Testing Ziftomenib against a placebo for acute myeloid leukemia (AML)
Official title: Studies to Assess Ziftomenib in Combination With Ven+Aza or 7+3 in Patients With Untreated NPM1-m or KMT2A-r AML
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
Interventions
Ziftomenib
Oral administration
Placebo
Oral administration
Venetoclax
Oral administration
Azacitidine (AZA)
Intravenous or subcutaneous administration
Daunorubicin
Intravenous administration
Cytarabine (Ara-C)
Intravenous administration
Canadian Sites (1)
Royal Victoria Hospital
Montreal, Quebec, Canada
Eligibility Criteria
See who this study is looking for28 criteria
The trial’s own eligibility text, word for word from the registry. Only the trial site can say who takes part.
Inclusion
- +Age ≥18 years at time of signing the informed consent formInformed consentThe process of being told what taking part involves, then choosing freely.Read more →.
- +i. Age ≥75, OR
- +The following criteria apply to both the Nonintensive Therapy Study and the Intensive Therapy Study unless otherwise noted:
- +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 protocolProtocolThe detailed plan a study must follow.Read more → requirements.
- +NONINTENSIVE THERAPY STUDY ONLY (VEN+AZA):
- +Documented NPM1-m.
- +Patients considered ineligible for Intensive Therapy defined by the following:
- +INTENSIVE THERAPY STUDY ONLY (7+3):
- +Documented NPM1-m or KMT2A-r (KMT2A-r patients with a partial tandem duplication are not eligible).
- +Ejection fraction of ≥50%.
- +Fit for Intensive Therapy per InvestigatorPrincipal investigatorThe doctor or researcher responsible for running the study at a site.Read more → opinion.
- +ii. Age \<75 with an ECOG performance status of 2 or cardiac, renal, or hepatic impairment per protocol criteria.
- +A female of childbearing potential must agree to use adequate contraception from the time of screeningScreeningThe checks done before joining, to see whether a study fits.Read more → 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.
- +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 SOCStandard of careThe treatment normally given for a condition outside a study.Read more →). Lack of access to an FLT3 inhibitor is not considered "ineligible" for FLT3-targeted therapy.
Exclusion
- −Known uncontrolled HIV infection or known active hepatitis B virus, hepatitis C virus infection, or other uncontrolled infection.
- −Prior therapy for AML (except hydroxyurea or leukapheresis for WBC controlControl groupThe group a new treatment is measured against.Read more →).
- −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
- −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/placeboPlaceboA dummy treatment with no active medicine, used for comparison.Read more →. Note: Hydroxyurea and/or leukapheresis are permitted to meet this criterion.
- −Uncontrolled intercurrent illness including but not limited to, cardiac illness as defined in the protocolProtocolThe detailed plan a study must follow.Read more →.
- −Women who are pregnant or lactating.
In plain language
Assembled directly from this trial’s registry record. Every sentence traces to a field below — nothing here is generated or interpreted.
What this study is
This study is testing a treatment for a condition.
Phase 3Phase 3A large study comparing a treatment against the current standard.Read more → — a large study comparing this against the current standard, usually across many hospitals.
What is being given or done in this study: Ziftomenib, PlaceboPlaceboA dummy treatment with no active medicine, used for comparison.Read more →, Venetoclax, Azacitidine (AZA), and 2 more.
From the trial registry
Built from these fields:
- designModule.designInfo.primaryPurpose
- designModule.phases
- armsInterventionsModule.interventions[].name
How the study is run
Which group you would be placed in is decided by chance, like a coin flip — not by you and not by your doctor.
You, the study team, the people giving the treatment, and the people analysing the results would all be kept unaware of which group you are in until the study ends.
From the trial registry
Built from these fields:
- designModule.designInfo.allocation
- designModule.designInfo.maskingInfo.masking
Is there a placebo?
One group receives a placeboPlaceboA dummy treatment with no active medicine, used for comparison.Read more → — a dummy treatment with no active medicine in it.
There are 5 groups in this study.
From the trial registry
Built from this field:
- armsInterventionsModule.armGroups[].type
Who the study is looking for
The study lists a minimum age of 18 years, with no upper limit given.
The study is open to people of any sex.
The study does not accept healthy volunteersHealthy volunteerSomeone without the condition being studied who takes part anyway.Read more →.
These are the criteria the study lists. Only the trial siteTrial siteA hospital or clinic where a study is actually run.Read more → can decide whether someone can take part.
From the trial registry
Built from these fields:
- eligibilityModule.minimumAge
- eligibilityModule.sex
- eligibilityModule.healthyVolunteers
How big and how long
The study aims to enrol about 1,300 people.
The study is currently expected to finish around November 2031.
The main measurement is taken over: Defined as the time from randomizationRandomisedWhich group you go into is decided by chance, not by you or your doctor.Read more → to date of death from any cause, assessed up to 36 months after last patient inclusionInclusion criteriaThe things you must have or be for a study to consider you.Read more →.
From the trial registry
Built from these fields:
- designModule.enrollmentInfo.count
- statusModule.completionDateStruct.date
- outcomesModule.primaryOutcomes[].timeFrame
What the study measures
Nonintensive Therapy Study: (Primary EndpointPrimary endpointThe single main question the study is designed to answer.Read more → for all countries): Overall survival (OS) — measured over Defined as the time from randomizationRandomisedWhich group you go into is decided by chance, not by you or your doctor.Read more → to date of death from any cause, assessed up to 36 months after last patient inclusionInclusion criteriaThe things you must have or be for a study to consider you.Read more →.
Nonintensive Therapy Study: (Dual Primary EndpointEndpointThe specific thing a study measures to answer its question.Read more → for US & US reference countries only): Complete remission (CR) — measured over Assessed up to 36 months after last patient inclusion.
Intensive Therapy Study: (Primary Endpoint for all countries): Event-free survival (EFS) — measured over 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 — measured over Assessed up to 36 months after last patient inclusion.
From the trial registry
Built from these fields:
- outcomesModule.primaryOutcomes[].measure
- outcomesModule.primaryOutcomes[].timeFrame
Source: NCT07007312 on ClinicalTrials.gov. The full registry text is further down this page — this summary never replaces it.
Not medical advice. What do these terms mean?
What is being tested — in plain terms
About ZiftomenibDrug
Oral administration
From the trial registry — its own words, unedited.
What a drug is here: A medicine — a chemical or small-molecule treatment — given as the thing being studied.
Read the full explanation → · in clinical review
About PlaceboDrug
Oral administration
From the trial registry — its own words, unedited.
What a drug is here: A medicine — a chemical or small-molecule treatment — given as the thing being studied.
Read the full explanation → · in clinical review
About VenetoclaxDrug
Oral administration
From the trial registry — its own words, unedited.
What a drug is here: A medicine — a chemical or small-molecule treatment — given as the thing being studied.
Read the full explanation → · in clinical review
About Azacitidine (AZA)Drug
Intravenous or subcutaneous administration
From the trial registry — its own words, unedited.
What a drug is here: A medicine — a chemical or small-molecule treatment — given as the thing being studied.
Read the full explanation → · in clinical review
About DaunorubicinDrug
Intravenous administration
From the trial registry — its own words, unedited.
What a drug is here: A medicine — a chemical or small-molecule treatment — given as the thing being studied.
Read the full explanation → · in clinical review
About Cytarabine (Ara-C)Drug
Intravenous administration
From the trial registry — its own words, unedited.
What a drug is here: A medicine — a chemical or small-molecule treatment — given as the thing being studied.
Read the full explanation → · in clinical review
Common questions
Answered from this trial’s registry record. Where the record doesn’t say, these answers say so rather than filling the gap.
Am I eligible for this trial?
Only the trial siteTrial siteA hospital or clinic where a study is actually run.Read more → can decide whether someone can take part. Concord cannot make that determination, and neither can any tool that has not examined you.
What the study lists: a minimum age of 18 years.
The full inclusionInclusion criteriaThe things you must have or be for a study to consider you.Read more → and exclusion criteriaExclusion criteriaThe things that would prevent someone from taking part.Read more → are published on this page, exactly as the study team wrote them.
The useful next step is to bring this trial to your doctor. Answering a few questions first gives them something concrete to review.
From the trial registry
- eligibilityModule.minimumAge
- eligibilityModule.eligibilityCriteria
Is there a placebo?
Yes. This study includes a placeboPlaceboA dummy treatment with no active medicine, used for comparison.Read more → group — a dummy treatment with no active medicine in it.
From the trial registry
- armsInterventionsModule.armGroups[].type
Would I know which treatment I am getting?
You, the study team, the people giving the treatment, and the people analysing the results would all be kept unaware of which group you are in until the study ends.
Which group you would be placed in is decided by chance, like a coin flip — not by you and not by your doctor.
From the trial registry
- designModule.designInfo.maskingInfo.masking
- designModule.designInfo.allocation
Who can join?
The study lists a minimum age of 18 years, with no upper limit given.
It is open to people of any sex.
It does not accept healthy volunteersHealthy volunteerSomeone without the condition being studied who takes part anyway.Read more →.
Only the trial siteTrial siteA hospital or clinic where a study is actually run.Read more → can confirm whether a particular person can take part.
From the trial registry
- eligibilityModule.minimumAge
- eligibilityModule.sex
- eligibilityModule.healthyVolunteers
How long would this take?
The study's main measurement is taken over: Defined as the time from randomizationRandomisedWhich group you go into is decided by chance, not by you or your doctor.Read more → to date of death from any cause, assessed up to 36 months after last patient inclusionInclusion criteriaThe things you must have or be for a study to consider you.Read more →.
The study as a whole is currently expected to finish around 2031-11.
How long any one person takes part can differ from the study length. The study team can tell you what the schedule looks like in practice.
From the trial registry
- outcomesModule.primaryOutcomes[].timeFrame
- statusModule.completionDateStruct.date
How many people are taking part?
The study aims to enrol about 1,300 people.
From the trial registry
- designModule.enrollmentInfo.count
Where is this happening?
This study lists 6 locations, including: Montreal, Quebec, Canada; Ann Arbor, Michigan, United States; Detroit, Michigan, United States; Minneapolis, Minnesota, United States; Buffalo, New York, United States; New York, New York, United States.
Sites can open and close during a study, so confirm with the team before travelling.
From the trial registry
- trial_locations
About This Trial
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".
Other Sites (7)
University of Michigan
Ann Arbor, Michigan, United States
Wayne State University School of Medicine
Detroit, Michigan, United States
University of Minnesota
Minneapolis, Minnesota, United States
State University of New York at Buffalo
Buffalo, New York, United States
Icahn School of Medicine at Mount Sinai
New York, New York, United States
Columbia University
New York, New York, United States
Weill Cornell Medical Center
New York, New York, United States
Worth passing on?
Save it to the Care Package and send it to them, or to their doctor, in one message. Whether they follow it up is theirs to decide.
Review the Care PackageThis page is for informational purposes only and does not constitute medical advice. Clinical trial eligibility can only be determined by the trial site after proper screening. Trial information is sourced from ClinicalTrials.gov and may not reflect the most current status. Always consult your healthcare provider before making decisions about clinical trial participation.