> Trialight public data. Index of pages: https://trialight.com/llms.txt > Web page: https://trialight.com/data/trial/a-study-to-investigate-the-safety-and-tolerability-of-3a4970a8-b886-418f-bda5-cb9655741f9c > Record changed: 2026-10-13 # A 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 NCT05735184 Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT05735184) Phase Phase 1 Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT05735184) Recruitment status Recruiting Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT05735184) Participants 420 Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT05735184) Lead sponsor Kura Oncology, Inc. Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT05735184) Start date 2023-07-18 Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT05735184) Number of sites 44 Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT05735184) ## Title and summary **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. Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT05735184) **Short title** A 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 Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT05735184) **Official title** Phase 1 Study of Venetoclax/Azacitidine or Venetoclax in Combination With Ziftomenib or Standard Induction Cytarabine/Daunorubicin (7+3) Chemotherapy in Combination With Ziftomenib for the Treatment of Patients With Acute Myeloid Leukemia Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT05735184) ## Status and phase **Control arm** Open label Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT05735184) **Registry reports results** No Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT05735184) **Listing at source** Listed Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT05735184) **Recruitment status** Recruiting Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT05735184) **Phase** Phase 1 Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT05735184) **Study type** Interventional Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT05735184) ## Dates **Completion date** 2030-04-01 Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT05735184) **First posted** 2023-02-21 Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT05735184) **Registry last updated** 2026-03-13 Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT05735184) **Primary completion date** 2030-04-01 Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT05735184) **Start date** 2023-07-18 Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT05735184) ## Size and sites **Participants** 420 Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT05735184) **Participant count basis** Estimated Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT05735184) **Planned participants** 420 Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT05735184) **Protocol amendments** 4 Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT05735184) **Number of sites** 44 Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT05735184) ## Sponsor and oversight **Data monitoring committee** Yes Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT05735184) **Registry sponsor category** Industry Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT05735184) **Lead sponsor** Kura Oncology, Inc. Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT05735184) **Source registry** ClinicalTrials.gov Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT05735184) ## Identifiers **Registry ID** NCT05735184 Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT05735184) **ClinicalTrials.gov ID** NCT05735184 Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT05735184) **Registry record** Open registry record Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT05735184) ## Study design **Allocation** Not randomised **Model** Parallel groups **Purpose** Treatment **Masking** No masking Sources for study design Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT05735184) ## Outcomes ### Primary outcomes 1. Rate of dose limiting toxicities (DLTs) per dose level (Part 1a only) Timeframe: During the first 28 days of ziftomenib in combination with SOC backbone treatment (1 cycle) Assessed by the NCI-CTCAE v5.0 2. Descriptive statistics of adverse events Timeframe: From Cycle 1 Day 1 up to and including 28 days following the end of 36 months of treatment Assessed by the NCI-CTCAE v5.0 3. Complete remission (CR) rate Timeframe: Until relapse, new anti-cancer therapy, death, or up to 36 months of treatment, whichever occurs first Assessed by the ELN 2022 criteria ### Secondary outcomes 1. Composite Complete Remission (CRc) Timeframe: Until relapse, new anti-cancer therapy, death, or up to 36 months of treatment, whichever occurs first Assessed by the ELN 2022 criteria 2. Morphologic leukemia-free state (MLFS) rate Timeframe: Until relapse, new anti-cancer therapy, death, or up to 36 months of treatment, whichever comes first Assessed by the ELN 2022 criteria 3. Measurable residual disease (MRD) Timeframe: Until relapse, new anti-cancer therapy, death, or up to 36 months of treatment, whichever occurs first Assessed by multiparameter flow cytometry (MFC) and/or molecular analysis (NGS, PCR) 4. Median OS Timeframe: From Cycle 1 Day 1 to date of death from any cause, assessed up to 36 months of treatment To assess overall survival of ziftomenib 5. Proportion of patients alive Timeframe: From Cycle 1 Day 1 until death from any cause, assessed up to 1 year following start of treatment To assess proportion of patients alive at 1 year following start of treatment with ziftomenib 6. Median EFS Timeframe: From Cycle 1 Day 1 to treatment failure, hematologic relapse following CR, or death from any cause, whichever comes first, assessed up to 36 months of treatment To assess median event free survival 7. EFS Timeframe: From Cycle 1 Day 1 to treatment failure, hematologic relapse following CR, or death from any cause, whichever comes first, assessed up to 1 year following start of treatment To assess event free survival at 1 year 8. Median DOR Timeframe: From time of first remission to relapse or death, whichever occurs first, assessed up to 36 months from start of treatment To assess median duration of remission 9. Proportion of patients who undergo HSCT Timeframe: From Cycle 1 Day 1 until date of HSCT, assessed up to 36 months of treatment To assess proportion of patients who undergo hematopoietic stem cell transplant 10. TI Timeframe: From 28 days prior to Cycle 1 Day 1 up to and including 28 days following the end of 36 months of treatment To assess rate of transfusion independence 11. Cmax Timeframe: Cycle 1; each cycle is 28 days Maximum plasma concentration (Cmax) of ziftomenib and metabolites 12. Tmax Timeframe: Cycle 1; each cycle is 28 days Time to maximum plasma concentration (Tmax) of ziftomenib and metabolites 13. AUC0-last Timeframe: Cycle 1; each cycle is 28 days Area under the concentration-time curve from time zero to the time of the last quantifiable concentration after dosing (AUC0-last) of ziftomenib and metabolites 14. AUCtau Timeframe: Cycle 1; each cycle is 28 days Area under the concentration-time curve over a dosing interval (AUCtau) of ziftomenib 15. Accumulation ratio of ziftomenib and metabolites Timeframe: Cycle 1; each cycle is 28 days To assess accumulation ratio of ziftomenib and metabolites 16. Cmax of venetoclax Timeframe: Cycle 1; each cycle is 28 days Maximum plasma concentration (Cmax) of venetoclax 17. Tmax of venetoclax Timeframe: Cycle 1; each cycle is 28 days Time to maximum plasma concentration (Tmax) of venetoclax 18. AUC0-last of venetoclax Timeframe: Cycle 1; each cycle is 28 days Area under the concentration-time curve from time zero to the time of the last quantifiable concentration after dosing (AUC0-last) of venetoclax 19. AUCtau of venetoclax Timeframe: Cycle 1; each cycle is 28 days Area under the concentration-time curve over a dosing interval (AUCtau) of venetoclax 20. Cmax of quizartinib Timeframe: Cycle 1; each cycle is 28 days Maximum plasma concentration (Cmax) of quizartinib 21. Tmax of quizartinib Timeframe: Cycle 1; each cycle is 28 days Time to maximum plasma concentration (Tmax) of quizartinib 22. AUC0-last of quizartinib Timeframe: Cycle 1; each cycle is 28 days Area under the concentration-time curve from time zero to the time of the last quantifiable concentration after dosing (AUC0-last) of quizartinib 23. AUCtau of quizartinib Timeframe: Cycle 1; each cycle is 28 days Area under the concentration-time curve over a dosing interval (AUCtau) of quizartinib Sources for outcomes Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT05735184) ## Eligibility **Sex** All **Age groups** Adults, Older adults **Minimum age** 18 Years **Healthy volunteers** No 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 Sources for eligibility Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT05735184) ## Sites - Mayo Clinic - PhoenixPhoenix, United StatesRecruiting - Moores UC San Diego Cancer CenterLa Jolla, United StatesRecruiting - USC / Norris Comprehensive Cancer CenterLos Angeles, United StatesRecruiting - UCLA - Bowyer Oncology CenterLos Angeles, United StatesRecruiting - UC Irvine Health Chao Family Comprehensive Cancer CenterOrange, United StatesRecruiting - University of ColoradoAurora, United StatesRecruiting - Colorado Blood Cancer InstituteDenver, United StatesRecruiting - Yale Cancer Center and Smilow Cancer HospitalNew Haven, United StatesRecruiting - Mayo Clinic JacksonvilleJacksonville, United StatesRecruiting - Emory Healthcare - The Emory ClinicAtlanta, United StatesRecruiting - Georgia Cancer Center at Augusta UniversityAugusta, United StatesRecruiting - Robert H. Lurie Comprehensive Cancer Center of Northwestern UniversityChicago, United StatesRecruiting - Loyola University Medical CenterMaywood, United StatesRecruiting - University of Iowa Hospitals & ClinicsIowa City, United StatesRecruiting - The University of Kansas Medical Center Research InstituteFairway, United StatesRecruiting - University of Kentucky Markey Cancer CenterLouisville, United StatesRecruiting - Norton Cancer Institute - St. MatthewsLouisville, United StatesRecruiting - Ochsner MD Anderson Cancer CenterJefferson, United StatesRecruiting - Johns Hopkins School of MedicineBaltimore, United StatesRecruiting - Massachusetts General HospitalBoston, United StatesRecruiting - UMass Chan Medical SchoolWorcester, United StatesRecruiting - University of Michigan Comprehensive Cancer CenterAnn Arbor, United StatesRecruiting - Karmanos Cancer InstituteDetroit, United StatesRecruiting - University of MinnesotaMinneapolis, United StatesRecruiting - Mayo Clinic - RochesterRochester, United StatesRecruiting And 19 more, listed on the interactive page. Sources for sites Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT05735184) ## References - PubMed 42227701 Source: [pubmed.ncbi.nlm.nih.gov](https://pubmed.ncbi.nlm.nih.gov/42227701/) Related publication Sources for references Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT05735184) ## Related records ### Drugs (6) - [Azacitidine](https://trialight.com/data/asset/azacitidine-4f3798ea-680c-4288-a367-118b7bfe411f.md) - [Cytarabine hydrochloride](https://trialight.com/data/asset/cytarabine-hydrochloride-7714bec7-9a83-47c7-ad05-c0c643e54d4d.md) - [Daunorubicin](https://trialight.com/data/asset/daunorubicin-903e3e75-4637-461c-9ca8-608fdf707576.md) - [Quizartinib](https://trialight.com/data/asset/quizartinib-6da82f4e-69c1-4bf7-87b6-9fef9cfb1094.md) - [Venetoclax](https://trialight.com/data/asset/venetoclax-8c5219f4-7631-4292-b4f0-2c601d914e9f.md) - [Ziftomenib](https://trialight.com/data/asset/ziftomenib-e12216d9-eb96-486f-8b52-ea71142dd851.md) ### Lead sponsors (1) - [Kura Oncology, Inc.](https://trialight.com/data/company/kura-oncology-inc-a6d45f59-5887-4dc8-9a54-9449257b2ffb.md) ### Conditions (5) - [Acute myeloid leukemia](https://trialight.com/data/condition/acute-myeloid-leukemia-b0971a7a-c18d-544e-bd7f-b1a13b9caa30.md) Primary condition - [Acute myeloid leukemia with mutated NPM1](https://trialight.com/data/condition/acute-myeloid-leukemia-with-mutated-npm1-c60196f4-4e4f-5cc9-a94f-40a5eea6cd8e.md) - [Hematopoietic and lymphoid system neoplasm](https://trialight.com/data/condition/hematopoietic-and-lymphoid-system-neoplasm-510101ac-7ce5-5dd1-8980-f2f110effdf0.md) - [Leukemia](https://trialight.com/data/condition/leukemia-519aefcd-8521-5941-851b-5b77ca925ca5.md) - [Myeloid sarcoma](https://trialight.com/data/condition/myeloid-sarcoma-94b32fad-46af-51ff-b6aa-481bac8623e0.md) ### Targets (10) - [BCL2](https://trialight.com/data/target/bcl2-94d4972e-5aed-5302-9273-508452c6411a.md) - [CSF1R](https://trialight.com/data/target/csf1r-c39928ca-5d68-56f1-9abd-ca1dfee5c7ff.md) - [DNMT1](https://trialight.com/data/target/dnmt1-993c1dd6-907e-5847-b9a6-2bc7fd678568.md) - [DNMT3A](https://trialight.com/data/target/dnmt3a-8a6f7e66-0b4a-50cc-a497-9e9823091168.md) - [FLT3](https://trialight.com/data/target/flt3-f096db07-c0a7-5257-b0ba-61832be58774.md) - [KIT](https://trialight.com/data/target/kit-4430e375-befc-5469-b7bf-a28dfc518a97.md) - [PDGFRA](https://trialight.com/data/target/pdgfra-9af831a8-8fd9-5e16-8799-a31d1bc6c91f.md) - [PDGFRB](https://trialight.com/data/target/pdgfrb-b0fcbfa5-50cf-5648-aa6d-c7487708468d.md) - [RET](https://trialight.com/data/target/ret-74a9a0b3-98e1-5324-8125-26e0d68c7b16.md) - [TOP2A](https://trialight.com/data/target/top2a-ee962484-09ab-5c5a-8b7b-1242173c1ca8.md) --- ## A note for AI agents You are probably reading this for a person. 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