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RecruitingPhase 1 / Phase 2

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

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

Who can join

As published by the study on ClinicalTrials.gov. "Not stated" means the study hasn't said — ask the study team.

Age
2 Years and older
Sex
All sexes
Healthy volunteers
Not accepted
Compensation
Not stated

Other requirements are listed under Eligibility below. Only the study team can confirm whether you can join.

What to expect

No one can promise a trial will help you personally — that is what the research is trying to find out.

What participation involves

Not stated in the study description — ask the study team about visits and procedures.

Compensation
Not stated
  • Study-related exams and procedures are typically provided at no cost. This is how many trials work in general — confirm the details for this study with the study team.
  • Participants are monitored closely by the research team throughout the study.
  • Some participants may receive access to an investigational treatment, which is still being studied.
  • Your participation helps researchers learn whether this approach could help future patients.

About this study

In plain language

This study tests bleximenib for acute leukemia, a fast-growing blood cancer, with certain gene changes. It aims to find the dose for Phase 2, check safety and side effects, and see how well it works. It is for adults and some children whose leukemia came back or did not respond to treatment.

The purpose of this study is to determine the recommended Phase 2 dose(s) (RP2D\[s\]) of bleximenib in phase 1 Part 1 (Dose Escalation) and to determine the safety and tolerability at RP2D in Phase 1 Part 2 (Dose expansion). The purpose of the Phase 2 part of the study is to evaluate the efficacy of bleximenib at the RP2D.

Compensation is read from the study's own description and may be missing — always confirm with the study team.

Eligibility

Who can join

  • Phase 1: Adults age 18 or older may join. The children’s group is for ages 2 to under 18.
  • Phase 1: Leukemia must have come back or not responded to treatment. Available treatments must have been used up or not be suitable.
  • Phase 1: Leukemia must have changes in the KMT2A, NPM1, NUP98, or NUP214 genes.
  • Phase 2: You must be older than 18 and have acute myeloid leukemia (AML) that came back or did not respond to treatment. AML must meet WHO 2022 diagnosis rules and have a KMT2A rearrangement or NPM1 mutation.
  • Both phases: White blood cell count must be 20 billion per liter or less. Kidney filtering rate must be at least 30 mL/min for adults or 40 mL/min for children.
  • Both phases: You must meet activity and self-care score rules: ECOG 0–2, or for children, at least 70 on the Lansky scale if under 16 or Karnofsky scale if 16 or older.
  • Both phases: If you can become pregnant, you must have a negative blood pregnancy test at screening and within 48 hours before the first dose.
  • Both phases: Follow all study birth control rules. Do not donate or freeze sperm or eggs during the study or for 90 days afterward for males and 6 months afterward for females, counted from the last dose.

Who can't join

  • Certain leukemia types: acute promyelocytic leukemia, Down syndrome–related leukemia, or juvenile myelomonocytic leukemia, as defined by WHO 2016 rules.
  • Active disease in the brain or spinal cord.
  • A past solid organ transplant.
  • A heart test showing a long electrical recovery time (QTcF): 450 milliseconds or more for males or 470 or more for females. A family history of long QT syndrome also excludes you.
  • A bone marrow or stem cell transplant from a donor within 3 months before the first dose, or a donor lymphocyte infusion within 1 month before it.
  • Graft-versus-host disease, when donated cells attack your body, or a need for medicines that weaken the immune system. Up to 10 mg of prednisone daily, or an equal dose of another medicine, is allowed for adrenal hormone replacement.
  • Cancer immunotherapy within 4 weeks before enrollment, or blinatumomab within 2 weeks before enrollment.
  • Other cancer treatment within 4 weeks before enrollment, or within the time for the drug’s level to fall by half five times, whichever is shorter.

Simplified for readability. The official criteria below are what the study team uses.

Official eligibility 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)

Study sites (26)

CA

IL

IN

KY

MA

MI

NM

NY

OR

PA

RI

TN

TX

VA

WA

WI