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Contact Information:

Mrs Ellice Marwood
RA-JanssenUKRegistry@ITS.JNJ.com


Dr Sponsor Contact
medinfo@its.jnj.com


Study Location:

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Be Part of Research - Trial Details - A study of bleximenib in combination with acute myeloid leukemia-directed therapies
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A study of bleximenib in combination with acute myeloid leukemia-directed therapies

Not Recruiting

Open to: All Genders

Age: Mixed

Manchester London Oxford

Medical Conditions

Acute myeloid leukemia

This information is provided directly by researchers, and we recognise that it isn't always easy to understand. We are working with researchers to improve the accessibility of this information. In some summaries, you may come across links to external websites. These websites will have more information to help you better understand the study.


Leukaemia (cancer of the white blood cells) is diagnosed as acute when it progresses quickly and aggressively and usually requires immediate treatment. Acute myeloid leukaemia (AML) affects the myeloid cells that fight bacterial infections and other conditions. KMT2A, NPM1, or NUP gene alterations can be associated with AML. Treatment options for AML are limited, survival rates are poor, and many patients are ineligible for standard chemotherapy treatments due to toxicity. The study purpose is to determine the recommended phase II study dose(s) of bleximenib in combination with AML-directed therapies and further to evaluate the safety and tolerability of bleximenib in combination with AML-directed therapies at these dose(s).

Start dates may differ between countries and research sites. The research team are responsible for keeping the information up-to-date.  

The recruitment start and end dates are as follows:

04 Oct 2022 07 Jul 2025

AML patients will be enrolled into 1 of 3 study arms. Initially, patients with relapsed/refractory AML will be enrolled (Arm A). Once it has been determined that the combination treatment is safe in these patients, patients with newly diagnosed AML will be enrolled based on eligibility for intensive chemotherapy: ineligible (Arm B) or eligible (Arm C). Bleximenib will be given once or twice daily on a 28-day cycle, with cohort-specific AML-directed therapies.

The study has a screening (up to 28 days), treatment, and follow-up phase. The treatment phase will continue for as long as participants receive benefits from the combination treatment and/or until their participation is ended for any reason. While taking study treatment and during follow-up, participants will come to the clinic for health exams and tests.

The follow-up period will depend on the response to the study treatment and can last up to 18 months.


Male and female participants with AML, relapsed/refractory or newly diagnosed, with KMT2A , NPM1, NUP98, or NUP214 gene alterations.

You can take part if:


Current inclusion criteria as of 25/11/2025:
1. Adolescent participants (defined as greater than or equal to [>=] 12 and less than [<] 18 years of age) are only eligible for the relapsed/refractory (R/R) cohort (Arm A, cohort A4)
2. Diagnosis of AML according to World Health Organization (WHO) 2016 criteria:
2.1. De novo or secondary AML
2.2. Relapsed /refractory (Arm A)
2.3. Harboring NPM1, KMT2Am NUP98 or NUP214 alterations
3. Pretreatment clinical laboratory values meeting the following criteria -listed below: White blood cell (WBC) count: less than or equal to <=25 x 10^9 per liter (/L), adequate liver and renal function
4. Eastern Cooperative Oncology Group (ECOG) performance status grade of 0, 1 or 2. Adolescent participants only: Performance status >70 by Lansky scale (for participants <16 years of age) or >70 Karnofsky scale (for participants >16 years of age)
5. A female of childbearing potential must have a negative highly sensitive serum beta-human chorionic gonadotropin at screening and within 48 hours prior to the first dose of study treatment
6. Must sign an informed consent form (ICF) indicating participant (or their legally authorised representative) understands the purpose of the study and procedures required for the study and is willing to participate in the study.
7. Willing and able to adhere to the prohibitions and restriction


You may not be able to take part if:


Current exclusion criteria as of 25/11/2025:1. Acute promyelocytic leukemia, diagnosis of Downs Syndrome associated lekaemia or diagnosis of Down syndrome associated leukemia according to WHO 2016 criteria2. Leukemic involvement of the central nervous system3. Recipient of solid organ transplant4. Cardiovascular disease that is uncontrolled, increases the risk for Torsades de Pointes or that was diagnosed within 6 months prior to the first dose of study treatment including, but not limited to:4.1. Myocardial infarction4.2. Severe or unstable angina4.3. Clinically significant cardiac arrhythmias, including bradycardia (less than [<] 50 beats per minute)4.4. Uncontrolled (persistent) hypertension: (example, blood pressure greater than [>] 140/90 millimeters of mercury [mm Hg]4.5. Acute neurologic events such as stroke or transient ischemic attack, intracranial or subarachnoid hemorrhage, intracranial trauma4.6. Venous thromboembolic events (for example, pulmonary embolism) within 1 month prior to the first dose of study treatment (uncomplicated Grade less than or equal to [≤]2 deep vein thrombosis is not considered exclusionary)4.7. Congestive heart failure (NYHA class III to IV); (h) Pericarditis or clinically significant pericardial effusion4.8. Myocarditis4.9. Endocarditis4.10. Clinically significant hypokalemia, hypomagnesemia, hypocalcemia (corrected for hypoalbuminemia)5. Any toxicity (except for alopecia, stable peripheral neuropathy, thrombocytopenia, neutropenia, anemia) from previous anticancer therapy that has not resolved to baseline or to grade 1 or less6. Pulmonary compromise that requires the need for supplemental oxygen use to maintain adequate oxygenation7. Participants with diagnosis of Fanconi anemia, Kostmann syndrome, Shwachman diamond syndrome, or any other known bone marrow failure syndrome

Previous exclusion criteria:1. Acute promyelocytic leukemia according to WHO 2016 criteria 2. Leukemic involvement of the central nervous system 3. Recipient of solid organ transplant 4. Cardiovascular disease that is uncontrolled, increases the risk for Torsades de Pointes or that was diagnosed within 6 months prior to the first dose of study treatment including, but not limited to:4.1. Myocardial infarction4.2. Severe or unstable angina4.3. Clinically significant cardiac arrhythmias, including bradycardia (less than [<] 50 beats per minute)4.4. Uncontrolled (persistent) hypertension: (example, blood pressure greater than [>] 140/90 millimeters of mercury [mm Hg]4.5. Acute neurologic events such as stroke or transient ischemic attack, intracranial or subarachnoid hemorrhage, intracranial trauma4.6. Venous thromboembolic events (for example, pulmonary embolism) within 1 month prior to the first dose of study treatment (uncomplicated Grade less than or equal to [≤]2 deep vein thrombosis is not considered exclusionary)4.7. Congestive heart failure (NYHA class III to IV); (h) Pericarditis or clinically significant pericardial effusion4.8. Myocarditis4.9. Endocarditis 4.10. Clinically significant hypokalemia, hypomagnesemia, hypocalcemia (corrected for hypoalbuminemia) 5. Any toxicity (except for alopecia, stable peripheral neuropathy, thrombocytopenia, neutropenia, anemia) from previous anticancer therapy that has not resolved to baseline or to grade 1 or less 6. Pulmonary compromise that requires the need for supplemental oxygen use to maintain adequate oxygenation


Below are the locations for where you can take part in the trial. Please note that not all sites may be open.

  • The Christie NHS Foundation Trust
    550 Wilmslow Road Withington
    Manchester
    M20 4BX
  • University College London Hospitals NHS Foundation Trust
    250 Euston Road
    London
    NW1 2PG
  • Oxford University Hospitals NHS Foundation Trust
    Churchill Hospital Old Road Headington
    Oxford
    OX3 7LE

Taking bleximenib in combination with AML-directed therapies may improve AML. However, this cannot be guaranteed because bleximenib is still under investigation as a treatment and it is not known whether the combination study treatment will work. It is also possible that known effects from the individual medications may worsen when given in combination.
Participants may experience some benefit from participation in the study that is not due to receiving study treatment but due to regular visits and assessments monitoring overall health.
Participation may help other people with myeloid in the future.
1. Blood cell count effects
2. Changes to heart rhythm
3. Fertility effects
4. Tumour Lysis Syndrome (when large numbers of leukaemia cells die in a short period of time)
5. Differentiation Syndrome (DS involves a large, rapid release of cytokines (immune substances) from leukemia cells after treatment with anticancer drugs)
6. Infections
There may also be other potential risks associated with bleximenib in combination with AML directed therapies. Side effects from the drugs or procedures used in this study may be mild to severe and even life-threatening, and they can vary from person to person. All possible discomforts, side effects, and risks related to the study treatment and procedures will be explained in full in the participant information sheet and discussed during the informed consent process.
To minimise the risk associated with taking part in the study, participants are frequently evaluated for any side effects. Participants are educated to report any such events to the study doctor who will provide appropriate medical care. Any serious side effects that are reported to the sponsor are thoroughly reviewed by the global study team. The study doctor will tell participants as soon as possible about any new information that might make them change their mind about being in the study, such as new risks. There are no costs to participants to be in the study. The sponsor will pay for the study treatment and tests that are part of the study. The participant will receive reasonable reimbursement for study-related costs (e.g., travel/parking fees/occasional meals).

Dr Sponsor Contact
medinfo@its.jnj.com


Mrs Ellice Marwood
RA-JanssenUKRegistry@ITS.JNJ.com



The study is sponsored by Janssen Pharmaceutica NV and funded by Janssen Research and Development.



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Read full details for Trial ID: ISRCTN12278036

Or CPMS: 52246

Last updated 16 February 2026

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