A Phase 3 interventional study of Ziftomenib and Placebo in Acute Myeloid Leukemia (AML), sponsored by Kura Oncology, Inc.. Recruiting at 136 sites in 17 countries. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2026-10-13.
Sponsored by Kura Oncology, Inc. · Phase 3, Interventional, and Treatment
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".
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:
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:
Patients will be randomized to treatment arms in a double-blind manner.
3,667 studies on the registry are indexed under Leukemia, Myeloid, Acute; 816 are open to participants now.
This study's planned enrollment of 1,300 is above the median of 40 across 3,005 interventional studies indexed under Leukemia, Myeloid, Acute.
Browse Leukemia, Myeloid, Acute studies →Kura Oncology, Inc. is the lead sponsor of 18 studies on the registry; 8 are open to participants now.
Counted across the registry records on this site, refreshed daily.
Key Inclusion Criteria:
The following criteria apply to both the Nonintensive Therapy Study and the Intensive Therapy Study unless otherwise noted:
NONINTENSIVE THERAPY STUDY ONLY (VEN+AZA):
Patients considered ineligible for Intensive Therapy defined by the following:
INTENSIVE THERAPY STUDY ONLY (7+3):
Key Exclusion Criteria:
History of other active concurrent prior to study entry except:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
A scale of how much an illness limits everyday activity. For people being assessed to join a study, scores run from 0 (fully active) to 4; lower is more active.
Studies most often ask for 0–1 or 0–2. Your doctor gives the score; the WHO scale uses the same steps. The full scale also has 5, which records death.
Able to become pregnant: from the first period until menopause, unless permanently unable to through surgery (removal of the womb, or of both ovaries or both fallopian tubes).
Criteria for people of childbearing potential usually ask for a negative pregnancy test and for contraception during the study and for a time after it.
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
The share of blood the heart’s main pumping chamber (the left ventricle) pushes out with each beat, measured by an echocardiogram (heart ultrasound) or a MUGA scan. About 50–70% is normal.
Malignancy means cancer. Criteria about another or earlier malignancy ask whether you have had a different cancer, usually within a set number of years.
Some are commonly allowed, such as treated skin cancers other than melanoma and early cancers removed completely — check the exceptions this study lists.
Neoadjuvant treatment is given before the main treatment (usually surgery), often to shrink a tumour. Adjuvant treatment is given after it, to lower the chance of the cancer coming back.
The human immunodeficiency virus, which weakens the immune system.
Many studies now accept people whose HIV is controlled on treatment, judged by the viral load and the CD4 count. Check the limits this study sets.
Hepatitis B is a virus that infects the liver. These are separate tests: HBsAg positive usually means a current infection (a recent vaccination can briefly cause it too); total anti-HBc (HBcAb) positive means an infection now or in the past; HBV DNA measures how much virus is in the blood.
Some studies accept a past infection if HBV DNA is undetectable, sometimes with antiviral medicine during the study. Check what this study allows.
Hepatitis C is a virus that infects the liver. An HCV antibody test can stay positive after an infection has cleared (and is occasionally positive without one); an HCV RNA test shows whether the virus is there now.
After treatment, hepatitis C counts as cured when HCV RNA is still undetectable 12 weeks or more after the treatment ended. Studies often accept people cured this way.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
ECOG performance status
A scale of how much an illness limits everyday activity. For people being assessed to join a study, scores run from 0 (fully active) to 4; lower is more active.
Studies most often ask for 0–1 or 0–2. Your doctor gives the score; the WHO scale uses the same steps. The full scale also has 5, which records death.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Childbearing potential
Able to become pregnant: from the first period until menopause, unless permanently unable to through surgery (removal of the womb, or of both ovaries or both fallopian tubes).
Criteria for people of childbearing potential usually ask for a negative pregnancy test and for contraception during the study and for a time after it.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Contraception
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
LVEF (left ventricular ejection fraction)
The share of blood the heart’s main pumping chamber (the left ventricle) pushes out with each beat, measured by an echocardiogram (heart ultrasound) or a MUGA scan. About 50–70% is normal.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Cancer (malignancy)
Malignancy means cancer. Criteria about another or earlier malignancy ask whether you have had a different cancer, usually within a set number of years.
Some are commonly allowed, such as treated skin cancers other than melanoma and early cancers removed completely — check the exceptions this study lists.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Adjuvant and neoadjuvant
Neoadjuvant treatment is given before the main treatment (usually surgery), often to shrink a tumour. Adjuvant treatment is given after it, to lower the chance of the cancer coming back.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
HIV
The human immunodeficiency virus, which weakens the immune system.
Many studies now accept people whose HIV is controlled on treatment, judged by the viral load and the CD4 count. Check the limits this study sets.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Hepatitis B tests
Hepatitis B is a virus that infects the liver. These are separate tests: HBsAg positive usually means a current infection (a recent vaccination can briefly cause it too); total anti-HBc (HBcAb) positive means an infection now or in the past; HBV DNA measures how much virus is in the blood.
Some studies accept a past infection if HBV DNA is undetectable, sometimes with antiviral medicine during the study. Check what this study allows.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Hepatitis C tests
Hepatitis C is a virus that infects the liver. An HCV antibody test can stay positive after an infection has cleared (and is occasionally positive without one); an HCV RNA test shows whether the virus is there now.
After treatment, hepatitis C counts as cured when HCV RNA is still undetectable 12 weeks or more after the treatment ended. Studies often accept people cured this way.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Ziftomenib in combination with venetoclax+azacitidine
Drug: Ziftomenib · Drug: Venetoclax · Drug: Azacitidine (AZA)
Placebo in combination with venetoclax+azacitidine
Drug: Placebo · Drug: Venetoclax · Drug: Azacitidine (AZA)
Ziftomenib+cytarabine+daunorubicin (induction), ziftomenib+cytarabine (consolidation), ziftomenib (maintenance)
Drug: Ziftomenib · Drug: Daunorubicin · Drug: Cytarabine (Ara-C)
Ziftomenib+cytarabine+daunorubicin (induction), ziftomenib+cytarabine (consolidation), placebo (maintenance)
Drug: Ziftomenib · Drug: Placebo · Drug: Daunorubicin · Drug: Cytarabine (Ara-C)
Placebo+cytarabine+daunorubicin (induction), placebo+cytarabine (consolidation), placebo (maintenance)
Drug: Placebo · Drug: Daunorubicin · Drug: Cytarabine (Ara-C)
Oral administration
Also known as: KO-539
Oral administration
Oral administration
Also known as: Venclexta, Venclyxto
Intravenous or subcutaneous administration
Also known as: Vidaza, Azadine
Intravenous administration
Also known as: Cerubidine, daunomycin
Intravenous administration
Also known as: cytosine arabinoside (ara-C), Cytosar-U, Tarabine PFS
Nonintensive Therapy Study: (Primary Endpoint for all countries): Overall survival (OS)
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
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
Time frame: Assessed up to 36 months after last patient inclusion
Intensive Therapy Study: (Primary Endpoint for all countries): Event-free survival (EFS)
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
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
Time frame: Assessed up to 36 months after last patient inclusion
Nonintensive Therapy Study: (EU & EU reference countries only): Complete remission (CR)
CR rate per ELN 2022 criteria per Investigator assessment
Time frame: Up to 36 months after last patient inclusion
Nonintensive Therapy Study: Bone marrow (BM) measurable residual disease (MRD) negativity
Central BM MRD negativity rate
Time frame: 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
Time frame: Up to 36 months after last patient inclusion
Nonintensive Therapy Study: Descriptive statistics of Adverse Events (AEs)
Assessed by NCI-CTCAE v5.0
Time frame: 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
Time frame: 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
Time frame: 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.
Time frame: 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
Time frame: Up to 36 months after last patient inclusion
Intensive Therapy Study: Overall survival (OS)
OS
Time frame: 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
Time frame: 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.
Time frame: 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
Time frame: 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
Time frame: During treatment for up to 36 months after last patient inclusion
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Started Sep 2025 (actual). Primary completion is estimated for Nov 2031, 74 months after the start.
Of 42 industry-sponsored phase 3 interventional studies under Leukemia, Myeloid, Acute, half reached primary completion within 42 months of starting (the median), counting those still running; a quarter within 30 and three quarters within 62. This study's planned 74 months is above that.
Counting every sponsor, half of 120 studies reached it within 74 months; a quarter within 39 and three quarters within 99.
How these figures are worked out, and which studies are left out of them.
From the registry record's own update history. This site started tracking changes on Sep 25, 2026; for anything earlier, see the record history on ClinicalTrials.gov ↗
Eligibility is decided by the study team. Share this record with your doctor or contact the team directly.
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Studies of the drugs tested here: Ziftomenib · Venetoclax · Azacitidine · Daunorubicin · Cytarabine
Kura Oncology, Inc.