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NCT04811560From ClinicalTrials.govRecruiting

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)

  • Acute Leukemia

At a glance

Phase
Phase 1
Study type
Interventional
Sponsor
Janssen Research & Development, LLC
Enrolment target
420
Started
19 May 2021
Main results due
26 February 2027
Study sites
102
Registry updated
25 September 2026

Can you take part?

  • Aged 2 years and over.
  • Open to any sex.
  • You need the condition being studied — healthy volunteers are not accepted.

These are the headline rules only. Every study has a longer list, and whether you are eligible is decided by the research team at the site — never by this page.

Read the full 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)

What this study is about

In the sponsor’s own words, from the registry.

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.

Study sites(102)

  • City of Hope

    Duarte, California, United States

    Recruiting

  • University of California Irvine Medical Center

    Orange, California, United States

    Completed

  • University of California San Francisco

    San Francisco, California, United States

    Recruiting

  • UCSF Benioff Children's Hospital

    San Francisco, California, United States

    Recruiting

  • University of Chicago

    Chicago, Illinois, United States

    Recruiting

  • St Francis Hospital & Health Centers Indiana Blood and Marrow Transplantation

    Indianapolis, Indiana, United States

    Recruiting

  • Norton Cancer Institute

    Louisville, Kentucky, United States

    Completed

  • Massachusetts General Hospital

    Boston, Massachusetts, United States

    Recruiting

  • Dana Farber Cancer Institute

    Boston, Massachusetts, United States

    Recruiting

  • Karmanos Cancer Institute

    Detroit, Michigan, United States

    Completed

  • Start Midwest

    Grand Rapids, Michigan, United States

    Recruiting

  • University of New Mexico

    Albuquerque, New Mexico, United States

    Recruiting

  • Roswell Park Comprehensive Cancer Center

    Buffalo, New York, United States

    Recruiting

  • NYU Langone Medical Center

    New York, New York, United States

    Recruiting

  • Montefiore Medical Center

    The Bronx, New York, United States

    Recruiting

  • Oregon Health And Science University

    Portland, Oregon, United States

    Recruiting

  • Fox Chase Cancer Center

    Philadelphia, Pennsylvania, United States

    Completed

  • Maine Health

    Providence, Rhode Island, United States

    Recruiting

  • University of Tennessee Medical Center

    Knoxville, Tennessee, United States

    Recruiting

  • Baylor University Medical Center

    Dallas, Texas, United States

    Recruiting

  • Houston Methodist Hospital

    Houston, Texas, United States

    Recruiting

  • MD Anderson

    Houston, Texas, United States

    Recruiting

  • San Antonio Methodist TX Transplant Physicians Group

    San Antonio, Texas, United States

    Recruiting

  • Virginia Commonwealth University

    Richmond, Virginia, United States

    Recruiting

  • Swedish Cancer Institute

    Seattle, Washington, United States

    Recruiting

  • Medical College of WI at Froedtert

    Milwaukee, Wisconsin, United States

    Recruiting

  • Monash Medical Centre

    Clayton, Australia

    Recruiting

  • Royal Perth Hospital

    Perth, Australia

    Recruiting

  • Gold Coast University Hospital

    Southport, Australia

    Recruiting

  • Ghent University Hospital

    Ghent, Belgium

    Recruiting

  • IDOR - Regional Brasilia

    Brasília, Brazil

    Recruiting

  • Liga Norte Riograndense Contra O Cancer

    Natal, Brazil

    Recruiting

  • Ministerio da Saude Instituto Nacional do Cancer

    Rio de Janeiro, Brazil

    Recruiting

  • Fundacao Faculdade de Medicina - Instituto do Cancer do Estado de Sao Paulo

    São Paulo, Brazil

    Recruiting

  • IDOR - Regional Sao Paulo

    São Paulo, Brazil

    Recruiting

  • Fundacao Antonio Prudente A C Camargo Cancer Center

    São Paulo, Brazil

    Recruiting

  • Vancouver Coastal Health

    Vancouver, British Columbia, Canada

    Recruiting

  • Princess Margaret Cancer Centre University Health Network

    Toronto, Ontario, Canada

    Recruiting

  • Hopital Maisonneuve Rosemont

    Montreal, Quebec, Canada

    Recruiting

  • Peking University First Hospital

    Beijing, China

    Recruiting

  • The First Hospital of Jilin University

    Changchun, China

    Recruiting

  • West China Hospital Si Chuan University

    Chengdu, China

    Recruiting

  • Nanfang Hospital of Southern Medical Hospital

    Guangzhou, China

    Completed

  • First Affiliated Hospital Medical School of Zhejiang University

    Hangzhou, China

    Recruiting

  • Qilu Hospital of Shandong University

    Jinan, China

    Recruiting

  • The First Affiliated Hospital of NanChang University

    Nanchang, China

    Recruiting

  • Zhongda Hospital Southeast University

    Nanjing, China

    Recruiting

  • Institute of Hematology and Blood Diseases Hospital

    Tianjin, China

    Recruiting

  • The First Affiliated Hospital of Wenzhou Medical University

    Wenzhou, China

    Recruiting

  • Union Hospital Tongji Medical College of Huazhong University of Science and Technology

    Wuhan, China

    Recruiting

  • Henan Cancer Hospital

    Zhengzhou, China

    Recruiting

  • Hopital Jean Minjoz

    Besançon, France

    Recruiting

  • Institut Paoli Calmettes

    Marseille, France

    Recruiting

  • CHU de Nantes hotel Dieu

    Nantes, France

    Recruiting

  • Hopital Saint Louis

    Paris, France

    Recruiting

  • Hopital trousseau- APHP

    Paris, France

    Recruiting

  • Centre Hospitalier Universitaire (CHU) de Bordeaux Hopital HautLeveque Centre Francois Magendie

    Pessac, France

    Completed

  • CHU Lyon Sud

    Pierre-Bénite, France

    Recruiting

  • Hôpitaux Universitaires de Strasbourg - Hôpital de Hautepierre

    Strasbourg, France

    Recruiting

  • Institut Universitaire du Cancer Toulouse Oncopole

    Toulouse, France

    Recruiting

Showing 60 of 102 sites — filter to narrow it down. 85 of the 102 sites on this study are recruiting right now — a site can stop enrolling while the study as a whole is still open.

Source: ClinicalTrials.gov record NCT04811560. Trialion does not run this study, is not paid to refer anyone to it, and cannot enrol you. Eligibility is always decided by the research team at the site.

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