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Phase 1/2 Study of Bleximenib for Acute Leukemia

This study is testing a drug called bleximenib for people with acute leukemia that has come back or hasn't responded to other treatments. You might be able to join if you have specific genetic changes in your leukemia, such as KMT2A or NPM1. The main goals are to find the safest and most effective dose of bleximenib (Phase 1) and then to see how well it works (Phase 2). The study is currently in an unclear status and plans to enroll up to 420 participants.

Study design
This is a Phase 1/2 study, meaning it first looks at safety and dosing, then at how well the treatment works. It plans to enroll up to 420 participants.
What's involved
Not specified in the trial record.
Compensation
Not stated in the trial record.
Follow-up
Participants will be followed for safety for up to 4 years and 9 months.

AI-generated from the public study record. Only the study team can confirm whether you're eligible — confirm details with them before making decisions.

NCT04811560

A Phase 1/2 Study of Bleximenib in Participants With Acute Leukemia (cAMeLot-1)

Recruiting
PHASE1Ages 2+InterventionalTreatment
Janssen Research & Development, LLC
~420 participants
Updated 2026-08-28 on ClinicalTrials.gov
What's tested:Bleximenib

At a glance

Recruiting sites
86 of 102 listed sites are recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
Phase 1: Number of Participants with Adverse Events (AEs) as a Measure of Safety and Tolerability
Measured over Up to 4 years and 9 months
+3 more outcomes measured

Conditions

Acute Leukemia

Explore related trials

Acute LeukemiaAcute Myeloid LeukemiaKMT2A GeneNPM1 GeneNUP214 GeneNUP98 Gene

Where it's being run

102 sites across 30 states
France13
China12
Japan11
Spain9
United Kingdom8
Brazil6
California4
Texas4

Study leadership

  • Janssen Research & Development, LLC Clinical Trial · STUDY_DIRECTOR · Janssen Research & Development, LLC

Who to contact

Study Contact
[email protected]
844-434-4210
Email the study team

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Do you actually qualify for this trial?

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Eligibility criteria

Inclusion

Age 2 years to less than (\<) 18 years of age (pediatric cohort only), all other cohorts 18 years and above
Relapsed or refractory (R/R) acute leukemia and has exhausted, or is ineligible for, available therapeutic options
Acute leukemia harboring histone-lysine N-methyltransferase 2A (KMT2A), nucleophosmin 1 gene (NPM1) or nucleoporin 98 gene or nucleoporin 214 gene (NUP98 or NUP214) alterations
Participants greater than 18 years are eligible
Must have had an initial diagnosis of acute myeloid leukemia (AML) per the WHO 2022 classification criteria and have relapsed/refractory disease
AML harboring KMT2A-r (gene rearrangement/translocation) or NPM1 mutations only
Pretreatment clinical laboratory values meeting the following criteria: (a) Hematology: white blood cell (WBC) count \<= 20\*10\^9/liter (L) and (b) renal function; For adult participants, estimated or measured glomerular filtration rate \>= 30 milliliter per minute (mL/min) per four variable MDRD equation. For pediatric participants an estimated or measured glomerular filtration rate \>=40 mL/min per the CKiD (Chronic Kidney Disease in Children) Schwartz formula
Eastern Cooperative Oncology Group (ECOG) performance status grade of 0, 1 or 2. Pediatric 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
Participant must agree to all protocol required contraception requirements and avoid sperm or egg donations or freezing for future reproductive use while on study and for 90 days (males) or 6 months (females) after the last dose of study treatment

Exclusion

Acute promyelocytic leukemia, diagnosis of Down syndrome associated leukemia or juvenile myelomonocytic leukemia according to World Health Organization (WHO) 2016 criteria
Active central nervous system (CNS) disease
Prior solid organ transplantation
QTc according to Fridericia's formula (QTcF) for males \>= 450 millisecond (msec) or for females \>= 470 msec. Participants with a family history of Long QT syndrome are excluded
Prior cancer immunotherapy within 4 weeks prior to enrollment or blinatumomab within 2 weeks prior to enrollment. Additional prior cancer therapies must not be given within 4 weeks prior to enrollment or 5 half-lives of the agent (whichever is shorter)

What this trial measures

  • Phase 1: Number of Participants with Adverse Events (AEs) as a Measure of Safety and TolerabilityUp to 4 years and 9 months

    An AE is any untoward medical occurrence in a participant participating in a clinical study that does not necessarily have a causal relationship with the pharmaceutical/biological agent under study.

  • Phase 1: Number of Participants with AEs by SeverityUp to 4 years and 9 months

    Severity will be graded according to the National Cancer Institute Common Terminology Criteria for Adverse Events (NCI-CTCAE) version 5.0. Severity scale ranges from Grade 1 (Mild) to Grade 5 (Death). Grade 1= Mild, Grade 2= Moderate, Grade 3= Severe, Grade 4= Life-threatening and Grade 5= Death related to adverse event.

  • Phase 1: Part 1: Percentage of Participants with Dose-Limiting Toxicity (DLT)Up to 28 days Cycle 1

    Percentage of participants with DLT will be assessed accordingly to national cancer institute common terminology criteria for adverse events (NCI-CTCAE) version 5.

  • Phase 2: Rate of Complete Remission or Complete Remission with Partial Hematologic Recovery (CR/CRh)Up to 4 years and 9 months

    Rate of CR/CRh is defined as the percentage of participants achieving a CR or CRh at any time post-treatment.