Acute Myeloid Leukemia (AML)Phase 3NCT07007312
Studies to Assess Ziftomenib in Combination With Ven+Aza or 7+3 in Patients With Untreated NPM1-m or KMT2A-r AML
A Phase 3 study testing Ziftomenib, Venetoclax and Azacitidine (AZA) in people with Acute Myeloid Leukemia (AML), run by Kura Oncology, Inc. at 136 sites in 17 countries.
Trial summary
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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Acute Myeloid Leukemia (AML)
- What is being tested?
- Ziftomenib, Venetoclax and Azacitidine (AZA)
- Type of treatment
- Pill or oral medicine, Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 1,300 participants
- Where
- 136 sitesin 17 countries
- Study dates
- 26 Sep 2025to Nov 2031
- Phase
- Phase 3
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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.
Show all 16Show fewer
- NONINTENSIVE THERAPY STUDY ONLY (VEN+AZA):
- Documented NPM1-m.
- 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):
- Documented NPM1-m or KMT2A-r (KMT2A-r patients with a partial tandem duplication are not eligible).
- 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.
- Ejection fraction of ≥50%.
- Fit for Intensive Therapy per Investigator opinion.
Who cannot join
- 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:
- Basal cell skin cancer or localized squamous cell cancer of the skin
- Previous malignancy confined and locally resected (or treated with other modalities) with curative intent
Show all 12Show fewer
- 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.
Trial locations and contacts
136 sites in 17 countries. Contact the study team or a site near you to ask about taking part.
136 sites in 17 countries
United States44 sites, 44 recruiting
- Recruiting
University of New Mexico
Albuquerque, New Mexico, 87131
- Recruiting
University of Michigan
Ann Arbor, Michigan, 48109
- Recruiting
University of Colorado
Aurora, Colorado, 80045
- Recruiting
Texas Oncology-Austin Midtown
Austin, Texas, 78705
- Recruiting
State University of New York at Buffalo
Buffalo, New York, 14263
- Recruiting
University of Vermont Medical Center
Burlington, Vermont, 05401
- Recruiting
University of North Carolina, Chapel Hill
Chapel Hill, North Carolina, 27514
- Recruiting
University of Virginia School of Medicine
Charlottesville, Virginia, 22903
- Recruiting
University of California, Fresno
Clovis, California, 93611
- Recruiting
Ohio State University
Columbus, Ohio, 43210
- Recruiting
Texas Oncology-Presbyterian Cancer Center
Dallas, Texas, 75231
- Recruiting
Colorado Blood Cancer Institute
Denver, Colorado, 80218
- Recruiting
Wayne State University School of Medicine
Detroit, Michigan, 48201
- Recruiting
Duke University Medical Center
Durham, North Carolina, 27710
- Recruiting
Willamette Valley Cancer Institute
Eugene, Oregon, 97401
- Recruiting
University of Kansas
Fairway, Kansas, 66205
- Recruiting
Banner MD Anderson Cancer Center
Gilbert, Arizona, 85234
- Recruiting
Hartford HealthCare Cancer Institute
Hartford, Connecticut, 06106
- Recruiting
University of Texas
Houston, Texas, 77030
- Recruiting
University of Iowa
Iowa City, Iowa, 52246
- Recruiting
University of California, San Diego
La Jolla, California, 92093
- Recruiting
University of Kentucky
Lexington, Kentucky, 40536
- Recruiting
Cedars-Sinai Medical Center
Los Angeles, California, 90048
- Recruiting
University of California, Los Angeles
Los Angeles, California, 90095
- Recruiting
University of Wisconsin
Madison, Wisconsin, 53792
- Recruiting
Virginia Cancer Specialists
Manassas, Virginia, 20110
- Recruiting
Baptist Clinical Research Institute
Memphis, Tennessee, 38120
- Recruiting
University of Miami
Miami, Florida, 33136
- Recruiting
Froedtert & Medical College of Wisconsin
Milwaukee, Wisconsin, 53226
- Recruiting
University of Minnesota
Minneapolis, Minnesota, 55455
- Recruiting
Tennessee Oncology
Nashville, Tennessee, 37203
- Recruiting
TriStar Centennial Medical Center
Nashville, Tennessee, 37203
- Recruiting
Rutgers Biomedical and Health Sciences
New Brunswick, New Jersey, 08903
- Recruiting
Yale University School of Medicine
New Haven, Connecticut, 06510
- Recruiting
Icahn School of Medicine at Mount Sinai
New York, New York, 10003
- Recruiting
Columbia University
New York, New York, 10032
- Recruiting
Weill Cornell Medical Center
New York, New York, 10065
- Recruiting
University of California, Irvine
Orange, California, 92868
- Recruiting
University of Pennsylvania
Philadelphia, Pennsylvania, 19104
- Recruiting
HealthPartners Cancer Center at Regions Hospital (MN)
Saint Paul, Minnesota, 55101
- Recruiting
Texas Oncology - San Antonio Medical Center
San Antonio, Texas, 78240
- Recruiting
Moffitt Cancer Center & Research Institute
Tampa, Florida, 33612
- Recruiting
WVU Medicine Wheeling Hospital
Wheeling, West Virginia, 26003
- Recruiting
University of Massachusetts
Worcester, Massachusetts, 01605
France17 sites, 17 recruiting
- Recruiting
Centre Hospitalier de Béziers
Béziers, Occitanie, 35400
- Recruiting
Hôpital Avicenne
Bobigny, Île-de-France Region, 93009
- Recruiting
Centre Hospitalier Universitaire Estaing - Clermont-Ferrand
Clermont-Ferrand, Auvergne-Rhône-Alpes, 63003
- Recruiting
Hôpital Universitaire Henri Mondor
Créteil, Val-de-Marne, 94010
- Recruiting
Centre Hospitalier de Versailles
Le Chesnay, Île-de-France Region, 78150
- Recruiting
Centre Hospitalier de Roubaix
Lille, Nord, 59000
- Recruiting
Centre Hospitalier Universitaire de Marseille - Hôpital de la Conception
Marseille, Provence-Alpes-Côte d'Azur Region, 13385
- Recruiting
Groupe Hospitalier de la Region de Mulhouse et Sud Alsace Hôpital Emile Muller
Mulhouse, Grand Est, 68100
- Recruiting
Centre Hospitalier Universitaire de Nantes
Nantes, Loire-Atlantique, 44000
- Recruiting
Hôpital Saint Louis
Paris, Île-de-France Region, 75010
- Recruiting
Hôpital Pitié-Salpêtrière
Paris, Île-de-France Region, 75013
- Recruiting
Centre Hospitalier Universitaire de Bordeaux
Pessac, New Aquitaine, 33604
- Recruiting
Hôpital Lyon Sud
Pierre-Bénite, Auvergne-Rhône-Alpes, 69495
- Recruiting
Centre Henri Becquerel
Rouen, Normandy, 76038
- Recruiting
Centre Hospitalier Universitaire de Saint Etienne
Saint-Priest-en-Jarez, Auvergne-Rhône-Alpes, 42270
- Recruiting
Centre Hospitalier Régional et Universitaire de Nancy Hôpitaux de Brabois
Vandœuvre-lès-Nancy, Grand Est, 54510
- Recruiting
Institut Gustave Roussy
Villejuif, Val-De-Marne, 94805
Spain11 sites, 11 recruiting
- Recruiting
Hospital General Universitario de Albacete
Albacete, Castille-La Mancha, 02006
- Recruiting
Hospital Universitario De Burgos
Burgos, Castille and León, 09006
- Recruiting
Hospital Universitario Infanta Leonor
Madrid, Madrid, 28031
- Recruiting
Hospital Universitario Fundación Jiménez Díaz
Madrid, Madrid, 28040
- Recruiting
Hospital Universitario 12 de Octubre
Madrid, Madrid, 28041
- Recruiting
Hospital Universitario Central de Asturias
Oviedo, Principality of Asturias, 33011
- Recruiting
Hospital Clinico Universitario de Salamanxa
Salamanca, Castille and León, 37007
- Recruiting
University Hospital Virgen Del Rocio S.L.
Seville, Andalusia, 41013
- Recruiting
Hospital Clinico Universitario de Valencia
Valencia, València, 46010
- Recruiting
Hospital Universitario Y Politecnico La Fe
Valencia, València, 46026
- Recruiting
Hospital Clinico Universitario Lozano Blesa
Zaragoza, Aragon, 50009
Italy10 sites, 10 recruiting
- Recruiting
Azienda Ospedaliera Universitaria SS Antonio e Biagio e Cesare Arrigo
Alessandria, Piedmont, 15121
- Recruiting
Azienda Sanitaria Territoriale di Ascoli Piceno
Ascoli Piceno, The Marches, 63100
- Recruiting
ASST Papa Giovanni XXIII
Bergamo, Lombardy, 94805
- Recruiting
L'IRCCS Azienda Ospedaliero - Universitaria di Bologna
Bologna, Emilia-Romagna, 40138
- Recruiting
ASST Spedali Civili di Brescia
Brescia, Lombardy, 25123
- Recruiting
Azienda Ospedaliero - Universitaria DI Modena
Modena, Emilia-Romagna, 41124
- Recruiting
Azienda Ospedaliero-Universitaria
Perugia, Umbria, 42270
- Recruiting
Hospital Santa Maria delle Croci
Ravenna, Emilia-Romagna, 48121
- Recruiting
ASUGI Ospedale Maggiore di Trieste
Trieste, Friuli Venezia Giulia, 76038
- Recruiting
Ematologia Ospedale San Bortolo
Vicenza, Veneto, 36100
South Korea10 sites, 10 recruiting
- Recruiting
Pusan National University Hospital
Busan-si, Gyeongsangnam-do, 49241
- Recruiting
Daegu Catholic University Hospital
Daegu, Gyeongsangbuk-do, 42472
- Recruiting
Chungnam National University Daejeon Hospital
Daejeon, Daejeon Gwang'Yeogsi, 35015
- Recruiting
Chonnam National University Hwasun Hospital
Hwasun, Jeollanam-do, 58128
- Recruiting
Gachon University Gil Medical Center
Namdong, Incheon Gwang'Yeogsi, 21565
- Recruiting
Dong-A University Hospital
Pusan, Gyeongsangnam-do, 49201
- Recruiting
Anam Hospital Korea University
Seoul, Seoul-T'Ukpyolsi, 02841
- Recruiting
The Catholic University of Korea Seoul St. Mary's Hospital
Seoul, Seoul-T'Ukpyolsi, 06591
- Recruiting
Samsung Medical Center
Seoul, Seoul-T'Ukpyolsi, 35015
- Recruiting
Ulsan University Hospital
Ulsan, Gyeongsangnam-do, 44033
Australia6 sites, 6 recruiting
- Recruiting
Austin Health
Heidelberg, Victoria, 3084
- Recruiting
Box Hill Hospital
Heidelberg, Victoria, 3128
- Recruiting
Gachon University Gil Medical Center
Kurralta Park, South Australia, 5037
- Recruiting
Northern Health Epping
Melbourne, Victoria, 3004
- Recruiting
Fiona Stanley Hospital
Murdoch, Western Australia, 6150
- Recruiting
Calvary Mater Newcastle
Waratah, New South Wales, 02298
Philippines6 sites, 6 recruiting
- Recruiting
Baguio General Hospital and Medical Center
Baguio City, Benguet, 02600
- Recruiting
Perpetual Succour Hospital
Cebu City, Central Visayas, 6000
- Recruiting
Philippine General Hospital
Ermita, National Capital Region, 1000
- Recruiting
University of Santo Tomas
Manila, National Capital Region, 1015
- Recruiting
The Medical City
Pasig, National Capital Region, 1605
- Recruiting
St. Luke's Medical Center - Quezon City
Quezon City, National Capital Region, 1112
Belgium5 sites, 5 recruiting
- Recruiting
Algemeen Ziekenhuis Sint-Jan Brugge
Bruges, West-Vlaanderen, 8800
- Recruiting
Universitair Ziekenhuis Gent
Ghent, Oost-Vlaanderen, 9000
- Recruiting
Universitair Ziekenhuis Brussel
Jette, Brussels Capital, 1090
- Recruiting
Algemeen Ziekenhuis Delta VZW
Roeselare, West-Vlaanderen, 8800
- Recruiting
Centre Hospitalier Régional Verviers East Belgium
Verviers, Walloonia, 4800
Greece5 sites, 5 recruiting
- Recruiting
University General Hospital of Alexandroupoli
Alexandroupoli, East Macedonia and Thrace, 68100
- Recruiting
Laiko General Hospital of Athens
Athens, Attica, 11527
- Recruiting
Attikon University General Hospital of Athens
Chaïdári, Attica, 12462
- Recruiting
General Hospital of Thessaloniki G. Papanikolaou
Exochi, Central Macedonia, 57010
- Recruiting
University General Hospital of Ioannina
Ioannina, Epirus, 45500
Taiwan4 sites, 4 recruiting
- Recruiting
Chang Bing Show Chwan Memorial Hospital
Lugang, Central Taiwan, 505
- Recruiting
Taichung Veterans General Hospital
Taichung, Central Taiwan, 407
- Recruiting
National Taiwan University Hospital
Taipei, Northern Taiwan, 10002
- Recruiting
Taipei Veterans General Hospital
Taipei, Northern Taiwan, 613
United Kingdom4 sites, 4 recruiting
- Not yet recruiting
St. Bartholomew's Hospital
London, England, EC1A 7BE
- Recruiting
St. Bartholomew's Hospital
London, England, EC1A 7BE
- Not yet recruiting
St. George's, University of London
London, England, SW17 0QT
- Recruiting
St. George's, University of London
London, England, SW17 0QT
Canada3 sites, 3 recruiting
- Recruiting
Royal Victoria Hospital
Montreal, Quebec, H4A3J1
- Recruiting
Centre hospitalier de l'Université de Montréal (CHUM)
Montreal, Quebec, QC H2x 3E4
- Recruiting
Gordon and Leslie Diamond Health Cancer Centre-Leukemia/BMT Program of BC
Vancouver, British Columbia, V5Z1M9
Hungary3 sites, 3 recruiting
- Recruiting
DPC-Hospital
Budapest, Budapest, 1097
- Recruiting
Győr-Moson-Sopron Vármegyei Petz Aladár Egyetemi Oktató Kórház
Győr, Gyor-Moson-Sopron County, 9024
- Recruiting
Kaposi Mor County Hospital
Kaposvár, Somogy County, 7400
Czechia2 sites, 2 recruiting
- Recruiting
Fakultni Nemocnice Ostrava
Ostrava-Poruba, Moravian-Silesian, 70852
- Recruiting
Fakultní Nemocnice Královské Vinohrady
Prague, Prague, 10034
Germany2 sites, 2 recruiting
- Recruiting
Universitätsklinikum Aachen
Aachen, North Rhine-Westphalia, 52074
- Recruiting
Helios Klinikum Bad Saarow
Bad Saarow, Brandenburg, 15526
Poland2 sites, 2 recruiting
- Recruiting
University Clinical Hospital Gdansk
Gdansk, Pomeranian Voivodeship, 80-214
- Recruiting
Szpital Wojewodzki w Opolu
Opole, Opole Voivodeship, 45-061
Portugal2 sites, 2 recruiting
- Recruiting
Hospital de Braga, Centro Clínico Académico de Braga
Braga, 1099-023
- Recruiting
Instituto Português de Oncologia de Lisboa Francisco Gentil - EPE
Lisbon, Lisbon District, 4200-072
About this study
Official title: 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".
Read the 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.
- Conditions
- Acute Myeloid Leukemia (AML)
- Being tested
- Ziftomenib, Placebo, Venetoclax, Azacitidine (AZA), Daunorubicin
- Keywords
- AML, Hematological malignancy, KMT2A, NPM1, Menin, Acute Leukemia, Leukemia, Acute Myeloid Leukemia, Newly diagnosed AML, Newly diagnosed KMT2A-r AML, Newly diagnosed NPM1m AML, Untreated AML, Untreated NPM1m AML, Untreated KMT2A-r AML, MLL
- Medical subject headings
- Leukemia, Myeloid, Acute, Hematologic Neoplasms, Leukemia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ZiftomenibDrug
Also called KO-539
Oral administration
PlaceboDrug
Oral administration
VenetoclaxDrug
Also called Venclexta, Venclyxto
Oral administration
Azacitidine (AZA)Drug
Also called Vidaza, Azadine
Intravenous or subcutaneous administration
DaunorubicinDrug
Also called Cerubidine, daunomycin
Intravenous administration
Cytarabine (Ara-C)Drug
Also called cytosine arabinoside (ara-C), Cytosar-U, Tarabine PFS
Intravenous administration
| Study group | Role | What they receive |
|---|---|---|
| Nonintensive Therapy Study, Arm A | Experimental | Ziftomenib in combination with venetoclax+azacitidine Receives: Drug: Ziftomenib; Drug: Venetoclax; Drug: Azacitidine (AZA) |
| Nonintensive Therapy Study, Arm B | Placebo comparator | Placebo in combination with venetoclax+azacitidine Receives: Drug: Placebo; Drug: Venetoclax; Drug: Azacitidine (AZA) |
| Intensive Therapy Study, Arm A | Experimental | Ziftomenib+cytarabine+daunorubicin (induction), ziftomenib+cytarabine (consolidation), ziftomenib (maintenance) Receives: Drug: Ziftomenib; Drug: Daunorubicin; Drug: Cytarabine (Ara-C) |
| Intensive Therapy Study, Arm B | Experimental | Ziftomenib+cytarabine+daunorubicin (induction), ziftomenib+cytarabine (consolidation), placebo (maintenance) Receives: Drug: Ziftomenib; Drug: Placebo; Drug: Daunorubicin; Drug: Cytarabine (Ara-C) |
| Intensive Therapy Study, Arm C | Placebo comparator | Placebo+cytarabine+daunorubicin (induction), placebo+cytarabine (consolidation), placebo (maintenance) Receives: Drug: Placebo; Drug: Daunorubicin; Drug: Cytarabine (Ara-C) |
What is being measured
The primary outcome is the main question the study is designed to answer.
Nonintensive Therapy Study: (Primary Endpoint for all countries): Overall survival (OS)
OS
Measured: 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)
CR rate per European Leukemia Network (ELN) 2022 criteria per Investigator assessment
Measured: Assessed up to 36 months after last patient inclusion
Intensive Therapy Study: (Primary Endpoint for all countries): Event-free survival (EFS)
EFS
Measured: 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
CR rate per ELN 2022 criteria per Investigator assessment with central BM MRD negativity
Measured: Assessed up to 36 months after last patient inclusion
Secondary outcomes (13)
Nonintensive Therapy Study: (EU & EU reference countries only): Complete remission (CR)
CR rate per ELN 2022 criteria per Investigator assessment
Measured: Up to 36 months after last patient inclusion
Nonintensive Therapy Study: Bone marrow (BM) measurable residual disease (MRD) negativity
Central BM MRD negativity rate
Measured: Up to 36 months after last patient inclusion
Nonintensive Therapy Study: Complete remission (CR) + complete remission with partial hematologic recovery (CRh)
CR + CRh rate per ELN 2022 criteria per Investigator assessment
Measured: Up to 36 months after last patient inclusion
Nonintensive Therapy Study: Descriptive statistics of Adverse Events (AEs)
Assessed by NCI-CTCAE v5.0
Measured: 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
To characterize the AUC of ziftomenib and venetoclax
Measured: During treatment for up to 36 months after last patient inclusion
Nonintensive Therapy Study: Trough concentration (Ctrough) of ziftomenib and venetoclax
To characterize the Ctrough of ziftomenib and venetoclax
Measured: During treatment for up to 36 months after last patient inclusion
Nonintensive Therapy Study: Health-related patient-reported outcomes (PRO) assessments
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.
Measured: 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
CR rate per ELN 2022 criteria per Investigator assessment with central BM MRD negativity
Measured: Up to 36 months after last patient inclusion
Intensive Therapy Study: Overall survival (OS)
OS
Measured: 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)
Assessed by NCI-CTCAE v5.0
Measured: From start of treatment to 28 days from last dose of ziftomenib or placebo
Intensive Therapy Study: Health-related patient-reported outcomes (PRO) assessments
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.
Measured: Up to 36 months after last patient inclusion
Intensive Therapy Study: Area under the concentration-time curve (AUC) of ziftomenib
To characterize the AUC of ziftomenib
Measured: During treatment for up to 36 months after last patient inclusion
Intensive Therapy Study: Trough concentration (Ctrough) of ziftomenib
To characterize the Ctrough of ziftomenib
Measured: During treatment for up to 36 months after last patient inclusion
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted5 Jun 2025
- Study start26 Sep 2025
- Primary completionNov 2031 (estimated)
- Study completionNov 2031 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Acute Myeloid Leukemia (AML)Studies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 1,300 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Kura Oncology, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07007312
- Sponsor's protocol ID
- KO-MEN-017
- Other IDs
- 2025-521314-25-00, 2025
- Status last verified
- Aug 2026
- First submitted
- 16 May 2025
- Last updated
- 13 Oct 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Undecided
- Expanded access
- Available