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Testing bleximenib for treating acute leukemia

A Phase 1/2, First-in-Human Study of the Menin-KMT2A (MLL1) Inhibitor Bleximenib in Participants With Acute Leukemia (cAMeLot-1)

PHASE1; PHASE2 · Janssen Research & Development, LLC · NCT04811560

This study is testing a new drug called bleximenib to see if it can safely help teenagers and adults with tough cases of acute leukemia.

Quick facts

PhasePHASE1; PHASE2
Study typeInterventional
Enrollment420 (estimated)
Ages2 Years and up
SexAll
SponsorJanssen Research & Development, LLC (industry)
Drugs / interventionsblinatumomab, immunotherapy, prednisone
Locations103 sites (Goodyear, Arizona and 102 other locations)
Trial IDNCT04811560 on ClinicalTrials.gov

What this trial studies

This study aims to evaluate the safety, tolerability, and efficacy of bleximenib in patients with acute leukemia. It consists of two phases: the first phase focuses on determining the recommended dose through dose escalation and expansion, while the second phase assesses the drug's effectiveness at this dose. Participants include adolescents and adults with relapsed or refractory acute leukemia who have specific genetic alterations. The study is designed to provide insights into a new treatment option for this challenging condition.

Who should consider this trial

Good fit: Ideal candidates include adolescents aged 12-18 and adults over 18 with relapsed or refractory acute myeloid leukemia or acute lymphoblastic leukemia harboring specific genetic alterations.

Not a fit: Patients with acute leukemia who do not have the specified genetic alterations or those who are not relapsed or refractory may not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment could offer a new therapeutic option for patients with acute leukemia who have limited treatment choices.

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

Eligibility criteria

Show full inclusion / exclusion criteria 
Inclusion Criteria:

Phase 1:

* 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

Phase: 2

* 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

For Both Phase 1 and 2:

* 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 Criteria:

* 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
* Exclusion criteria related to stem cell transplant: a. Received prior treatment with allogenic bone marrow or stem cell transplant \<=3 months before the first dose of study treatment ; b. Has evidence of graft versus host disease; c. Received donor lymphocyte infusion \<=1 month before the first dose of study treatment; d. Requires immunosuppressant therapy (exception: daily doses \<=10 milligrams (mg) prednisone or equivalent are allowed for adrenal replacement)
* 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)

Where this trial is running

Goodyear, Arizona and 102 other locations

+53 more sites — see ClinicalTrials.gov for the full list.

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 →

Conditions: Acute Leukemia

Last reviewed 2026-05-15 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.