First in Human Study of Ziftomenib in Relapsed or Refractory Acute Myeloid Leukemia

A Phase 1/2 First in Human Study of the Menin-MLL(KMT2A) Inhibitor KO-539 in Patients With Relapsed or Refractory Acute Myeloid Leukemia

In this trial, ziftomenib, a menin-MLL(KMT2A) inhibitor, will be tested in patients for the first time. The trial includes a Main Study and four sub-studies. In the Main Study (including Phase 1a, Phase 1b, and Phase 2 portions), ziftomenib will be evaluated in patients with relapsed or refractory (R/R) acute myeloid leukemia (AML). The main study has completed enrollment.

In Sub-studies 1 and 2, the effects of taking ziftomenib and other common drugs at the same time will be investigated in AML patients. In Sub-study 3, ziftomenib will be evaluated in patients with R/R acute lymphoblastic leukemia (ALL). In Sub-study 4, ziftomenib will be evaluated in patients with R/R AML with certain genetic mutations.

Study Overview

• Status: Active, not recruiting
• Conditions: Acute Myeloid Leukemia; Acute Lymphoblastic Leukemia; Acute Leukemia of Ambiguous Lineage; Advanced Malignant Neoplasm; Mixed Phenotype Acute Leukemia; Mixed Lineage Leukemia; Mixed Lineage Acute Leukemia
• Intervention / Treatment: Drug: Midazolam; Drug: Ziftomenib; Drug: Itraconazole

Detailed Description

This first-in-human (FIH), open-label study will assess ziftomenib, a menin-MLL(KMT2A) inhibitor, in patients with relapsed or refractory (R/R) acute myeloid leukemia (AML). The trial includes a Main Study and four sub-studies.

The Main Study is a Phase 1/2 dose-escalation and dose-validation/expansion study to assess ziftomenib in patients with R/R AML. The dose-escalation part of the study (Phase 1a) will determine the maximum tolerated dose (MTD) and/or the recommended Phase 2 dose (RP2D). The dose-validation/expansion part of the study (Phase 1b) will determine the safety, tolerability, and minimal biologically effective dose (MBED) of ziftomenib in biomarker-specific dosing cohorts from among doses that demonstrated early biological activity and are determined to be safe in the dose-escalation phase. The Phase 2 portion of the study will determine the safety, tolerability, and anti-leukemia activity of ziftomenib in patients with nucleophosmin 1-mutant (NPM1-m) AML.

In Sub-study 1, the effects of co-administration of ziftomenib on the pharmacokinetics (PK) of midazolam will be studied in patients with R/R AML with certain genetic mutations.

In Sub-study 2, the effects of co-administration of itraconazole on the PK of ziftomenib will be studied in patients with R/R AML with certain genetic mutations.

In Sub-study 3, the safety, tolerability, and MBED/RP2D of ziftomenib will be studied in patients with R/R KMT2A-rearranged (KMT2A-r) ALL (Phase 1a dose escalation). These parameters will be investigated further for the RP2D in a Phase 1b dose-validation/cohort expansion part of the sub-study.

In Sub-study 4, the clinical activity of ziftomenib will be studied in patients with R/R AML with certain genetic mutations.

• Study Type: Interventional
• Enrollment (Estimated): 263
• Phase: Phase 2; Phase 1

Expanded Access

Available outside the clinical trial. See expanded access record.

Contacts and Locations

Study Locations

• Belgium
  • Roeselare, Belgium, 8800
    • AZ Delta - Campus Rumbeke
• Canada
  • Nova Scotia
    • Halifax, Nova Scotia, Canada, B3H 1V7
      • Queen Elizabeth II Health Sciences Centre
  • Quebec
    • Montreal, Quebec, Canada, H1T 2M4
      • Hôpital Maisonneuve-Rosemont
    • Québec, Quebec, Canada, G1J 1Z4
      • Hopital de l'Enfant-Jesus - Centre Integre en Cancerologie du CHU de Quebec - Universite Laval
• France
  • Lille, France, 59037
    • Centre Hospitalier Universitaire De Lille
  • Nantes, France, 44093
    • Centre Hospitalier Universitaire de Nantes
  • Paris, France, 75475
    • Hopital Saint Louis
  • Pessac, France, 33600
    • Magendie Hopital Haut-Leveque
  • Pierre-Bénite, France, 69310
    • Centre Hospitalier Lyon Sud
  • Villejuif, France, 94800
    • Institut Gustave Roussy
• Germany
  • Greifswald, Germany, 17475
    • University Medicine Greifswald
  • Hanover, Germany, 30625
    • Medizinische Hochsschule Hannover
• Italy
  • Bologna, Italy, 40138
    • Institute of Hematology and Medical Oncology "L. and A. Seragnoli"
  • Meldola, Italy, 47014
    • IRCCS Istituto Romagnolo per lo studio dei tumori "Dino Amadori"
  • Ravenna, Italy, 48121
    • UO Ematologia Ospedale di Ravenna
  • Roma, Italy
    • Institution Fondazione Policlinico Tor Vergata
• Spain
  • Barcelona, Spain, 08035
    • Hospital Universitari Vall d'Hebrón
  • Barcelona, Spain, 08035
    • Universitat de Barcelona
  • Madrid, Spain, 28033
    • MD Anderson Cancer Center
  • Madrid, Spain, 28050
    • Hospital Universitario HM Sanchinarro
  • Oviedo, Spain, 33011
    • Hospital Universitario Central de Asturias
  • Seville, Spain, 41013
    • Hospital Universitario Virgen del Rocío
  • Valencia, Spain, 46026
    • Hospital Universitari i Politècnic La Fe
• United States
  • Arizona
    • Gilbert, Arizona, United States, 85234
      • Banner MD Anderson Cancer Center
  • California
    • Los Angeles, California, United States, 90095
      • UCLA Ronald Reagan Medical Center
  • Florida
    • Jacksonville, Florida, United States, 32224
      • Mayo Clinic
  • Illinois
    • Chicago, Illinois, United States, 60611
      • Robert H. Lurie Comprehensive Cancer Center of Northwestern University
  • Maryland
    • Baltimore, Maryland, United States, 21201
      • University of Maryland Greenebaum Comprehensive Cancer Center
  • Massachusetts
    • Boston, Massachusetts, United States, 02114
      • Massachusetts General Hospital
  • Michigan
    • Ann Arbor, Michigan, United States, 48109
      • University of Michigan Hospitals
    • Detroit, Michigan, United States, 48201
      • Karmanos Cancer Institute
  • Minnesota
    • Rochester, Minnesota, United States, 55905
      • Mayo Clinic
  • New Jersey
    • Hackensack, New Jersey, United States, 07601
      • Hackensack University Medical Center - John Theurer Cancer Center
  • New York
    • Buffalo, New York, United States, 14203
      • Roswell Park Comprehensive Cancer Center
    • New York, New York, United States, 10029
      • The Mount Sinai Hospital
    • New York, New York, United States, 10021
      • Weill Cornell Medical College - NY Presbyterian Hospital
  • North Carolina
    • Durham, North Carolina, United States, 27710
      • Duke Cancer Institute
  • Oklahoma
    • Oklahoma City, Oklahoma, United States, 73117
      • Oklahoma University Health - Stephenson Cancer Center
  • Pennsylvania
    • Pittsburgh, Pennsylvania, United States, 15232
      • UPMC Hillman Cancer Center
  • Tennessee
    • Nashville, Tennessee, United States, 37232
      • Vanderbilt-Ingram Cancer Center
  • Texas
    • Dallas, Texas, United States, 75390
      • Harold C. Simmons Comprehensive Cancer Center - UT Southwestern Medical Center
    • Houston, Texas, United States, 77030
      • MD Anderson Cancer Center
  • Washington
    • Seattle, Washington, United States, 98109
      • Fred Hutchinson Cancer Research Center

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No

Description

Key Inclusion Criteria:

Patients with refractory or relapsed AML defined as the reappearance of ≥ 5% blasts in the bone marrow and who have also failed or are ineligible for any approved standard of care therapies, including HSCT.

1. Phase 1b:

   • Patients with a documented lysine[K]-specific methyltransferase 2-rearrangement (KMT2A-r), or
   • Patients with a documented nucleophosmin 1 mutation (NPM1-m)

2. Phase 2:

   • Patients with a documented nucleophosmin 1 mutation (NPM1-m)

3. Sub-studies:

   • Sub-studies 1 and 2: Patients with R/R AML with NPM1-m or other mutations associated with MEIS1 overexpression.
   • Sub-study 3: Patients with R/R Acute Lymphoblastic Leukemia (ALL) with KMT2A-r.
   • Sub-study 4: Patients with R/R AML with mutations associated with MEIS1 overexpression.
4. ≥ 18 years of age.
5. Eastern Cooperative Oncology Group (ECOG) performance status of 0 to 2, and a life expectancy of at least 2 months.
6. Adequate liver and kidney function according to protocol requirements.
7. Peripheral white blood cell (WBC) counts ≤ 30,000/μL. Patients may receive hydroxyurea to control and maintain white blood cell count prior to enrollment.
8. Women of childbearing potential must be willing to use a highly effective method of contraception throughout the study and for at least 187 days after the last dose of study treatment.
9. Males with female partners of childbearing potential must agree to use a highly effective method of contraception throughout the study and for at least 97 days after the last dose of study treatment.

Key Exclusion Criteria:

1. Diagnosis of acute promyelocytic leukemia.
2. Diagnosis of chronic myelogenous leukemia in blast crisis.
3. Donor lymphocyte infusion < 30 days prior to study entry.
4. Clinically active central nervous system (CNS) leukemia.
5. Undergone HSCT and have not had adequate hematologic recovery.
6. Receiving immunosuppressive therapy post HSCT within 2 weeks of Cycle 1 Day 1.
7. Grade ≥ 2 active graft-versus-host disease (GVHD), moderate or severe limited chronic GVHD, or extensive chronic GVHD of any severity.
8. Received chemotherapy immunotherapy, radiotherapy, or any ancillary therapy that is considered to be investigational (i.e., used for non-approved indications(s) and in the context of a research investigation) < 14 days prior to the first dose of ziftomenib or within 5 drug half-lives prior to the first dose of study drug.
9. Not recovered to < Grade 2 (National Cancer Institute Common Terminology Criteria for Adverse Events v5.0) from all acute toxicities or deemed back to a stable baseline.

10. Treatment with concomitant drugs that are strong inhibitors or inducers of cytochrome P450-isozyme 3A4 (CYP3A4), as follows:

    • Phase 1a, 1b, 2, and sub-studies 3 and 4: with the exception of antibiotics, antifungals, and antivirals that are used as standard of care or to prevent or treat infections and other such drugs that are considered absolutely essential for the care of the patient.
    • Sub-studies 1 and 2: No exceptions will be allowed except for the use of moderate CYP3A4 antifungal prophylaxis such as fluconazole or isavuconazole which is at steady state on Cycle 1 Day 1 and will continue through the completion of PKs on Cycle 1 Day 15 (for sub-study 1) or Cycle 1 Day 18 (for sub-study 2).
11. Detectable viral load for human immunodeficiency virus, hepatitis C, or hepatitis B surface antigen indicative of active infection. Patients with controlled disease will not be excluded from study enrollment.
12. Pre-existing disorder predisposing the patient to a serious or life-threatening infection (e.g. cystic fibrosis, congenital or acquired immunodeficiency, bleeding disorder, or cytopenias not related to AML).
13. Active uncontrolled acute or chronic systemic fungal, bacterial, viral, or other infection.
14. Significant cardiovascular disease including unstable angina pectoris, uncontrolled hypertension or arrhythmia, history of cerebrovascular accident including transient ischemic attack within the past 6 months, congestive heart failure (NYHA Class III or IV) related to primary cardiac disease, ischemic or severe valvular heart disease, or a myocardial infarction within 6 months prior to the first dose of study treatment.
15. Mean QTcF >480 ms on triplicate ECG.
16. Major surgery within 4 weeks prior to the first dose of study treatment.
17. Women who are pregnant or lactating. All female patients with reproductive potential must have a negative serum pregnancy test within 72 hours prior to starting treatment.
18. For sub-studies 1 and 2: Any intake of grapefruit, grapefruit juice, Seville oranges, Seville orange marmalade, or other products containing grapefruit or Seville oranges within 7 days of the first administration of ziftomenib until the end of Cycle 1.
19. For sub-studies 1 and 2: Moderate (Child-Pugh class B) or severe (Child-Pugh class C) hepatic impairment.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: None (Open Label)

Number of Arms

7

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Phase 1a - Dose Escalation; AML patients will receive multiple doses of ziftomenib	Drug: Ziftomenib; Oral administration; Other Names: KO-539
Experimental: Phase 1b - Dose-Validation Expansion; Cohort 1: KMT2A-r / NPM1-m R/R AML patients will receive ziftomenib Cohort 2: KMT2A-r / NPM1-m R/R AML patients will receive ziftomenib	Drug: Ziftomenib; Oral administration; Other Names: KO-539
Experimental: Phase 2; NPM1-m R/R AML patients will receive the recommended phase 2 ziftomenib dose	Drug: Ziftomenib; Oral administration; Other Names: KO-539
Experimental: Sub-study 1; R/R AML patients with mutations associated with MEIS1 overexpression will receive ziftomenib + midazolam	Drug: Midazolam; Oral administration; Other Names: Dormicum; Hypnovel; Seizalam; Drug: Ziftomenib; Oral administration; Other Names: KO-539
Experimental: Sub-study 2; R/R AML patients with mutations associated with MEIS1 overexpression will receive ziftomenib + itraconazole	Drug: Ziftomenib; Oral administration; Other Names: KO-539; Drug: Itraconazole; Oral administration; Other Names: Sporanox; Tolsura; Onmel
Experimental: Sub-study 3; Part 1a: KMT2A-r R/R ALL patients will receive multiple ziftomenib doses Part 1b: KMT2A-r R/R ALL patients will receive ziftomenib	Drug: Ziftomenib; Oral administration; Other Names: KO-539
Experimental: Sub-study 4; R/R AML patients with mutations associated with MEIS1 overexpression will receive ziftomenib	Drug: Ziftomenib; Oral administration; Other Names: KO-539

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Phase 1a: Maximum tolerated dose (MTD) and/or the recommended Phase 2 dose (RP2D)	MTD is defined as the highest dose that is not expected to cause dose limiting toxicity (DLT) in more than 20% of patients.	Dose Limiting Toxicities (DLTs) will be evaluated during the first 28 days (1 cycle)
Phase 1b: Number of patients who experience Adverse Events (AEs) and Serious Adverse Events (SAEs)	Assessed by NCI-CTCAE v5.0	During treatment and up to approximately 28 days after treatment discontinuation, or until immediately before the initiation of another anticancer therapy, whichever occurs first.
Phase 1b: Minimum biologically effective dose	Minimum biologically effective dose in dosing cohorts which have demonstrated biological activity and have been determined to be safe as a part of Part 1a	For at least 12 months following end of treatment
Phase 1a, 1b, and 2: Evidence of anti-leukemia activity	Assessed by the CR + CRh rate	For at least 12 months following end of treatment
Sub-study 1: Time to observed maximum plasma concentration (Tmax) of ziftomenib and midazolam	Tmax of ziftomenib, its metabolites, midazolam, and 1-hydroxymidazolam	Cycle 1 on Days 1 and 15 at predose and postdose
Sub-study 1: Area under the plasma concentration-time curve from time 0 to time t (AUC0-t) of ziftomenib and midazolam	AUC0-t of ziftomenib, its metabolites, midazolam, and 1-hydroxymidazolam	Cycle 1 on Days 1 and 15 at predose and postdose
Sub-study 1: Maximum observed plasma concentration (Cmax) of ziftomenib and midazolam	Cmax of ziftomenib, its metabolites, midazolam, and 1-hydroxymidazolam	Cycle 1 on Days 1 and 15 at predose and postdose
Sub-study 2: Time to observed maximum plasma concentration (Tmax) of ziftomenib and itraconazole	Tmax of ziftomenib, its metabolites, and itraconazole	Cycle 1 on Days 1, 15, and 22 at predose and postdose
Sub-study 2: Area under the plasma concentration-time curve from time 0 to time t (AUC0-t) of ziftomenib and itraconazole	AUC0-t of ziftomenib, its metabolites, and itraconazole	Cycle 1 on Days 1, 15, and 22 at predose and postdose
Sub-study 2: Maximum observed plasma concentration (Cmax) of ziftomenib and itraconazole	Cmax of ziftomenib, its metabolites, and itraconazole	Cycle 1 on Days 1, 15, and 22 at predose and postdose
Sub-study 3: Minimum biologically effective dose (MBED) and/or the recommended Phase 2 dose (RP2D)	Assessed by the number of patients that experience Adverse Events (AEs) and Serious Adverse Events (SAEs) per NCI-CTCAE v5.0	During treatment and up to approximately 28 days after treatment discontinuation, or until immediately before the initiation of another anticancer therapy, whichever occurs first
Sub-study 3: Minimum biologically effective dose (MBED) and/or the recommended Phase 2 dose (RP2D)	Assessed by CR	For at least 12 months following end of treatment
Sub-study 3: Change in Eastern Cooperative Oncology Group (ECOG) status	To assess the change in ECOG status	Timeframe: from Baseline to End of Treatment
Sub-study 3: Time to observed maximum plasma concentration (Tmax) of ziftomenib	Tmax of ziftomenib	Postdose on Cycle 1 Day 1 and Cycle 2 Day 1. Predose at Cycle 2 onwards
Sub-study 3: Area under the plasma concentration-time curve from time 0 to time t (AUC0-t) of ziftomenib	AUC0-t of ziftomenib	Postdose on Cycle 1 Day 1 and Cycle 2 Day 1. Predose at Cycle 2 onwards
Sub-study 3: Maximum observed plasma concentration (Cmax) of ziftomenib	Cmax of ziftomenib	Postdose on Cycle 1 Day 1 and Cycle 2 Day 1. Predose at Cycle 2 onwards.
Sub-study 4: Complete remission (CR) and complete remission with partial hematologic recovery (CRh)	To assess the CR+CRh rate	For at least 12 months following end of treatment

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Phase 1a: Tmax	Time to observed maximum plasma concentration of ziftomenib and/or its metabolites	Cycle 1 and Cycle 2. Each cycle is 28 days.
Phase 1a: Cmax	Maximum plasma concentration of ziftomenib and/or its metabolites	Cycle 1 and Cycle 2. Each cycle is 28 days.
Phase 1a and 2: Number of patients that experience Adverse Events (AEs) and Serious Adverse Events (SAEs)	Assessed by NCI-CTCAE v5.0	During treatment and up to approximately 28 days after treatment discontinuation, or until immediately before the initiation of another anticancer therapy, whichever occurs first.
Phase 1a: AUC(0-t)	Area under the plasma concentration-time curve from time 0 to time t of ziftomenib and/or its metabolites	Cycle 1 and Cycle 2. Each cycle is 28 days.
Phases 1a, 1b, and 2: Complete remission (CR) and complete remission with partial hematologic recovery (CRh) measurable residual disease (MRD) negativity	To assess the CR/CRh MRD negativity	For at least 12 months following discontinuation of treatment
Phases 1a, 1b, and 2: Duration of response (DOR)	To assess the DOR, defined as the duration of CR/CRh	For at least 12 months following discontinuation of treatment
Phases 1a, 1b, and 2: Transfusion independence (TI)	To assess transfusion independence	For at least 12 months following discontinuation of treatment
Phases 1a, 1b, and 2: Overall response rate (ORR)	To assess the ORR	For at least 12 months following discontinuation of treatment
Phases 1a, 1b, and 2: Event-free survival (EFS)	To assess event-free survival	For at least 12 months following end of treatment
Phases 1a, 1b, and 2: Overall survival (OS)	To assess overall survival	For at least 12 months following end of treatment
Phases 1a, 1b, and 2: Composite complete remission (CRc)	To assess CRc	For at least 12 months following discontinuation of treatment
Phases 1b and 2: Composite complete remission (CRc) measurable residual disease (MRD) negativity	To assess the CRc MRD negativity	For at least 12 months following discontinuation of treatment
Phases 1b and 2: Overall response rate (ORR) measurable residual disease (MRD) negativity	To assess the ORR MRD negativity	For at least 12 months following discontinuation of treatment
Sub-study 2: Corrected QT (QTc) intervals	Assessed by QTc intervals	During Cycle 1
Sub-study 3: Complete remission (CR) measurable residual disease (MRD) negativity	To assess the CR MRD negativity	For at least 12 months following discontinuation of treatment
Sub-studies 3 and 4: Composite complete remission (CRc)	To assess CRc	For at least 12 months following discontinuation of treatment
Sub-study 3: Duration of response (DOR)	To assess the DOR, defined as the duration of CR	For at least 12 months following discontinuation of treatment
Sub-studies 3 and 4: Overall survival (OS)	To assess overall survival	For at least 12 months following discontinuation of treatment
Sub-studies 3 and 4: Event-free survival (EFS)	To assess event-free survival	For at least 12 months following discontinuation of treatment
Sub-study 4: Transfusion independence (TI)	To assess transfusion independence	For at least 12 months following discontinuation of treatment
Sub-studies 3 and 4: Overall response rate (ORR) measurable residual disease (MRD) negativity	To assess the ORR MRD negativity	For at least 12 months following discontinuation of treatment
Sub-study 4: Number of patients that experience Adverse Events (AEs) and Serious Adverse Events (SAEs)	Assessed by NCI-CTCAE v5.0	During treatment and up to approximately 28 days after treatment discontinuation, or until immediately before the initiation of another anticancer therapy, whichever occurs first.
Sub-study 4: Duration of response (DOR)	To assess the DOR, defined as the duration of CR/CRh	For at least 12 months following discontinuation of treatment
Sub-study 4: Time to observed maximum plasma concentration (Tmax) of ziftomenib	Tmax of ziftomenib and its metabolites	Postdose on Cycle 1 Day 1 and Cycle 2 Day 1. Predose on Cycle 2 onwards
Sub-study 4: Area under the plasma concentration-time curve from time 0 to time t (AUC0-t) of ziftomenib	AUC0-t of ziftomenib and its metabolites	Postdose on Cycle 1 Day 1 and Cycle 2 Day 1. Predose on Cycle 2 onwards
Sub-study 4: Maximum plasma concentration (Cmax) of ziftomenib	Cmax of ziftomenib and its metabolites	Postdose on Cycle 1 Day 1 and Cycle 2 Day 1. Predose on Cycle 2 onwards

Collaborators and Investigators

• Sponsor: Kura Oncology, Inc.

Publications and helpful links

General Publications

• Jimenez JA, Apfelbaum AA, Hawkins AG, Svoboda LK, Kumar A, Ruiz RO, Garcia AX, Haarer E, Nwosu ZC, Bradin J, Purohit T, Chen D, Cierpicki T, Grembecka J, Lyssiotis CA, Lawlor ER. EWS-FLI1 and Menin Converge to Regulate ATF4 Activity in Ewing Sarcoma. Mol Cancer Res. 2021 Jul;19(7):1182-1195. doi: 10.1158/1541-7786.MCR-20-0679. Epub 2021 Mar 19.
• Sasca D, Guezguez B, Kuhn MWM. Next generation epigenetic modulators to target myeloid neoplasms. Curr Opin Hematol. 2021 Sep 1;28(5):356-363. doi: 10.1097/MOH.0000000000000673.
• Wang ES, Montesinos P, Foran J, Erba H, Rodriguez-Arboli E, Fedorov K, Heiblig M, Heidel FH, Altman JK, Baer MR, Ades L, Pettit K, Peterlin P, Papayannidis C, Berthon C, Walter RB, Shah MV, Balasubramanian S, Khawandanah M, Salamero Garcia O, Bergeron J, Madanat YF, Roboz GJ, Ulrickson M, Redner RL, McCloskey J, Pigneux A, de la Fuente Burguera A, Mitra A, Soifer HS, Tabachri M, Zhang Z, Riches M, Corum D, Leoni M, Issa GC, Fathi AT; KOMET-001. Ziftomenib in Relapsed or Refractory NPM1-Mutated AML. J Clin Oncol. 2025 Nov;43(31):3381-3390. doi: 10.1200/JCO-25-01694. Epub 2025 Sep 25.
• Wang ES, Issa GC, Erba HP, Altman JK, Montesinos P, DeBotton S, Walter RB, Pettit K, Savona MR, Shah MV, Kremyanskaya M, Baer MR, Foran JM, Schiller G, Ades L, Heiblig M, Berthon C, Peterlin P, Rodriguez-Arboli E, Salamero O, Patnaik MM, Papayannidis C, Grembecka J, Cierpicki T, Clegg B, Ray J, Linhares BM, Nie K, Mitra A, Ahsan JM, Tabachri M, Soifer HS, Corum D, Leoni M, Dale S, Fathi AT. Ziftomenib in relapsed or refractory acute myeloid leukaemia (KOMET-001): a multicentre, open-label, multi-cohort, phase 1 trial. Lancet Oncol. 2024 Oct;25(10):1310-1324. doi: 10.1016/S1470-2045(24)00386-3.

Study record dates

Study Major Dates

• Study Start (Actual): September 12, 2019
• Primary Completion (Estimated): October 16, 2028
• Study Completion (Estimated): October 16, 2028

Study Registration Dates

• First Submitted: June 25, 2019
• First Submitted That Met QC Criteria: August 22, 2019
• First Posted (Actual): August 26, 2019

Study Record Updates

• Last Update Posted (Actual): May 13, 2026
• Last Update Submitted That Met QC Criteria: May 12, 2026
• Last Verified: May 1, 2026

More Information

Terms related to this study

• Keywords: Acute Myeloid Leukemia; AML; ALL; Leukemia; Acute Lymphoblastic Leukemia; Acute Leukemia; Hematological malignancy; KMT2A; NPM1; Menin; MLL; MEIS1
• Additional Relevant MeSH Terms: Neoplasms by Site; Neoplasms; Immune System Diseases; Neoplasms by Histologic Type; Hematologic Diseases; Lymphatic Diseases; Lymphoproliferative Disorders; Immunoproliferative Disorders; Leukemia, Myeloid; Leukemia, Lymphoid; Hemic and Lymphatic Diseases; Leukemia; Leukemia, Myeloid, Acute; Hematologic Neoplasms; Precursor Cell Lymphoblastic Leukemia-Lymphoma; Leukemia, Biphenotypic, Acute; Heterocyclic Compounds, 1-Ring; Heterocyclic Compounds; Heterocyclic Compounds, 2-Ring; Heterocyclic Compounds, Fused-Ring; Azoles; Benzazepines; Benzodiazepines; Triazoles; Piperazines; Midazolam; Itraconazole
• Other Study ID Numbers: KO-MEN-001

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: UNDECIDED

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No