Recruiting

Phase 1

Ziftomenib, Venetoclax, AzacitidineA Study to Investigate the Safety and Tolerability of Ziftomenib in Combination With Venetoclax/Azacitidine, Venetoclax, 7+3, or 7+3+Quizartinib in Patients With AML

Sponsor:

Kura Oncology, Inc.

Code:

NCT05735184

Conditions

Acute Myeloid Leukemia

Mixed Lineage Leukemia Gene Mutation

Refractory AML

AML With Mutated NPM1

Acute Myeloid Leukemia Recurrent

Eligibility Criteria

Sex: All

Age: 18+

Healthy Volunteers: Not accepted

Interventions

Ziftomenib

Venetoclax

Azacitidine

Daunorubicin

Cytarabine

Study Details

Brief summary:

Ziftomenib is an investigational drug in development for the treatment of patients with acute myeloid leukemia (AML) with certain genetic alterations.

This protocol has 3 separate arms that will investigate the benefits and risks of adding ziftomenib to standard-of-care (SOC) drug treatments in patients who have AML with certain genetic mutations. Both newly diagnosed and relapsed refractory patients with AML will be assigned to different cohorts based on specific study criteria and physician discretion.

The purpose of this study is to assess the safety, tolerability, and early signs of efficacy of ziftomenib in combination with SOC drugs to treat AML.

Conditions

Acute Myeloid Leukemia

Mixed Lineage Leukemia Gene Mutation

Refractory AML

AML With Mutated NPM1

Acute Myeloid Leukemia Recurrent

Study ID

NCT05735184

Start date

Jul 18, 2023

Status verified date

Mar, 2026

Completion date

Apr, 2030

Anticipated

Primary completion date

Apr, 2030

Anticipated

Eligibility Criteria

Sex: All

Age: 18+

Healthy Volunteers: Not accepted

Key Inclusion Criteria:

Patients must have a documented NPM1 mutation or KMT2A rearrangement and have either newly diagnosed or relapsed/refractory AML

Those intending treatment with intensive chemotherapy in Arm C should be NPM1-m and FLT3-ITD+ with an allelic ratio ≥0.05 and eligible for FLT3-targeted treatment
Eastern Cooperative Oncology Group (ECOG) performance status of 0, 1, or 2
Adequate liver, renal, and cardiac function according to protocol defined criteria
A female of childbearing potential must agree to use adequate contraception as well as a double barrier method from the time of screening through 180 days following the last dose of study intervention. A male of childbearing potential must agree to use abstinence or use a double barrier method of contraception from the time of screening through 180 days following the last dose of study intervention

Female patients of childbearing potential who receive quizartinib in Arm C should use a highly effective method of contraception during quizartinib treatment and for 7 months after the last dose

Key Exclusion Criteria:

Diagnosis of either acute promyelocytic leukemia or blast phase chronic myeloid leukemia
Known history of BCR-ABL alteration
Advanced malignant hepatic tumor
Administration of live attenuated vaccines within 14 days prior to, during, or after treatment until B-cell recovery
Active central nervous system (CNS) involvement by AML.
Clinical signs/symptoms of leukostasis or WBC > 25,000 / microliter. Hydroxyurea and/or leukapheresis and/or up to 2 doses of cytarabine if used per institutional SOC for control of leukocytosis are permitted to meet this criterion
Not recovered to Grade ≤1 (NCI-CTCAE v5.0) from all nonhematological toxicities except for alopecia
Known clinically active human immunodeficiency virus, active hepatitis B or active hepatitis C infection
For newly diagnosed cohorts: received prior chemotherapy for leukemia, except hydroxyurea and/or leukapheresis and/or up to 2 doses of cytarabine per institutional standards to control leukocytosis, or prior treatment with all-transretinoic acid for initially suspected acute promyelocytic leukemia
For relapsed/refractory cohorts: received chemotherapy, immunotherapy, radiotherapy, or any ancillary therapy that is considered to be investigational < 14 days prior to the first dose of ziftomenib or within 5 drug half-lives prior to the first dose of study drug
Uncontrolled intercurrent illness including, but not limited to, cardiac illness as defined in the protocol
Mean QT interval corrected for heart rate by Fredericia's formula (QTcF)

Arm A and Arm B: >480 ms on triplicate ECGs
Arm C: >450 ms on triplicate ECGs
Uncontrolled infection
Women who are pregnant or lactating
An active malignancy and currently receiving chemotherapy for that malignancy or disease that is uncontrolled/progressing
Patients who have active GVHD requiring >0.5 mg/kg prednisone or any new or increase in immunosuppressants in the prior 2 weeks for GVHD treatment

Study Design

Enrollment

420 participants

Anticipated

Allocation

Non randomized

Intervention Model

Parallel Assignment

Primary purpose

Treatment

Interventions and Outcome Measures

Arms

experimental: Dose Escalation: Ziftomenib with Venetoclax and Azacitidine in R/R NPM1-m (A-1)

Ziftomenib with Venetoclax and Azacitidine in relapsed/refractory NPM1-m AML patients who have failed at least one prior line of therapy

experimental: Dose Validation/Expansion: Ziftomenib with Venetoclax and Azacitidine in R/R NPM1-m (A-1)

Ziftomenib with Venetoclax and Azacitidine in relapsed/refractory NPM1-m AML patients who have failed at least one prior line of therapy

experimental: Dose Escalation: Ziftomenib with 7+3 in 1L NPM1-m/FLT3 wildtype (A-2)

Ziftomenib with 7+3 in newly diagnosed NPM1-m AML patients who are candidates for intensive chemotherapy and must be FLT3 wildtype or ITD ratio <0.05

experimental: Dose Validation/Expansion: Ziftomenib with 7+3 in 1L NPM1-m/FLT3 wildtype (A-2)

Ziftomenib with 7+3 in newly diagnosed NPM1-m AML patients who are candidates for intensive chemotherapy and must be FLT3 wildtype or ITD ratio <0.05

experimental: Dose Validation/Expansion: Ziftomenib with Venetoclax in R/R NPM1-m (A-3)

Ziftomenib with Venetoclax in relapsed/refractory NPM1-m AML patients who have failed at least one prior line of therapy

experimental: Dose Validation/Expansion: Ziftomenib with Venetoclax and Azacitidine in 1L NPM1-m (A-4)

Ziftomenib with Venetoclax and Azacitidine in newly diagnosed NPM1-m AML patients

experimental: Dose Escalation: Ziftomenib with Venetoclax and Azacitidine in R/R KMT2A-r (B-1)

Ziftomenib with Venetoclax and Azacitidine in relapsed/refractory KMT2A-r AML patients who have failed at least one prior line of therapy

experimental: Dose Validation/Expansion: Ziftomenib with Venetoclax and Azacitidine in R/R KMT2A-r (B-1)

Ziftomenib with Venetoclax and Azacitidine in relapsed/refractory KMT2A-r AML patients who have failed at least one prior line of therapy

experimental: Dose Escalation: Ziftomenib with 7+3 in 1L KMT2A-r (B-2)

Ziftomenib with 7+3 in newly diagnosed KMT2A-r AML patients who are candidates for intensive chemotherapy

experimental: Dose Validation/Expansion: Ziftomenib with 7+3 in 1L KMT2A-r (B-2)

Ziftomenib with 7+3 in newly diagnosed KMT2A-r AML patients who are candidates for intensive therapy

experimental: Dose Validation/Expansion: Ziftomenib with Venetoclax + Azacitidine in 1L KMT2A-r (B-3)

Ziftomenib with Venetoclax and Azacitidine in newly diagnosed KMT2A-r AML patients

experimental: Dose Escalation: Ziftomenib with 7+3+quizartinib in 1L NPM1-m/FLT3-ITD+ AML patients (C-1)

Ziftomenib with 7+3 and quizartinib in newly diagnosed NPM1-m and FLT3-ITD+ (with allelic ratio ≥0.05) AML patients who are candidates for IC and eligible to receive FLT3-targeted therapy

experimental: Dose Validation/Expansion: Ziftomenib with 7+3+quizartinib in 1L NPM1-m/FLT3-ITD+ AML patients (C-1)

Ziftomenib with 7+3 and quizartinib in newly diagnosed NPM1-m and FLT3-ITD+ (with allelic ratio ≥0.05) AML patients who are candidates for IC and eligible to receive FLT3-targeted therapy

Interventions

Ziftomenib

Oral Administration

Venetoclax

Oral Administration

Azacitidine

Subcutaneous or Intravenous Administration

Daunorubicin

Intravenous Administration

Cytarabine

Intravenous Administration

Quizartinib

Oral Administration

Primary outcome measure

Rate of dose limiting toxicities (DLTs) per dose level (Part 1a only) [ Time Frame: During the first 28 days of ziftomenib in combination with SOC backbone treatment (1 cycle) ]
Descriptive statistics of adverse events [ Time Frame: From Cycle 1 Day 1 up to and including 28 days following the end of 36 months of treatment ]
Complete remission (CR) rate [ Time Frame: Until relapse, new anti-cancer therapy, death, or up to 36 months of treatment, whichever occurs first ]

Central Contacts and Locations

Central contacts

Kura Medical Information

844-KURAONC medinfo@kuraoncology.com

Locations

Mayo Clinic - Phoenix

Recruiting

Phoenix, Arizona, United States, 85054

Contacts

Clinical Trials Referral Office

855-776-0015

Moores UC San Diego Cancer Center

Recruiting

La Jolla, California, United States, 92093

Contacts

Krisma Montalvo

k1montalvo@health.ucsd.edu

USC / Norris Comprehensive Cancer Center

Recruiting

Los Angeles, California, United States, 90033

Contacts

Christine Duran

323-865-0371 duran_c@med.usc.edu

UCLA - Bowyer Oncology Center

Recruiting

Los Angeles, California, United States, 90095

Contacts

Bruck Habtemariam

310-794-0242 bhabtemariam@mednet.ucla.edu

UC Irvine Health Chao Family Comprehensive Cancer Center

Recruiting

Orange, California, United States, 92868

Contacts

Research Line

877-827-8839 ucstudy@hs.uci.edu

University of Colorado

Recruiting

Aurora, Colorado, United States, 80045

Colorado Blood Cancer Institute

Recruiting

Denver, Colorado, United States, 80218

Contacts

Patient Clinic

720-754-4800 PSLMDLCBCINewClinicPatient@HCAHealthcare.com

Yale Cancer Center and Smilow Cancer Hospital

Recruiting

New Haven, Connecticut, United States, 06510

Contacts

Farah Fasihuddin

203-737-3472 farah.fasihuddin@yale.edu

Mayo Clinic Jacksonville

Recruiting

Jacksonville, Florida, United States, 32224

Contacts

Clinical Trials Referral Office

855-776-0015

Emory Healthcare - The Emory Clinic

Recruiting

Atlanta, Georgia, United States, 30308

Contacts

Gigi Stoneback

gissela.blanco.stoneback@emory.edu

Georgia Cancer Center at Augusta University

Recruiting

Augusta, Georgia, United States, 30912

Contacts

Amanda Spires

amspires@augusta.edu

Robert H. Lurie Comprehensive Cancer Center of Northwestern University

Recruiting

Chicago, Illinois, United States, 60611

Contacts

312-695-9367 cancer@northwestern.edu

Loyola University Medical Center

Recruiting

Maywood, Illinois, United States, 60153

University of Iowa Hospitals & Clinics

Recruiting

Iowa City, Iowa, United States, 52242

Contacts

Cena Jones

cena-jones@uiowa.edu

The University of Kansas Medical Center Research Institute

Recruiting

Fairway, Kansas, United States, 66205

Contacts

Thania Medrano

913-945-7552 CTNurseNav@kumc.edu

University of Kentucky Markey Cancer Center

Recruiting

Louisville, Kentucky, United States, 40202

Contacts

Ashley Walton-Robbins

Ashley.Walton-Robbins@uky.edu

Norton Cancer Institute - St. Matthews

Recruiting

Louisville, Kentucky, United States, 40207

Contacts

502-899-3366 leukemia-NCIResearch@nortonhealthcare.org

Ochsner MD Anderson Cancer Center

Recruiting

Jefferson, Louisiana, United States, 70121

Contacts

Amanda Woolery, RN

504-842-0275 amanda.woolery@ochsner.org

Johns Hopkins School of Medicine

Recruiting

Baltimore, Maryland, United States, 21205

Contacts

I. Gojo

igojo1@jhmi.edu

Massachusetts General Hospital

Recruiting

Boston, Massachusetts, United States, 02114

Contacts

Christine Connelly

617-724-1124 cconnolly1@mgh.harvard.edu

UMass Chan Medical School

Recruiting

Worcester, Massachusetts, United States, 01655

Contacts

508-856-3216 Cancer.research@umassmed.edu

University of Michigan Comprehensive Cancer Center

Recruiting

Ann Arbor, Michigan, United States, 48109

Contacts

Cancer AnswerLine

800-865-1125 CancerAnswerLine@med.umich.edu

Karmanos Cancer Institute

Recruiting

Detroit, Michigan, United States, 48201

Contacts

Carmen Rush

800-527-6266 rushc@karmanos.org

University of Minnesota

Recruiting

Minneapolis, Minnesota, United States, 55455

Mayo Clinic - Rochester

Recruiting

Rochester, Minnesota, United States, 55905

Contacts

Clinical Trials Referral Office

855-776-0015

Hackensack University Medical Center

Recruiting

Hackensack, New Jersey, United States, 07601

Contacts

Oncology Clinical Research Referral Office

551-996-1777 OncologyResearchReferral@hmhn.org

Rutgers Cancer Institute

Recruiting

New Brunswick, New Jersey, United States, 08903

Contacts

Anita Trupiano

ar2069@cinj.rutgers.edu

Roswell Park Comprehensive Cancer Center

Recruiting

Buffalo, New York, United States, 14203

Contacts

877-275-7724 askrpci@roswellpark.org

New York - Presbyterian / Weill Cornell Medicine

Recruiting

New York, New York, United States, 10021

Contacts

Tania J Curcio

tjc9003@med.cornell.edu

Mount Sinai - Ruttenberg Treatment Center

Recruiting

New York, New York, United States, 10029

Contacts

Tina Czaplinska

tina.czaplinska@mssm.edu

Columbia University Medical Center

Recruiting

New York, New York, United States, 10032

Contacts

Research Nurse Navigator

212-342-5162 cancerclinicaltrials@cumc.columbia.edu

Stony Brook University Hospital

Recruiting

Stony Brook, New York, United States, 11794

Contacts

Pushpa Talanki

631-638-0815 Pushpa.Talanki@stonybrookmedicine.edu

Duke Blood Cancer Center

Recruiting

Durham, North Carolina, United States, 27705

Contacts

Quinna Lawson

quinna.marshburn@duke.edu

University Hospitals Cleveland Medical Center

Recruiting

Cleveland, Ohio, United States, 44106

Contacts

Sarah Gollwitzer

Sarah.Gollwitzer@UHhospitals.org

Cleveland Clinic Taussig Cancer Institute

Recruiting

Cleveland, Ohio, United States, 44195

Contacts

Cancer Answer Line

866-223-8100 ADVANIA@ccf.org

The James Cancer Hospital and Solove Research Institute

Recruiting

Columbus, Ohio, United States, 43210

Contacts

Hanna Cordes

Hanna.cordes@osumc.edu

OU Health Stephenson Cancer Center

Recruiting

Oklahoma City, Oklahoma, United States, 73104

Contacts

Cynthia Lowery

Cynthia-Lowery@ouhsc.edu

Hospital of the University of Pennsylvania

Recruiting

Philadelphia, Pennsylvania, United States, 19104

Contacts

Jessica Ritter

Jessica.Ritter@pennmedicine.upenn.edu

Camilla Ludlow

camila.ludlow@pennmedicine.upenn.edu

TriStar Bone Marrow Transplant

Recruiting

Nashville, Tennessee, United States, 37203

Contacts

Ask Sarah

844-482-4812

Sarah Cannon Research Institute - St. David's South Austin Medical Center / Texas Oncology South Austin

Recruiting

Austin, Texas, United States, 78704

Contacts

Amy Hammack

amy.hammack@sarahcannon.com

UT Southwestern - Simmons Cancer Center

Recruiting

Dallas, Texas, United States, 75235

Contacts

Phase I Team

214-648-7097 HemeTeam@groups.utsouthwestern.edu

MD Anderson Cancer Center

Recruiting

Houston, Texas, United States, 77030

Contacts

Ramya Ganesh

713-792-5640 rganesh@mdanderson.org

University of Wisconsin Hospital and Clinics

Recruiting

Madison, Wisconsin, United States, 53792

Contacts

UW Cancer Connect

800-622-8922 clinicaltrials@cancer.wisc.edu

Medical College of Wisconsin Cancer Center

Recruiting

Milwaukee, Wisconsin, United States, 53226

Contacts

Medical College of WI Cancer Center Clinical Trials Office

414-805-8900 cccto@mcw.edu

More Information

Sponsor

Kura Oncology, Inc.

Last update posted

Mar 13, 2026

Last verified

Mar, 2026

Keywords

Leukemia
Myeloid
AML
Hematological malignancy
KMT2A
NPM1
Menin
Acute Leukemia
Newly diagnosed AML
Untreated AML
venetoclax
cytarabine
daunorubicin
KMT2A-r
NPM1 mutation
Refractory AML
Acute Myeloid Leukemia, in Relapse
quizartinib

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-08. This information was provided to ClinicalTrials.gov by Kura Oncology, Inc. on 2026-03-13.