New hope for AML patients: targeted drug ziftomenib enters final testing phase

NCT ID NCT07007312

First seen Jun 27, 2026 · Last updated Jun 30, 2026 · Updated 2 times

Summary

This large Phase 3 trial is testing whether adding the experimental drug ziftomenib to standard chemotherapy helps people with untreated acute myeloid leukemia (AML) who have specific genetic mutations. About 1,300 participants will be randomly assigned to receive either ziftomenib or a placebo alongside their regular treatment. The study is double-blind, meaning neither patients nor doctors know who gets the real drug.

What this could mean

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

Active substance

ziftomenib

What this could lead to

If successful, this could lead to a new targeted treatment option for people with certain genetic types of AML, potentially improving survival and remission rates.

What could go wrong

This is a large Phase 3 trial, but the drug is still investigational. It may not prove more effective than standard therapy, and side effects from combining drugs are possible.

Disclaimer Read more

This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.

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Conditions

The condition(s) this trial relates to.

acute myeloid leukemia acute myeloid leukemia with mutated NPM1 acute myeloid leukemia, MLL gene rearrangement hematopoietic and lymphoid system neoplasm leukemia

As listed by the trial registrant

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

Contacts and locations

Study contacts

  • Contact

    Phone: •••-•••-•••• Email: •••••@•••••

Locations

  • Anam Hospital Korea University

    RECRUITING

    Seoul, Seoul-T'Ukpyolshi, 02841, South Korea

  • Attikon University General Hospital of Athens

    RECRUITING

    Chaïdári, Attica, 12462, Greece

  • Azienda Ospedaliero-Universitaria

    RECRUITING

    Perugia, Umbria, 42270, Italy

  • Azienda Sanitaria Territoriale di Ascoli Piceno

    RECRUITING

    Ascoli Piceno, The Marches, 63100, Italy

  • Baguio General Hospital and Medical Center

    RECRUITING

    Baguio City, Benguet, 02600, Philippines

  • Banner MD Anderson Cancer Center

    RECRUITING

    Gilbert, Arizona, 85234, United States

  • Baptist Clinical Research Institute

    RECRUITING

    Memphis, Tennessee, 38120, United States

  • Calvary Mater Newcastle

    RECRUITING

    Waratah, New South Wales, 02298, Australia

  • Cedars-Sinai Medical Center

    RECRUITING

    Los Angeles, California, 90048, United States

  • Centre Hospitalier Régional et Universitaire de Nancy Hôpitaux de Brabois

    RECRUITING

    Vandœuvre-lès-Nancy, Grand Est, 54510, France

  • Centre Hospitalier Universitaire Estaing - Clermont-Ferrand

    RECRUITING

    Clermont-Ferrand, Auvergne-Rhône-Alpes, 63003, France

  • Centre Hospitalier Universitaire de Bordeaux

    RECRUITING

    Pessac, New Aquitaine, 33604, France

  • Centre Hospitalier Universitaire de Nantes

    RECRUITING

    Nantes, Loire-Atlantique, 44000, France

  • Centre Hospitalier Universitaire de Saint Etienne

    RECRUITING

    Saint-Priest-en-Jarez, Auvergne-Rhône-Alpes, 42270, France

  • Centre Hospitalier de Béziers

    RECRUITING

    Béziers, Occitanie, 35400, France

  • Chang Bing Show Chwan Memorial Hospital

    RECRUITING

    Lugang, Central Taiwan, 505, Taiwan

  • Chonnam National University Hwasun Hospital

    RECRUITING

    Hwasun, 58128, South Korea

  • Chungnam National University Daejeon Hospital

    RECRUITING

    Daejeon, 35015, South Korea

  • Colorado Blood Cancer Institute

    RECRUITING

    Denver, Colorado, 80218, United States

  • Columbia University

    RECRUITING

    New York, New York, 10032, United States

  • Dong-A University Hospital

    RECRUITING

    Busan, 49201, South Korea

  • Duke University Medical Center

    RECRUITING

    Durham, North Carolina, 27710, United States

  • Fakultni Nemocnice Ostrava

    RECRUITING

    Ostrava-Poruba, Moravian-Silesian, 70852, Czechia

  • Fakultní Nemocnice Královské Vinohrady

    RECRUITING

    Prague, Prague, 10034, Czechia

  • Froedtert & Medical College of Wisconsin

    RECRUITING

    Milwaukee, Wisconsin, 53226, United States

  • Groupe Hospitalier de la Region de Mulhouse et Sud Alsace Hôpital Emile Muller

    RECRUITING

    Mulhouse, Grand Est, 68100, France

  • Hartford HealthCare Cancer Institute

    RECRUITING

    Hartford, Connecticut, 06106, United States

  • Helios Klinikum Bad Saarow

    RECRUITING

    Bad Saarow, 15526, Germany

  • Hospital Clinico Universitario Lozano Blesa

    RECRUITING

    Zaragoza, Aragon, 50009, Spain

  • Hospital Clinico Universitario de Valencia

    RECRUITING

    Valencia, València, 46010, Spain

  • Hospital General Universitario de Albacete

    RECRUITING

    Albacete, Castille-La Mancha, 02006, Spain

  • Hospital Universitario 12 de Octubre

    RECRUITING

    Madrid, Madrid, 28041, Spain

  • Hospital Universitario De Burgos

    RECRUITING

    Burgos, Castille and León, 09006, Spain

  • Hospital Universitario Infanta Leonor

    RECRUITING

    Madrid, Madrid, 28031, Spain

  • Hospital Universitario Y Politecnico La Fe

    RECRUITING

    Valencia, València, 46026, Spain

  • Hospital de Braga, Centro Clínico Académico de Braga

    RECRUITING

    Braga, 1099-023, Portugal

  • Hôpital Avicenne

    RECRUITING

    Bobigny, Île-de-France Region, 93009, France

  • Hôpital Lyon Sud

    RECRUITING

    Pierre-Bénite, Auvergne-Rhône-Alpes, 69495, France

  • Hôpital Saint Louis

    RECRUITING

    Paris, Île-de-France Region, 75010, France

  • Hôpital Universitaire Henri Mondor

    RECRUITING

    Créteil, Val-de-Marne, 94010, France

  • Icahn School of Medicine at Mount Sinai

    RECRUITING

    New York, New York, 10003, United States

  • L'IRCCS Azienda Ospedaliero - Universitaria di Bologna

    RECRUITING

    Bologna, Emilia-Romagna, 40138, Italy

  • Moffitt Cancer Center & Research Institute

    RECRUITING

    Tampa, Florida, 33612, United States

  • National Taiwan University Hospital

    RECRUITING

    Taipei, Northern Taiwan, 10002, Taiwan

  • Ohio State University

    RECRUITING

    Columbus, Ohio, 43210, United States

  • Pusan National University Hospital

    RECRUITING

    Busan, 49241, South Korea

  • Rutgers Biomedical and Health Sciences

    RECRUITING

    New Brunswick, New Jersey, 08903, United States

  • Samsung Medical Center

    RECRUITING

    Seoul, Seoul-T'Ukpyolshi, 35015, South Korea

  • State University of New York at Buffalo

    RECRUITING

    Buffalo, New York, 14263, United States

  • Taipei Veterans General Hospital

    RECRUITING

    Taipei, Northern Taiwan, 613, Taiwan

  • Tennessee Oncology

    RECRUITING

    Nashville, Tennessee, 37203, United States

  • Texas Oncology - San Antonio Medical Center

    RECRUITING

    San Antonio, Texas, 78240, United States

  • Texas Oncology-Austin Midtown

    RECRUITING

    Austin, Texas, 78705, United States

  • Texas Oncology-Presbyterian Cancer Center

    RECRUITING

    Dallas, Texas, 75231, United States

  • TriStar Centennial Medical Center

    RECRUITING

    Nashville, Tennessee, 37203, United States

  • Ulsan University Hospital

    RECRUITING

    Ulsan, 44033, South Korea

  • University General Hospital of Alexandroupoli

    RECRUITING

    Alexandroupoli, East Macedonia and Thrace, 68100, Greece

  • University Hospital Virgen Del Rocio S.L.

    RECRUITING

    Seville, Andalusia, 41013, Spain

  • University of California, Fresno

    RECRUITING

    Clovis, California, 93611, United States

  • University of California, Irvine

    RECRUITING

    Orange, California, 92868, United States

  • University of California, Los Angeles

    RECRUITING

    Los Angeles, California, 90095, United States

  • University of California, San Diego

    RECRUITING

    La Jolla, California, 92093, United States

  • University of Colorado

    RECRUITING

    Aurora, Colorado, 80045, United States

  • University of Iowa

    RECRUITING

    Iowa City, Iowa, 52246, United States

  • University of Kentucky

    RECRUITING

    Lexington, Kentucky, 40536, United States

  • University of Massachusetts

    RECRUITING

    Worcester, Massachusetts, 01605, United States

  • University of Miami

    RECRUITING

    Miami, Florida, 33136, United States

  • University of Michigan

    RECRUITING

    Ann Arbor, Michigan, 48109, United States

  • University of Minnesota

    RECRUITING

    Minneapolis, Minnesota, 55455, United States

  • University of New Mexico

    RECRUITING

    Albuquerque, New Mexico, 87131, United States

  • University of North Carolina, Chapel Hill

    RECRUITING

    Chapel Hill, North Carolina, 27514, United States

  • University of Pennsylvania

    RECRUITING

    Philadelphia, Pennsylvania, 19104, United States

  • University of Texas

    RECRUITING

    Houston, Texas, 77030, United States

  • University of Vermont Medical Center

    RECRUITING

    Burlington, Vermont, 05401, United States

  • University of Virginia School of Medicine

    RECRUITING

    Charlottesville, Virginia, 22903, United States

  • University of Wisconsin

    RECRUITING

    Madison, Wisconsin, 53792, United States

  • Virginia Cancer Specialists

    RECRUITING

    Manassas, Virginia, 20110, United States

  • WVU Medicine Wheeling Hospital

    RECRUITING

    Wheeling, West Virginia, 26003, United States

  • Wayne State University School of Medicine

    RECRUITING

    Detroit, Michigan, 48201, United States

  • Weill Cornell Medical Center

    RECRUITING

    New York, New York, 10065, United States

  • Willamette Valley Cancer Institute

    RECRUITING

    Eugene, Oregon, 97401, United States

  • Yale University School of Medicine

    RECRUITING

    New Haven, Connecticut, 06510, United States