functioning.
Bone marrow assessments will be performed periodically to evaluate how the leukemia is responding to treatment.
Your doctor will also monitor you for any side effects or complications related to the treatment.
4Treatment Duration
You will continue receiving treatment in 28-day cycles as long as you are benefiting from it and not experiencing unacceptable side effects.
The study will track two main outcomes: complete remission (CR), which means no detectable cancer cells, and overall survival (OS), which is how long patients live after starting treatment.
5End of Treatment
Your participation in the treatment phase will end if:
Your disease progresses despite treatment
You experience unacceptable side effects
You decide to withdraw from the study
Your doctor decides it’s in your best interest to stop the study treatment
The study itself is terminated
6Follow-up Period
After completing or discontinuing treatment, you will enter a follow-up period.
During this time, your health status will continue to be monitored through periodic check-ups and assessments.
The entire study is expected to run until 2029, though your active participation may be shorter depending on your individual treatment course.
Who Can Join the Study?
You must be 18 years of age or older (or the legal age of majority in your location, whichever is greater) when you sign the informed consent.
You must have previously untreated acute myeloid leukemia (a type of blood cancer) with specific genetic mutations called KMT2Ar or NPM1m, with at least 10% blasts (immature blood cells) according to 2022 criteria.
You must not be eligible for intensive chemotherapy due to one of these reasons:
You are 75 years or older and your doctor determines you’re not eligible, with an ECOG performance status (a measure of your ability to perform daily activities) of 0-2
You are between 18 and 74 years old with at least one of these health issues:
ECOG performance status of 2
Severe heart condition
Severe lung condition
Kidney impairment
Other health condition that makes you unsuitable for intensive chemotherapy
You must have adequate kidney and liver function before randomization, based on specific blood test results.
Your white blood cell (WBC) count must be less than 25 × 10^9/L (less than 25 billion white blood cells per liter of blood).
Who Cannot Join the Study?
You cannot participate if you are under 18 years old.
You cannot participate if you have received previous treatment for acute myeloid leukemia (a type of blood cancer affecting bone marrow cells).
You cannot participate if you have acute promyelocytic leukemia (a specific subtype of acute myeloid leukemia).
You cannot participate if you have an active central nervous system involvement with leukemia (cancer cells in the brain or spinal cord).
You cannot participate if you have another type of cancer that requires treatment during the study period.
You cannot participate if you have severe heart problems or uncontrolled high blood pressure.
You cannot participate if you have severe liver or kidney disease.
You cannot participate if you have an active, uncontrolled infection.
You cannot participate if you are pregnant or breastfeeding.
You cannot participate if you have had major surgery within 4 weeks before starting the study treatment.
You cannot participate if you are currently enrolled in another investigational drug study.
Bleximenib is a medication being studied for the treatment of acute myeloid leukemia (AML). It is designed to target specific genetic changes in cancer cells, particularly in patients with KMT2A rearrangements or NPM1 mutations. This medication is being tested to see if it can improve outcomes when added to other treatments.
Venetoclax is a medication that works by blocking a protein called BCL-2, which helps cancer cells survive. By blocking this protein, venetoclax can help trigger cancer cells to die. It is commonly used in combination with other medications to treat various blood cancers, including acute myeloid leukemia.
Azacitidine is a medication that affects how genes are expressed in cells. It helps bone marrow produce normal blood cells and kills abnormal cells in the bone marrow. Azacitidine is commonly used to treat certain types of blood disorders and cancers, including acute myeloid leukemia, particularly in patients who cannot tolerate intensive chemotherapy.
Acute Myeloid Leukemia – A rapidly developing cancer that originates in the bone marrow from myeloid stem cells that should normally develop into mature blood cells. In this disease, abnormal white blood cells accumulate in the bone marrow and interfere with the production of normal blood cells. As the disease progresses, these abnormal cells can spread to the bloodstream and potentially to other parts of the body including the lymph nodes, spleen, liver, and central nervous system. The disease typically develops quickly, over days or weeks, causing symptoms such as fatigue, shortness of breath, easy bruising and bleeding, and increased risk of infection. Without the proper production of healthy blood cells, individuals with this condition experience decreasing levels of red blood cells, platelets, and normal white blood cells.
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