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New pill shows promise for Hard-to-Treat leukemia

NCT ID NCT04811560

Disease control Sponsor: Janssen Research & Development, LLC Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now This study
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 25, 2026 · Last updated Aug 28, 2026 · Updated 5 times

Summary

This study tests a new oral drug called bleximenib for people with acute leukemia that has come back or not responded to treatment. The drug targets specific genetic changes in the cancer cells. The trial has two phases: first to find the safest dose, then to see how well it works in up to 420 participants.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance
bleximenib (a menin-KMT2A inhibitor pill)
What this could lead to
If successful, this could lead to a new targeted treatment option for people with acute leukemia who have certain genetic mutations and have not responded to other therapies.
What could go wrong
This is an early phase 1/2 trial, so safety and the right dose are still being figured out. It may not work for everyone, and side effects are possible.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 1/2

Runs two stages together: safety and dose first, then whether the treatment works.

Participants

About 420 people

The number the study aims to enrol. It can still change while the study runs.

Started

May 2021

Expected to finish

Sep 2030

An estimate. End dates often move.

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

2 years and older

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

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)

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Conditions

The condition(s) this trial relates to.

acute leukemia acute lymphoblastic leukemia acute myeloid leukemia

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Acute leukemia Acute leukemias Acute lymphoblastic leukemia Acute myeloid leukemia

How to take part

Only the study team decides who joins. These are the ways to reach them.

  1. The places running it

    86 sites in 12 countries. The list below names each one and where it is.

  2. The official record

    ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.

    Open the record ↗

  3. A doctor treating you

    A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.

Contacts and locations

Locations

  • Asan Medical Center

    RECRUITING

    Seoul, 05505, South Korea

  • Baylor University Medical Center

    RECRUITING

    Dallas, Texas, 75246, United States

  • CHRU Nancy Brabois

    RECRUITING

    Vandœuvre-lès-Nancy, 54511, France

  • CHRU de Nancy - Hopitaux de Brabois

    RECRUITING

    Vandœuvre-lès-Nancy, 54500, France

  • CHU Bretonneau

    RECRUITING

    Tours, 37044, France

  • CHU Lyon Sud

    RECRUITING

    Pierre-Bénite, 69310, France

  • CHU de Nantes hotel Dieu

    RECRUITING

    Nantes, 44093, France

  • Carmel Medical Center

    RECRUITING

    Haifa, 3436212, Israel

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

    COMPLETED

    Pessac, 33604, France

  • China Medical University Hospital

    RECRUITING

    Taichung, 404327, Taiwan

  • City of Hope

    RECRUITING

    Duarte, California, 91010, United States

  • Clinica Univ. de Navarra

    RECRUITING

    Pamplona, 31008, Spain

  • Dana Farber Cancer Institute

    RECRUITING

    Boston, Massachusetts, 02115, United States

  • First Affiliated Hospital Medical School of Zhejiang University

    RECRUITING

    Hangzhou, 310009, China

  • Fox Chase Cancer Center

    COMPLETED

    Philadelphia, Pennsylvania, 19111, United States

  • Fukushima Medical University Hospital

    ACTIVE_NOT_RECRUITING

    Fukushima, 960 1295, Japan

  • Fundacao Antonio Prudente A C Camargo Cancer Center

    RECRUITING

    São Paulo, 01509 900, Brazil

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

    RECRUITING

    São Paulo, 01246 000, Brazil

  • Ghent University Hospital

    RECRUITING

    Ghent, 9000, Belgium

  • Gold Coast University Hospital

    RECRUITING

    Southport, 4211, Australia

  • Gunmaken Saiseikai Maebashi Hospital

    COMPLETED

    Maebashi, 371-0821, Japan

  • Gustave Roussy

    RECRUITING

    Villejuif, 94805, France

  • Guys and St Thomas NHS Foundation Trust

    RECRUITING

    London, SE1 9RT, United Kingdom

  • Hadassah University Hospita Ein Kerem

    RECRUITING

    Jerusalem, 9112001, Israel

  • Henan Cancer Hospital

    RECRUITING

    Zhengzhou, 450003, China

  • Hokkaido University Hospital

    ACTIVE_NOT_RECRUITING

    Sapporo, 060-8648, Japan

  • Hopital Jean Minjoz

    RECRUITING

    Besançon, 25000, France

  • Hopital Maisonneuve Rosemont

    RECRUITING

    Montreal, Quebec, H1T 2M4, Canada

  • Hopital Saint Louis

    RECRUITING

    Paris, 75010, France

  • Hopital trousseau- APHP

    RECRUITING

    Paris, 75012, France

  • Hosp Clinic de Barcelona

    RECRUITING

    Barcelona, 08036, Spain

  • Hosp Clinico Univ de Salamanca

    RECRUITING

    Salamanca, 37007, Spain

  • Hosp Univ Fund Jimenez Diaz

    RECRUITING

    Madrid, 28040, Spain

  • Hosp Univ Vall D Hebron

    RECRUITING

    Barcelona, 08035, Spain

  • Hosp. Clinico Univ. de Valencia

    RECRUITING

    Valencia, 46010, Spain

  • Hosp. Gral. Univ. Gregorio Maranon

    RECRUITING

    Madrid, 28007, Spain

  • Hosp. Virgen Del Rocio

    RECRUITING

    Seville, 41013, Spain

  • Hospital Infantil Universitario Nino Jesus

    RECRUITING

    Madrid, 28009, Spain

  • Houston Methodist Hospital

    RECRUITING

    Houston, Texas, 77030-2740, United States

  • Hôpitaux Universitaires de Strasbourg - Hôpital de Hautepierre

    RECRUITING

    Strasbourg, 67098, France

  • IDOR - Regional Brasilia

    RECRUITING

    Brasília, 70390-140, Brazil

  • IDOR - Regional Sao Paulo

    RECRUITING

    São Paulo, 01401 002, Brazil

  • Institut Paoli Calmettes

    RECRUITING

    Marseille, 13009, France

  • Institut Universitaire du Cancer Toulouse Oncopole

    RECRUITING

    Toulouse, 31059, France

  • Institute of Hematology and Blood Diseases Hospital

    RECRUITING

    Tianjin, 301609, China

  • Kanazawa University Hospital

    ACTIVE_NOT_RECRUITING

    Kanazawa, 920 8641, Japan

  • Karmanos Cancer Institute

    COMPLETED

    Detroit, Michigan, 48201 2013, United States

  • Kobe City Medical Center General Hospital

    ACTIVE_NOT_RECRUITING

    Kobe, 650 0047, Japan

  • Kyushu University Hospital

    ACTIVE_NOT_RECRUITING

    Fukuoka, 812-0054, Japan

  • Leeds Teaching Hospitals NHS Trust

    RECRUITING

    Leeds, LS9 7TF, United Kingdom

  • Liga Norte Riograndense Contra O Cancer

    RECRUITING

    Natal, 59062 000, Brazil

  • MD Anderson

    RECRUITING

    Houston, Texas, 77030, United States

  • Maine Health

    RECRUITING

    Providence, Rhode Island, 02903, United States

  • Massachusetts General Hospital

    RECRUITING

    Boston, Massachusetts, 02114, United States

  • Medical College of WI at Froedtert

    RECRUITING

    Milwaukee, Wisconsin, 53226, United States

  • Ministerio da Saude Instituto Nacional do Cancer

    RECRUITING

    Rio de Janeiro, 20230 130, Brazil

  • Monash Medical Centre

    RECRUITING

    Clayton, 3168, Australia

  • Montefiore Medical Center

    RECRUITING

    The Bronx, New York, 10467, United States

  • NTT Medical Center Tokyo

    ACTIVE_NOT_RECRUITING

    Tokyo, 141-8625, Japan

  • NYU Langone Medical Center

    RECRUITING

    New York, New York, 10016, United States

  • Nagoya University Hospital

    COMPLETED

    Nagoya, 466-8560, Japan

  • Nanfang Hospital of Southern Medical Hospital

    RECRUITING

    Guangzhou, 510515, China

  • National Cancer Center Hospital East

    ACTIVE_NOT_RECRUITING

    Kashiwa, 277-8577, Japan

  • National Cheng Kung University Hospital

    RECRUITING

    Tainan, 704, Taiwan

  • National Taiwan University Hospital

    RECRUITING

    Taipei, 10043, Taiwan

  • Norton Cancer Institute

    COMPLETED

    Louisville, Kentucky, 40207, United States

  • Oregon Health And Science University

    RECRUITING

    Portland, Oregon, 97239, United States

  • Oxford University Hospitals NHS Trust

    RECRUITING

    Oxfordshire, OX3 7LE, United Kingdom

  • Peking University First Hospital

    RECRUITING

    Beijing, 100034, China

  • Princess Margaret Cancer Centre University Health Network

    RECRUITING

    Toronto, Ontario, M5G 1X6, Canada

  • Qilu Hospital of Shandong University

    RECRUITING

    Jinan, 250012, China

  • Roswell Park Comprehensive Cancer Center

    RECRUITING

    Buffalo, New York, 14263, United States

  • Royal Perth Hospital

    RECRUITING

    Perth, 6000, Australia

  • Samsung Medical Center

    RECRUITING

    Seoul, 06351, South Korea

  • San Antonio Methodist TX Transplant Physicians Group

    RECRUITING

    San Antonio, Texas, 78229, United States

  • Seoul National University Hospital

    RECRUITING

    Seoul, 03080, South Korea

  • Sheba Medical Center

    RECRUITING

    Ramat Gan, 52621, Israel

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

    RECRUITING

    Indianapolis, Indiana, 46237, United States

  • Start Midwest

    RECRUITING

    Grand Rapids, Michigan, 49546, United States

  • Swedish Cancer Institute

    RECRUITING

    Seattle, Washington, 98104, United States

  • Tel Aviv Sourasky Medical Center

    RECRUITING

    Tel Aviv, 6423906, Israel

  • The Christie Nhs Foundation Trust

    RECRUITING

    Manchester, M20 4BX, United Kingdom

  • The Clatterbridge Cancer Centre

    RECRUITING

    Liverpool, L7 8YA, United Kingdom

  • The First Affiliated Hospital of NanChang University

    RECRUITING

    Nanchang, 330006, China

  • The First Affiliated Hospital of Wenzhou Medical University

    RECRUITING

    Wenzhou, 325000, China

  • The First Hospital of Jilin University

    RECRUITING

    Changchun, 130021, China

  • UCSF Benioff Children's Hospital

    RECRUITING

    San Francisco, California, 94158, United States

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

    RECRUITING

    Wuhan, 430022, China

  • University College London Hospitals

    RECRUITING

    London, W1T 7HA, United Kingdom

  • University Hospital of Wales

    RECRUITING

    Cardiff, CF14 4XW, United Kingdom

  • University Hospitals Plymouth NHS Trust

    RECRUITING

    Plymouth, PL6 8DH, United Kingdom

  • University of California Irvine Medical Center

    COMPLETED

    Orange, California, 92868, United States

  • University of California San Francisco

    RECRUITING

    San Francisco, California, 94143, United States

  • University of Chicago

    RECRUITING

    Chicago, Illinois, 60637, United States

  • University of Fukui Hospital

    COMPLETED

    Yoshida, 910-1193, Japan

  • University of New Mexico

    RECRUITING

    Albuquerque, New Mexico, 87131, United States

  • University of Tennessee Medical Center

    RECRUITING

    Knoxville, Tennessee, 37920, United States

  • Vancouver Coastal Health

    RECRUITING

    Vancouver, British Columbia, V5Z 1M9, Canada

  • Virginia Commonwealth University

    RECRUITING

    Richmond, Virginia, 23298, United States

  • West China Hospital Si Chuan University

    RECRUITING

    Chengdu, 610041, China

  • Yamagata University Hospital

    COMPLETED

    Yamagata, 990 2331, Japan

  • Zhongda Hospital Southeast University

    RECRUITING

    Nanjing, 210009, China

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