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Bleximenib combined with therapies for treating acute myeloid leukemia

A Phase 1b Study of Bleximenib in Combination With AML-Directed Therapies for Participants With Acute Myeloid Leukemia Harboring KMT2A or NPM1 Alterations

Phase 1 Interventional Janssen Research & Development, LLC · NCT05453903

This study is testing a new drug called bleximenib combined with standard treatments to see if it can help teenagers and adults with acute myeloid leukemia that hasn't responded to other treatments.

Quick facts

PhasePhase 1
Study typeInterventional
Enrollment200 (estimated)
Ages12 Years and up
SexAll
SponsorJanssen Research & Development, LLC Industry-sponsored
Locations32 sites (Birmingham, Alabama and 31 other locations)
Trial IDNCT05453903 on ClinicalTrials.gov

What this trial studies

This study aims to identify the recommended Phase 2 dose of bleximenib when used alongside established therapies for acute myeloid leukemia (AML). It will evaluate the safety and tolerability of this combination treatment in patients with AML, particularly those who are relapsed or refractory. The study includes adolescent participants and focuses on specific genetic alterations associated with AML. The approach involves administering bleximenib in conjunction with other AML-directed therapies such as venetoclax, azacitidine, cytarabine, and daunorubicin or idarubicin.

Who should consider this trial

Good fit: Ideal candidates include adolescents aged 12 to 18 with a diagnosis of AML, particularly those with relapsed or refractory disease and specific genetic alterations.

Not a fit: Patients with AML who do not meet the eligibility criteria, such as those older than 18 or without the specified genetic alterations, may not benefit from this study.

Why it matters

Potential benefit: If successful, this study could provide a new effective treatment option for patients with acute myeloid leukemia.

How similar studies have performed: Other studies have shown promise with similar combination therapies in AML, indicating potential for success in this approach.

Eligibility criteria

Show full inclusion / exclusion criteria 
Inclusion Criteria:

* 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)
* Diagnosis of AML according to World Health Organization (WHO) criteria: a) De novo or secondary AML; b) relapsed/refractory (Arm A only); c) harboring KMT2A, NPM1, NUP98, or NUP214 alterations; d) Participants may receive emergency leukapheresis and/or cytarabine as cytoreductive therapy according to local practice guidelines
* Pretreatment clinical laboratory values meeting the following criteria -listed below: White blood cell (WBC) count: less than or equal to (\<=) 25\*10\^9 per liter (/L), adequate liver and renal function
* 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)
* 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
* Must sign an informed consent form (ICF) indicating participant (or their legally authorized representative) understands the purpose of the study and procedures required for the study and is willing to participate in the study
* Willing and able to adhere to the prohibitions and restrictions specified in this protocol

Exclusion Criteria:

* Acute promyelocytic leukemia, diagnosis of Down syndrome associated leukemia or juvenile myelomonocytic leukemia according to WHO 2016 criteria
* Leukemic involvement of the central nervous system
* Recipient of solid organ transplant
* Cardiovascular disease that is uncontrolled, increases 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: (a) Myocardial infarction; (b) Severe or unstable angina; (c) Clinically significant cardiac arrhythmias, including bradycardia (\<50 beats per minute); (d) Uncontrolled (persistent) hypertension: (example, blood pressure greater than \[\>\] 140/90 millimeters of mercury \[mm Hg\]; (e) Acute neurologic events such as stroke or transient ischemic attack, intracranial or subarachnoid hemorrhage, intracranial trauma; (f) Venous thromboembolic events (example, pulmonary embolism) within 1 month prior to the first dose of study treatment ;(g) Congestive heart failure (NYHA class III to IV); (h) Pericarditis or clinically significant pericardial effusion; (i) Myocarditis; (j) Endocarditis (k) Clinically significant hypokalemia, hypomagnesemia, hypocalcemia (corrected for hypoalbuminemia)
* 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
* Pulmonary compromise that requires the need for supplemental oxygen use to maintain adequate oxygenation
* Participants with diagnosis of Fanconi anemia, Kostmann syndrome, Shwachman diamond syndrome, or any other known bone marrow failure syndrome

Where this trial is running

Birmingham, Alabama and 31 other locations

Study contacts

How to participate

  1. Review the eligibility criteria above with your treating physician.
  2. Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
  3. Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.
View on ClinicalTrials.gov → Find more trials like this →
Conditions Leukemia, Myeloid, Acute
Last reviewed 2026-06-13 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.