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Studies to Assess Ziftomenib in Combination With Ven+Aza or 7+3 in Patients With Untreated NPM1-m or KMT2A-r AML
NCT07007312 · Kura Oncology, Inc.
In plain English
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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
About this study
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.
Eligibility criteria
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 design
Enrollment target: 1300 participants
Allocation: randomized
Masking: quadruple
Age groups: adult, older_adult
Timeline
Starts: 2025-09-26
Estimated completion: 2031-11
Last updated: 2026-04-09
Interventions
Drug: ZiftomenibDrug: PlaceboDrug: VenetoclaxDrug: Azacitidine (AZA)Drug: DaunorubicinDrug: Cytarabine (Ara-C)
Primary outcomes
- • Nonintensive Therapy Study: (Primary Endpoint for all countries): Overall survival (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) (Assessed up to 36 months after last patient inclusion)
- • Intensive Therapy Study: (Primary Endpoint for all countries): Event-free survival (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)
Sponsor
Kura Oncology, Inc. · industry
Contacts & investigators
ContactKura Medical Information · contact · medinfo@kuraoncology.com · 844-KURAONC (844-587-2662)
All locations (39)
Banner MD Anderson Cancer CenterRecruiting
Gilbert, Arizona, United States
University of California, FresnoRecruiting
Clovis, California, United States
University of California, San DiegoRecruiting
La Jolla, California, United States
Cedars-Sinai Medical CenterRecruiting
Los Angeles, California, United States
University of California, IrvineRecruiting
Orange, California, United States
University of ColoradoRecruiting
Aurora, Colorado, United States
Colorado Blood Cancer InstituteRecruiting
Denver, Colorado, United States
Hartford HealthCare Cancer InstituteRecruiting
Hartford, Connecticut, United States
Yale University School of MedicineRecruiting
New Haven, Connecticut, United States
University of MiamiRecruiting
Miami, Florida, United States
Moffitt Cancer Center & Research InstituteRecruiting
Tampa, Florida, United States
University of KentuckyRecruiting
Lexington, Kentucky, United States
Wayne State University School of MedicineRecruiting
Detroit, Michigan, United States
University of MinnesotaRecruiting
Minneapolis, Minnesota, United States
Rutgers Biomedical and Health SciencesRecruiting
New Brunswick, New Jersey, United States
University of New MexicoRecruiting
Albuquerque, New Mexico, United States
Icahn School of Medicine at Mount SinaiRecruiting
New York, New York, United States
Weill Cornell Medical CenterRecruiting
New York, New York, United States
University of North Carolina, Chapel HillRecruiting
Chapel Hill, North Carolina, United States
Duke University Medical CenterRecruiting
Durham, North Carolina, United States
Ohio State UniversityRecruiting
Columbus, Ohio, United States
Willamette Valley Cancer InstituteRecruiting
Eugene, Oregon, United States
University of PennsylvaniaRecruiting
Philadelphia, Pennsylvania, United States
Baptist Clinical Research InstituteRecruiting
Memphis, Tennessee, United States
Tennessee OncologyRecruiting
Nashville, Tennessee, United States
TriStar Centennial Medical CenterRecruiting
Nashville, Tennessee, United States
Texas Oncology-Austin MidtownRecruiting
Austin, Texas, United States
Texas Oncology-Presbyterian Cancer CenterRecruiting
Dallas, Texas, United States
University of TexasRecruiting
Houston, Texas, United States
Texas Oncology - San Antonio Medical CenterRecruiting
San Antonio, Texas, United States
University of Virginia School of MedicineRecruiting
Charlottesville, Virginia, United States
Virginia Cancer SpecialistsRecruiting
Manassas, Virginia, United States
WVU Medicine Wheeling HospitalRecruiting
Wheeling, West Virginia, United States
Froedtert & Medical College of WisconsinRecruiting
Milwaukee, Wisconsin, United States
Centre Hospitalier de BéziersRecruiting
Béziers, France
Centre Hospitalier Universitaire de NantesRecruiting
Nantes, France
Dong-A University HospitalRecruiting
Busan, South Korea
Chungnam National University Daejeon HospitalRecruiting
Daejeon, South Korea
Chonnam National University Hwasun HospitalRecruiting
Hwasun, South Korea