Studies to Assess Ziftomenib in Combination With Ven+Aza or 7+3 in Patients With Untreated NPM1-m or KMT2A-r AML

August 19, 2026 updated by: Kura Oncology, Inc.

Phase 3 Randomized, Double-blind, Placebo-controlled Studies Assessing Ziftomenib in Combination With Either Standard of Care Nonintensive (Venetoclax+Azacitidine) or Intensive (7+3) Therapy in Patients With Untreated NPM1 Mutated or KMT2A Rearranged Acute Myeloid Leukemia

Ziftomenib is an investigational drug in development for the treatment of patients with acute myeloid leukemia (AML) with eligible genetic alterations. Ziftomenib is a type of therapy known to target the menin pathway in cancer cells.

This protocol has 2 separate studies that will investigate the benefits and risks of adding ziftomenib to standard-of-care (SOC) AML treatments in patients with certain genetic mutations who have not received any treatment for their AML. In the first study, the Nonintensive Therapy Study, older patients or those with serious medical problems will receive the SOC therapies venetoclax (ven) and azacitidine (aza), plus either ziftomenib or a placebo. In the second study, the Intensive Therapy Study, medically fit patients will receive (a) the SOC therapies cytarabine and daunorubicin, plus either ziftomenib or a placebo during a first treatment phase called induction, (b) cytarabine plus either ziftomenib or a placebo during a second treatment phase called consolidation, and (c) ziftomenib or a placebo during a third treatment phase called maintenance.

The physician will determine which study is the appropriate treatment for the patient, but neither the patient nor their physician will know whether the patient has been assigned to receive ziftomenib or a placebo. This design is called "double-blinded".

Study Overview

Status

Recruiting

Conditions

Intervention / Treatment

Detailed Description

This protocol encompasses two phase 3, randomized, double-blind, placebo-controlled clinical studies to assess the efficacy, safety, and tolerability of ziftomenib in combination with: (a) the standard of care (SOC) nonintensive regimen (venetoclax [ven]+azacitidine [aza]) in untreated adults with nucleophosmin 1 mutated (NPM1-m) acute myeloid leukemia (AML); or (b) the SOC intensive regimen (cytarabine+daunorubicin induction, referred to here as 7+3, and cytarabine consolidation) in untreated adults with NPM1-m or lysine[K]-specific methyltransferase 2A rearranged (KMT2A-r) AML, as well as a maintenance phase.

Nonintensive Therapy Study (Ven+Aza)

Eligible NPM1-m patients will be enrolled and randomized to receive:

  • Arm A: Ziftomenib in combination with ven+aza or
  • Arm B: Placebo in combination with ven+aza.

Patients will be randomized to treatment arms in a double-blind manner.

Intensive Therapy Study (Cytarabine+Daunorubicin)

Eligible NPM1-m or KMT2A-r patients will be enrolled and randomized to 1 of the following treatment arms:

  • Arm A: Ziftomenib+7+3 (induction), ziftomenib+cytarabine (consolidation), ziftomenib (maintenance) or
  • Arm B: Ziftomenib+7+3 (induction), ziftomenib+cytarabine (consolidation), placebo (maintenance) or
  • Arm C: Placebo+7+3 (induction), placebo+cytarabine (consolidation), placebo (maintenance).

Patients will be randomized to treatment arms in a double-blind manner.

Study Type

Interventional

Enrollment (Estimated)

1300

Phase

  • Phase 3

Expanded Access

Available outside the clinical trial. See expanded access record.

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Locations

  • Australia
    • New South Wales
      • Waratah, New South Wales, Australia, 02298
        • Recruiting
        • Calvary Mater Newcastle
    • Victoria
      • Heidelberg, Victoria, Australia, 3128
        • Recruiting
        • Box Hill Hospital
    • Western Australia
      • Murdoch, Western Australia, Australia, 6150
        • Recruiting
        • Fiona Stanley Hospital
  • Belgium
    • Brussels Capital
      • Jette, Brussels Capital, Belgium, 1090
        • Recruiting
        • Universitair Ziekenhuis Brussel
    • Oost-Vlaanderen
      • Ghent, Oost-Vlaanderen, Belgium, 9000
        • Recruiting
        • Universitair Ziekenhuis Gent
    • Walloonia
      • Verviers, Walloonia, Belgium, 4800
        • Recruiting
        • Centre Hospitalier Régional Verviers East Belgium
    • West-Vlaanderen
      • Bruges, West-Vlaanderen, Belgium, 8800
        • Recruiting
        • Algemeen Ziekenhuis Sint-Jan Brugge
      • Roeselare, West-Vlaanderen, Belgium, 8800
        • Recruiting
        • Algemeen Ziekenhuis Delta VZW
  • Canada
    • Quebec
      • Montreal, Quebec, Canada, H4A3J1
        • Recruiting
        • Royal Victoria Hospital
  • Czechia
    • Moravian-Silesian
      • Ostrava-Poruba, Moravian-Silesian, Czechia, 70852
        • Recruiting
        • Fakultni nemocnice Ostrava
    • Prague
      • Prague, Prague, Czechia, 10034
        • Recruiting
        • Fakultni nemocnice Kralovske Vinohrady
  • France
    • Auvergne-Rhône-Alpes
      • Clermont-Ferrand, Auvergne-Rhône-Alpes, France, 63003
        • Recruiting
        • Centre Hospitalier Universitaire Estaing - Clermont-Ferrand
      • Pierre-Bénite, Auvergne-Rhône-Alpes, France, 69495
        • Recruiting
        • Hopital Lyon Sud
      • Saint-Priest-en-Jarez, Auvergne-Rhône-Alpes, France, 42270
        • Recruiting
        • Centre Hospitalier Universitaire de Saint Etienne
    • Grand Est
      • Mulhouse, Grand Est, France, 68100
        • Recruiting
        • Groupe Hospitalier de la Region de Mulhouse et Sud Alsace Hôpital Emile Muller
      • Vandœuvre-lès-Nancy, Grand Est, France, 54510
        • Recruiting
        • Centre Hospitalier Régional et Universitaire de Nancy Hôpitaux de Brabois
    • Loire-Atlantique
      • Nantes, Loire-Atlantique, France, 44000
        • Recruiting
        • Centre Hospitalier Universitaire De Nantes
    • New Aquitaine
      • Pessac, New Aquitaine, France, 33604
        • Recruiting
        • Centre hospitalier universitaire de Bordeaux
    • Nord
      • Lille, Nord, France, 59000
        • Recruiting
        • Centre Hospitalier de Roubaix
    • Normandy
      • Rouen, Normandy, France, 76038
        • Recruiting
        • Centre Henri Becquerel
    • Occitanie
      • Béziers, Occitanie, France, 35400
        • Recruiting
        • Centre Hospitalier de Beziers
    • Provence-Alpes-Côte d'Azur Region
      • Marseille, Provence-Alpes-Côte d'Azur Region, France, 13385
        • Recruiting
        • Centre Hospitalier Universitaire de Marseille - Hôpital de la Conception
    • Val-De-Marne
      • Villejuif, Val-De-Marne, France, 94805
        • Recruiting
        • Institut Gustave Roussy
    • Val-de-Marne
      • Créteil, Val-de-Marne, France, 94010
        • Recruiting
        • Hôpital Universitaire Henri Mondor
    • Île-de-France Region
      • Bobigny, Île-de-France Region, France, 93009
        • Recruiting
        • Hôpital Avicenne
      • Le Chesnay, Île-de-France Region, France, 78150
        • Recruiting
        • Centre Hospitalier De Versailles
      • Paris, Île-de-France Region, France, 75010
        • Recruiting
        • Hopital Saint Louis
      • Paris, Île-de-France Region, France, 75013
        • Recruiting
        • Hôpital Pitié-Salpêtrière
  • Germany
    • Brandenburg
      • Bad Saarow, Brandenburg, Germany, 15526
        • Recruiting
        • Helios Klinikum Bad Saarow
    • North Rhine-Westphalia
      • Aachen, North Rhine-Westphalia, Germany, 52074
        • Recruiting
        • Universitätsklinikum Aachen
  • Greece
    • Attica
      • Athens, Attica, Greece, 11527
        • Recruiting
        • Laiko General Hospital of Athens
      • Chaïdári, Attica, Greece, 12462
        • Recruiting
        • Attikon University General Hospital of Athens
    • Central Macedonia
      • Exochi, Central Macedonia, Greece, 57010
        • Recruiting
        • General Hospital of Thessaloniki G. Papanikolaou
    • East Macedonia and Thrace
      • Alexandroupoli, East Macedonia and Thrace, Greece, 68100
        • Recruiting
        • University General Hospital of Alexandroupoli
    • Epirus
      • Ioannina, Epirus, Greece, 45500
        • Recruiting
        • University General Hospital of Ioannina
  • Italy
    • Emilia-Romagna
      • Bologna, Emilia-Romagna, Italy, 40138
        • Recruiting
        • L'IRCCS Azienda Ospedaliero - Universitaria di Bologna
      • Ravenna, Emilia-Romagna, Italy, 48121
        • Recruiting
        • Hospital Santa Maria delle Croci
    • Friuli Venezia Giulia
      • Trieste, Friuli Venezia Giulia, Italy, 76038
        • Recruiting
        • ASUGI Ospedale Maggiore di Trieste
    • Lombardy
      • Brescia, Lombardy, Italy, 25123
        • Recruiting
        • ASST Spedali Civili di Brescia
    • The Marches
      • Ascoli Piceno, The Marches, Italy, 63100
        • Recruiting
        • Azienda Sanitaria Territoriale di Ascoli Piceno
    • Umbria
      • Perugia, Umbria, Italy, 42270
        • Recruiting
        • Azienda Ospedaliero-Universitaria
  • Philippines
    • Benguet
      • Baguio City, Benguet, Philippines, 02600
        • Recruiting
        • Baguio General Hospital and Medical Center
    • National Capital Region
      • Manila, National Capital Region, Philippines, 1015
        • Recruiting
        • University of Santo Tomas
      • Pasig, National Capital Region, Philippines, 1605
        • Recruiting
        • The Medical City
      • Quezon City, National Capital Region, Philippines, 1112
        • Recruiting
        • St. Luke's Medical Center - Quezon City
  • Poland
    • Opole Voivodeship
      • Opole, Opole Voivodeship, Poland, 45-061
        • Recruiting
        • Szpital Wojewodzki w Opolu
  • Portugal
      • Braga, Portugal, 1099-023
        • Recruiting
        • Hospital de Braga, Centro Clínico Académico de Braga
  • South Korea
    • Daejeon Gwang'Yeogsi
      • Daejeon, Daejeon Gwang'Yeogsi, South Korea, 35015
        • Recruiting
        • Chungnam National University Daejeon Hospital
    • Gyeongsangbuk-do
      • Daegu, Gyeongsangbuk-do, South Korea, 42472
        • Recruiting
        • Daegu Catholic University Hospital
    • Gyeongsangnam-do
      • Busan-si, Gyeongsangnam-do, South Korea, 49241
        • Recruiting
        • Pusan National University Hospital
      • Pusan, Gyeongsangnam-do, South Korea, 49201
        • Recruiting
        • Dong-A University Hospital
      • Ulsan, Gyeongsangnam-do, South Korea, 44033
        • Recruiting
        • Ulsan University Hospital
    • Incheon Gwang'Yeogsi
      • Namdong, Incheon Gwang'Yeogsi, South Korea, 21565
        • Recruiting
        • Gachon University Gil Medical Center
    • Jeollanam-do
      • Hwasun, Jeollanam-do, South Korea, 58128
        • Recruiting
        • Chonnam National University Hwasun Hospital
    • Seoul-T'Ukpyolsi
      • Seoul, Seoul-T'Ukpyolsi, South Korea, 02841
        • Recruiting
        • Anam Hospital Korea University
      • Seoul, Seoul-T'Ukpyolsi, South Korea, 35015
        • Recruiting
        • Samsung Medical Center
      • Seoul, Seoul-T'Ukpyolsi, South Korea, 06591
        • Recruiting
        • The Catholic University of Korea Seoul St. Mary'S Hospital
  • Spain
    • Andalusia
      • Seville, Andalusia, Spain, 41013
        • Recruiting
        • University Hospital Virgen Del Rocio S.L.
    • Aragon
      • Zaragoza, Aragon, Spain, 50009
        • Recruiting
        • Hospital Clinico Universitario Lozano Blesa
    • Castille and León
      • Burgos, Castille and León, Spain, 09006
        • Recruiting
        • Hospital Universitario de Burgos
    • Castille-La Mancha
      • Albacete, Castille-La Mancha, Spain, 02006
        • Recruiting
        • Hospital General Universitario de Albacete
    • Madrid
      • Madrid, Madrid, Spain, 28041
        • Recruiting
        • Hospital Universitario 12 de Octubre
      • Madrid, Madrid, Spain, 28031
        • Recruiting
        • Hospital Universitario Infanta Leonor
      • Madrid, Madrid, Spain, 28040
        • Recruiting
        • Hospital Universitario Fundacion Jimenez Diaz
    • Principality of Asturias
      • Oviedo, Principality of Asturias, Spain, 33011
        • Recruiting
        • Hospital Universitario Central de Asturias
    • València
      • Valencia, València, Spain, 46026
        • Recruiting
        • Hospital Universitario Y Politecnico La Fe
      • Valencia, València, Spain, 46010
        • Recruiting
        • Hospital Clinico Universitario de Valencia
  • Taiwan
    • Central Taiwan
      • Lugang, Central Taiwan, Taiwan, 505
        • Recruiting
        • Chang Bing Show Chwan Memorial Hospital
      • Taichung, Central Taiwan, Taiwan, 407
        • Recruiting
        • Taichung Veterans General Hospital
    • Northern Taiwan
      • Taipei, Northern Taiwan, Taiwan, 10002
        • Recruiting
        • National Taiwan University Hospital
      • Taipei, Northern Taiwan, Taiwan, 613
        • Recruiting
        • Taipei Veterans General Hospital
  • United Kingdom
    • England
      • London, England, United Kingdom, EC1A 7BE
        • Not yet recruiting
        • St. Bartholomew's Hospital
      • London, England, United Kingdom, SW17 0QT
        • Not yet recruiting
        • St. George's, University of London
  • United States
    • Arizona
      • Gilbert, Arizona, United States, 85234
        • Recruiting
        • Banner MD Anderson Cancer Center
    • California
      • Clovis, California, United States, 93611
        • Recruiting
        • University of California, Fresno
      • La Jolla, California, United States, 92093
        • Recruiting
        • University of California, San Diego
      • Los Angeles, California, United States, 90095
        • Recruiting
        • University of California, Los Angeles
      • Los Angeles, California, United States, 90048
        • Recruiting
        • Cedars-Sinai Medical Center
      • Orange, California, United States, 92868
        • Recruiting
        • University of California, Irvine
    • Colorado
      • Aurora, Colorado, United States, 80045
        • Recruiting
        • University Of Colorado
      • Denver, Colorado, United States, 80218
        • Recruiting
        • Colorado Blood Cancer Institute
    • Connecticut
      • Hartford, Connecticut, United States, 06106
        • Recruiting
        • Hartford HealthCare Cancer Institute
      • New Haven, Connecticut, United States, 06510
        • Recruiting
        • Yale University School of Medicine
    • Florida
      • Miami, Florida, United States, 33136
        • Recruiting
        • University of Miami
      • Tampa, Florida, United States, 33612
        • Recruiting
        • Moffitt Cancer Center & Research Institute
    • Iowa
      • Iowa City, Iowa, United States, 52246
        • Recruiting
        • University of Iowa
    • Kentucky
      • Lexington, Kentucky, United States, 40536
        • Recruiting
        • University Of Kentucky
    • Massachusetts
      • Worcester, Massachusetts, United States, 01605
        • Recruiting
        • University of Massachusetts
    • Michigan
      • Ann Arbor, Michigan, United States, 48109
        • Recruiting
        • University of Michigan
      • Detroit, Michigan, United States, 48201
        • Recruiting
        • Wayne State University School of Medicine
    • Minnesota
      • Minneapolis, Minnesota, United States, 55455
        • Recruiting
        • University of Minnesota
    • New Jersey
      • New Brunswick, New Jersey, United States, 08903
        • Recruiting
        • Rutgers Biomedical and Health Sciences
    • New Mexico
      • Albuquerque, New Mexico, United States, 87131
        • Recruiting
        • University of New Mexico
    • New York
      • Buffalo, New York, United States, 14263
        • Recruiting
        • State University of New York at Buffalo
      • New York, New York, United States, 10032
        • Recruiting
        • Columbia University
      • New York, New York, United States, 10065
        • Recruiting
        • Weill Cornell Medical Center
      • New York, New York, United States, 10003
        • Recruiting
        • Icahn School Of Medicine At Mount Sinai
    • North Carolina
      • Chapel Hill, North Carolina, United States, 27514
        • Recruiting
        • University of North Carolina, Chapel Hill
      • Durham, North Carolina, United States, 27710
        • Recruiting
        • Duke University Medical Center
    • Ohio
      • Columbus, Ohio, United States, 43210
        • Recruiting
        • Ohio State University
    • Oregon
      • Eugene, Oregon, United States, 97401
        • Recruiting
        • Willamette Valley Cancer Institute
    • Pennsylvania
      • Philadelphia, Pennsylvania, United States, 19104
        • Recruiting
        • University of Pennsylvania
    • Tennessee
      • Memphis, Tennessee, United States, 38120
        • Recruiting
        • Baptist Clinical Research Institute
      • Nashville, Tennessee, United States, 37203
        • Recruiting
        • Tennessee Oncology
      • Nashville, Tennessee, United States, 37203
        • Recruiting
        • TriStar Centennial Medical center
    • Texas
      • Austin, Texas, United States, 78705
        • Recruiting
        • Texas Oncology-Austin Midtown
      • Dallas, Texas, United States, 75231
        • Recruiting
        • Texas Oncology-Presbyterian Cancer Center
      • Houston, Texas, United States, 77030
        • Recruiting
        • University of Texas
      • San Antonio, Texas, United States, 78240
        • Recruiting
        • Texas Oncology - San Antonio Medical Center
    • Vermont
      • Burlington, Vermont, United States, 05401
        • Recruiting
        • University of Vermont Medical Center
    • Virginia
      • Charlottesville, Virginia, United States, 22903
        • Recruiting
        • University of Virginia School of Medicine
      • Manassas, Virginia, United States, 20110
        • Recruiting
        • Virginia Cancer Specialists
    • West Virginia
      • Wheeling, West Virginia, United States, 26003
        • Recruiting
        • WVU Medicine Wheeling Hospital
    • Wisconsin
      • Madison, Wisconsin, United States, 53792
        • Recruiting
        • University Of Wisconsin
      • Milwaukee, Wisconsin, United States, 53226
        • Recruiting
        • Froedtert & Medical College of Wisconsin

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Description

Key Inclusion Criteria:

The following criteria apply to both the Nonintensive Therapy Study and the Intensive Therapy Study unless otherwise noted:

  • Age ≥18 years at time of signing the informed consent form.
  • Diagnosis of AML per the 2022 WHO Classification of Hematolymphoid Tumors (5th Edition).
  • Eastern Cooperative Oncology Group (ECOG) performance status of 0, 1, or 2.
  • Adequate liver and kidney function according to protocol requirements.
  • A female of childbearing potential must agree to use adequate contraception from the time of screening through 180 days following the last dose of study intervention. A male with a female partner of childbearing potential must agree to use abstinence or adequate contraception from the time of screening through 90 days following the last dose of study intervention.

  • NONINTENSIVE THERAPY STUDY ONLY (VEN+AZA):

    1. Documented NPM1-m.

    2. Patients considered ineligible for Intensive Therapy defined by the following:

      • i. Age ≥75, OR
      • ii. Age <75 with an ECOG performance status of 2 or cardiac, renal, or hepatic impairment per protocol criteria.

  • INTENSIVE THERAPY STUDY ONLY (7+3):

    1. Documented NPM1-m or KMT2A-r (KMT2A-r patients with a partial tandem duplication are not eligible).
    2. Documented FLT3 wild-type or ITD ratio <0.05 OR ineligible to receive FLT3-targeted therapy (medically ineligible or mutation in which FLT3 inhibition is not SOC). Lack of access to an FLT3 inhibitor is not considered "ineligible" for FLT3-targeted therapy.
    3. Ejection fraction of ≥50%.
    4. Fit for Intensive Therapy per Investigator opinion.

Key Exclusion Criteria:

  • Prior therapy for AML (except hydroxyurea or leukapheresis for WBC control).
  • Diagnosis of acute promyelocytic leukemia (APL), blast phase chronic myeloid leukemia, or isolated myeloid sarcoma.
  • Known history of BCR-ABL mutation.

  • History of other active concurrent malignancies prior to study entry except:

    1. Basal cell skin cancer or localized squamous cell cancer of the skin
    2. Previous malignancy confined and locally resected (or treated with other modalities) with curative intent
    3. Prostate or breast cancer receiving adjuvant hormonal therapy.
  • Active central nervous system (CNS) involvement by AML.
  • Clinical signs/symptoms of leukostasis or white blood cells (WBC) >25×10^9/L prior to start of ziftomenib/placebo. Note: Hydroxyurea and/or leukapheresis are permitted to meet this criterion.
  • Known uncontrolled HIV infection or known active hepatitis B virus, hepatitis C virus infection, or other uncontrolled infection.
  • Uncontrolled intercurrent illness including but not limited to, cardiac illness as defined in the protocol.
  • Women who are pregnant or lactating.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: Quadruple

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Nonintensive Therapy Study, Arm A
Ziftomenib in combination with venetoclax+azacitidine
Drug: Ziftomenib
Oral administration
Other Names:
  • KO-539
Drug: Venetoclax
Oral administration
Other Names:
  • Venclexta
  • Venclyxto
Drug: Azacitidine (AZA)
Intravenous or subcutaneous administration
Other Names:
  • Vidaza
  • Azadine
Placebo Comparator: Nonintensive Therapy Study, Arm B
Placebo in combination with venetoclax+azacitidine
Drug: Placebo
Oral administration
Drug: Venetoclax
Oral administration
Other Names:
  • Venclexta
  • Venclyxto
Drug: Azacitidine (AZA)
Intravenous or subcutaneous administration
Other Names:
  • Vidaza
  • Azadine
Experimental: Intensive Therapy Study, Arm A
Ziftomenib+cytarabine+daunorubicin (induction), ziftomenib+cytarabine (consolidation), ziftomenib (maintenance)
Drug: Ziftomenib
Oral administration
Other Names:
  • KO-539
Drug: Daunorubicin
Intravenous administration
Other Names:
  • Cerubidine
  • daunomycin
Drug: Cytarabine (Ara-C)
Intravenous administration
Other Names:
  • Cytosar-U
  • Tarabine PFS
  • cytosine arabinoside (ara-C)
Experimental: Intensive Therapy Study, Arm B
Ziftomenib+cytarabine+daunorubicin (induction), ziftomenib+cytarabine (consolidation), placebo (maintenance)
Drug: Placebo
Oral administration
Drug: Ziftomenib
Oral administration
Other Names:
  • KO-539
Drug: Daunorubicin
Intravenous administration
Other Names:
  • Cerubidine
  • daunomycin
Drug: Cytarabine (Ara-C)
Intravenous administration
Other Names:
  • Cytosar-U
  • Tarabine PFS
  • cytosine arabinoside (ara-C)
Placebo Comparator: Intensive Therapy Study, Arm C
Placebo+cytarabine+daunorubicin (induction), placebo+cytarabine (consolidation), placebo (maintenance)
Drug: Placebo
Oral administration
Drug: Daunorubicin
Intravenous administration
Other Names:
  • Cerubidine
  • daunomycin
Drug: Cytarabine (Ara-C)
Intravenous administration
Other Names:
  • Cytosar-U
  • Tarabine PFS
  • cytosine arabinoside (ara-C)

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Nonintensive Therapy Study: (Primary Endpoint for all countries): Overall survival (OS)
Time Frame: Defined as the time from randomization to date of death from any cause, assessed up to 36 months after last patient inclusion
OS
Defined as the time from randomization to date of death from any cause, assessed up to 36 months after last patient inclusion
Nonintensive Therapy Study: (Dual Primary Endpoint for US & US reference countries only): Complete remission (CR)
Time Frame: Assessed up to 36 months after last patient inclusion
CR rate per European Leukemia Network (ELN) 2022 criteria per Investigator assessment
Assessed up to 36 months after last patient inclusion
Intensive Therapy Study: (Primary Endpoint for all countries): Event-free survival (EFS)
Time Frame: Defined as the time from randomization to treatment failure, hematologic relapse following CR, or death from any cause, whichever comes first, assessed up to 36 months after last patient inclusion
EFS
Defined as the time from randomization to treatment failure, hematologic relapse following CR, or death from any cause, whichever comes first, assessed up to 36 months after last patient inclusion
Intensive Therapy Study: (Dual Primary Endpoint for US & US reference countries only): Complete remission (CR) with bone marrow (BM) measurable residual disease (MRD) negativity in NPM1-m patients
Time Frame: Assessed up to 36 months after last patient inclusion
CR rate per ELN 2022 criteria per Investigator assessment with central BM MRD negativity
Assessed up to 36 months after last patient inclusion

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Nonintensive Therapy Study: (EU & EU reference countries only): Complete remission (CR)
Time Frame: Up to 36 months after last patient inclusion
CR rate per ELN 2022 criteria per Investigator assessment
Up to 36 months after last patient inclusion
Nonintensive Therapy Study: Bone marrow (BM) measurable residual disease (MRD) negativity
Time Frame: Up to 36 months after last patient inclusion
Central BM MRD negativity rate
Up to 36 months after last patient inclusion
Nonintensive Therapy Study: Complete remission (CR) + complete remission with partial hematologic recovery (CRh)
Time Frame: Up to 36 months after last patient inclusion
CR + CRh rate per ELN 2022 criteria per Investigator assessment
Up to 36 months after last patient inclusion
Nonintensive Therapy Study: Descriptive statistics of Adverse Events (AEs)
Time Frame: From start of treatment to 28 days from last dose of ziftomenib or placebo
Assessed by NCI-CTCAE v5.0
From start of treatment to 28 days from last dose of ziftomenib or placebo
Nonintensive Therapy Study: Area under the concentration-time curve (AUC) of ziftomenib and venetoclax
Time Frame: During treatment for up to 36 months after last patient inclusion
To characterize the AUC of ziftomenib and venetoclax
During treatment for up to 36 months after last patient inclusion
Nonintensive Therapy Study: Trough concentration (Ctrough) of ziftomenib and venetoclax
Time Frame: During treatment for up to 36 months after last patient inclusion
To characterize the Ctrough of ziftomenib and venetoclax
During treatment for up to 36 months after last patient inclusion
Intensive Therapy Study: (EU & EU reference countries only): Complete remission (CR) with bone marrow (BM) measurable residual disease (MRD) negativity in NPM1-m patients
Time Frame: Up to 36 months after last patient inclusion
CR rate per ELN 2022 criteria per Investigator assessment with central BM MRD negativity
Up to 36 months after last patient inclusion
Intensive Therapy Study: Overall survival (OS)
Time Frame: Defined as the time from randomization to date of death from any cause, up to 36 months after last patient inclusion
OS
Defined as the time from randomization to date of death from any cause, up to 36 months after last patient inclusion
Intensive Therapy Study: Descriptive statistics of Adverse Events (AEs)
Time Frame: From start of treatment to 28 days from last dose of ziftomenib or placebo
Assessed by NCI-CTCAE v5.0
From start of treatment to 28 days from last dose of ziftomenib or placebo
Intensive Therapy Study: Area under the concentration-time curve (AUC) of ziftomenib
Time Frame: During treatment for up to 36 months after last patient inclusion
To characterize the AUC of ziftomenib
During treatment for up to 36 months after last patient inclusion
Intensive Therapy Study: Trough concentration (Ctrough) of ziftomenib
Time Frame: During treatment for up to 36 months after last patient inclusion
To characterize the Ctrough of ziftomenib
During treatment for up to 36 months after last patient inclusion
Nonintensive Therapy Study: Health-related patient-reported outcomes (PRO) assessments
Time Frame: Up to 36 months after last patient inclusion
Health-related quality of life (HRQoL) was evaluated by EORTC QLQ-C30 global health status/quality of life composite scale in all randomized participants. The QLQ-C30 is a cancer-specific, self-administered questionnaire that contains 30 questions, covering global, functional, and symptom scales. Scores range from 0 to 100. Higher scores on global and functional scales indicated better quality of life (QoL), while higher scores on the symptom scales indicated declining QoL.
Up to 36 months after last patient inclusion
Intensive Therapy Study: Health-related patient-reported outcomes (PRO) assessments
Time Frame: Up to 36 months after last patient inclusion
Health-related quality of life (HRQoL) was evaluated by EORTC QLQ-C30 global health status/quality of life composite scale in all randomized participants. The QLQ-C30 is a cancer-specific, self-administered questionnaire that contains 30 questions, covering global, functional, and symptom scales. Scores range from 0 to 100. Higher scores on global and functional scales indicated better quality of life (QoL), while higher scores on the symptom scales indicated declining QoL.
Up to 36 months after last patient inclusion

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Kura Oncology, Inc.

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

September 26, 2025

Primary Completion (Estimated)

November 1, 2031

Study Completion (Estimated)

November 1, 2031

Study Registration Dates

First Submitted

May 16, 2025

First Submitted That Met QC Criteria

May 28, 2025

First Posted (Actual)

June 5, 2025

Study Record Updates

Last Update Posted (Actual)

August 20, 2026

Last Update Submitted That Met QC Criteria

August 19, 2026

Last Verified

August 1, 2026

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • KO-MEN-017
  • 2025-521314-25-00 (Ctis)

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

UNDECIDED

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

Subscribe