Den här översättningen är inte klar ännu. Den här sidan är just nu på engelska.

Gå till den engelska sidan

Cure My Disease

Don't miss your cure!

 Log in / Sign up

New hope for tough leukemia: experimental drug targets genetic flaw

NCT ID NCT04067336

Disease control Sponsor: Kura Oncology, Inc. Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
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 This study
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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 27, 2026 · Last updated Jun 27, 2026

Summary

This study tests a new drug called ziftomenib (KO-539) in people with acute myeloid leukemia (AML) that has come back or not responded to treatment. The drug targets a specific genetic change (MLL rearrangement or NPM1 mutation) found in some patients. The goal is to find the safest dose and see if it can control the disease.

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 263 people

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

Started

Sep 2019

Expected to finish

Oct 2028

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

18 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.

Key Inclusion Criteria: Patients with refractory or relapsed AML defined as the reappearance of ≥ 5% blasts in the bone marrow and who have also failed or are ineligible for any approved standard of care therapies, including HSCT. 1. Phase 1b: * Patients with a documented lysine\[K\]-specific methyltransferase 2-rearrangement (KMT2A-r), or * Patients with a documented nucleophosmin 1 mutation (NPM1-m) 2. Phase 2: * Patients with a documented nucleophosmin 1 mutation (NPM1-m) 3. Sub-studies: * Sub-studies 1 and 2: Patients with R/R AML with NPM1-m or other mutations associated with MEIS1 overexpression. * Sub-study 3: Patients with R/R Acute Lymphoblastic Leukemia (ALL) with KMT2A-r. * Sub-study 4: Patients with R/R AML with mutations associated with MEIS1 overexpression. 4. ≥ 18 years of age. 5. Eastern Cooperative Oncology Group (ECOG) performance status of 0 to 2, and a life expectancy of at least 2 months. 6. Adequate liver and kidney function according to protocol requirements. 7. Peripheral white blood cell (WBC) counts ≤ 30,000/μL. Patients may receive hydroxyurea to control and maintain white blood cell count prior to enrollment. 8. Women of childbearing potential must be willing to use a highly effective method of contraception throughout the study and for at least 187 days after the last dose of study treatment. 9. Males with female partners of childbearing potential must agree to use a highly effective method of contraception throughout the study and for at least 97 days after the last dose of study treatment. Key Exclusion Criteria: 1. Diagnosis of acute promyelocytic leukemia. 2. Diagnosis of chronic myelogenous leukemia in blast crisis. 3. Donor lymphocyte infusion \< 30 days prior to study entry. 4. Clinically active central nervous system (CNS) leukemia. 5. Undergone HSCT and have not had adequate hematologic recovery. 6. Receiving immunosuppressive therapy post HSCT within 2 weeks of Cycle 1 Day 1. 7. Grade ≥ 2 active graft-versus-host disease (GVHD), moderate or severe limited chronic GVHD, or extensive chronic GVHD of any severity. 8. Received chemotherapy immunotherapy, radiotherapy, or any ancillary therapy that is considered to be investigational (i.e., used for non-approved indications(s) and in the context of a research investigation) \< 14 days prior to the first dose of ziftomenib or within 5 drug half-lives prior to the first dose of study drug. 9. Not recovered to \< Grade 2 (National Cancer Institute Common Terminology Criteria for Adverse Events v5.0) from all acute toxicities or deemed back to a stable baseline. 10. Treatment with concomitant drugs that are strong inhibitors or inducers of cytochrome P450-isozyme 3A4 (CYP3A4), as follows: * Phase 1a, 1b, 2, and sub-studies 3 and 4: with the exception of antibiotics, antifungals, and antivirals that are used as standard of care or to prevent or treat infections and other such drugs that are considered absolutely essential for the care of the patient. * Sub-studies 1 and 2: No exceptions will be allowed except for the use of moderate CYP3A4 antifungal prophylaxis such as fluconazole or isavuconazole which is at steady state on Cycle 1 Day 1 and will continue through the completion of PKs on Cycle 1 Day 15 (for sub-study 1) or Cycle 1 Day 18 (for sub-study 2). 11. Detectable viral load for human immunodeficiency virus, hepatitis C, or hepatitis B surface antigen indicative of active infection. Patients with controlled disease will not be excluded from study enrollment. 12. Pre-existing disorder predisposing the patient to a serious or life-threatening infection (e.g. cystic fibrosis, congenital or acquired immunodeficiency, bleeding disorder, or cytopenias not related to AML). 13. Active uncontrolled acute or chronic systemic fungal, bacterial, viral, or other infection. 14. Significant cardiovascular disease including unstable angina pectoris, uncontrolled hypertension or arrhythmia, history of cerebrovascular accident including transient ischemic attack within the past 6 months, congestive heart failure (NYHA Class III or IV) related to primary cardiac disease, ischemic or severe valvular heart disease, or a myocardial infarction within 6 months prior to the first dose of study treatment. 15. Mean QTcF \>480 ms on triplicate ECG. 16. Major surgery within 4 weeks prior to the first dose of study treatment. 17. Women who are pregnant or lactating. All female patients with reproductive potential must have a negative serum pregnancy test within 72 hours prior to starting treatment. 18. For sub-studies 1 and 2: Any intake of grapefruit, grapefruit juice, Seville oranges, Seville orange marmalade, or other products containing grapefruit or Seville oranges within 7 days of the first administration of ziftomenib until the end of Cycle 1. 19. For sub-studies 1 and 2: Moderate (Child-Pugh class B) or severe (Child-Pugh class C) hepatic impairment.

Get updates

Get notified about this study

Sign up to get updates when this study changes or when new studies for Acute leukemia of ambiguous lineage are added.

Vår säkerhetsrekommendation!

Genom att skicka in godkänner du våra Användarvillkor

Conditions

The condition(s) this trial relates to.

acute biphenotypic leukemia acute leukemia of ambiguous lineage acute lymphoblastic leukemia acute myeloid leukemia acute myeloid leukemia, MLL gene rearrangement cancer hematopoietic and lymphoid system neoplasm leukemia mixed phenotype acute leukemia Precursor Cell Lymphoblastic Leukemia-Lymphoma

As listed by the trial registrant

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

Acute leukemia of ambiguous lineage Acute lymphoblastic leukemia Acute myeloid leukemia Advanced malignant neoplasm Mixed lineage acute leukemia Mixed lineage leukemia Mixed phenotype acute leukemia

Contacts and locations

Locations

  • AZ Delta - Campus Rumbeke

    Roeselare, 8800, Belgium

  • Banner MD Anderson Cancer Center

    Gilbert, Arizona, 85234, United States

  • Centre Hospitalier Lyon Sud

    Pierre-Bénite, 69310, France

  • Centre Hospitalier Universitaire de Lille

    Lille, 59037, France

  • Centre Hospitalier Universitaire de Nantes

    Nantes, 44093, France

  • Duke Cancer Institute

    Durham, North Carolina, 27710, United States

  • Fred Hutchinson Cancer Research Center

    Seattle, Washington, 98109, United States

  • Hackensack University Medical Center - John Theurer Cancer Center

    Hackensack, New Jersey, 07601, United States

  • Harold C. Simmons Comprehensive Cancer Center - UT Southwestern Medical Center

    Dallas, Texas, 75390, United States

  • Hopital Maisonneuve-Rosemont

    Montreal, Quebec, H1T 2M4, Canada

  • Hopital Saint Louis

    Paris, 75475, France

  • Hopital de l'Enfant-Jesus - Centre Integre en Cancerologie du CHU de Quebec - Universite Laval

    Québec, Quebec, G1J 1Z4, Canada

  • Hospital Universitari Vall d'Hebron

    Barcelona, 08035, Spain

  • Hospital Universitari i Politecnic La Fe

    Valencia, 46026, Spain

  • Hospital Universitario Central de Asturias

    Oviedo, 33011, Spain

  • Hospital Universitario HM Sanchinarro

    Madrid, 28050, Spain

  • Hospital Universitario Virgen del Rocio

    Seville, 41013, Spain

  • IRCCS Istituto Romagnolo per lo Studio dei Tumori "Dino Amadori"

    Meldola, 47014, Italy

  • Institut Gustave Roussy

    Villejuif, 94800, France

  • Institute of Hematology and Medical Oncology "L. and A. Seragnoli"

    Bologna, 40138, Italy

  • Institution Fondazione Policlinico Tor Vergata

    Roma, Italy

  • Karmanos Cancer Institute

    Detroit, Michigan, 48201, United States

  • MD Anderson Cancer Center

    Houston, Texas, 77030, United States

  • MD Anderson Cancer Center

    Madrid, 28033, Spain

  • Magendie Hopital Haut-Leveque

    Pessac, 33600, France

  • Massachusetts General Hospital

    Boston, Massachusetts, 02114, United States

  • Mayo Clinic

    Jacksonville, Florida, 32224, United States

  • Mayo Clinic

    Rochester, Minnesota, 55905, United States

  • Medizinische Hochsschule Hannover

    Hanover, 30625, Germany

  • Oklahoma University Health - Stephenson Cancer Center

    Oklahoma City, Oklahoma, 73117, United States

  • Queen Elizabeth II Health Sciences Centre

    Halifax, Nova Scotia, B3H 1V7, Canada

  • Robert H. Lurie Comprehensive Cancer Center of Northwestern University

    Chicago, Illinois, 60611, United States

  • Roswell Park Comprehensive Cancer Center

    Buffalo, New York, 14203, United States

  • The Mount Sinai Hospital

    New York, New York, 10029, United States

  • UCLA Ronald Reagan Medical Center

    Los Angeles, California, 90095, United States

  • UO Ematologia Ospedale di Ravenna

    Ravenna, 48121, Italy

  • UPMC Hillman Cancer Center

    Pittsburgh, Pennsylvania, 15232, United States

  • Universitat de Barcelona

    Barcelona, 08035, Spain

  • University Medicine Greifswald

    Greifswald, 17475, Germany

  • University of Maryland Greenebaum Comprehensive Cancer Center

    Baltimore, Maryland, 21201, United States

  • University of Michigan Hospitals

    Ann Arbor, Michigan, 48109, United States

  • Vanderbilt-Ingram Cancer Center

    Nashville, Tennessee, 37232, United States

  • Weill Cornell Medical College - NY Presbyterian Hospital

    New York, New York, 10021, United States

More trials for these conditions

Other studies related to the condition(s) this trial covers.