A Study of SNDX-5613 in Combination With Intensive Chemotherapy in Participants With Acute Myeloid Leukemias

June 29, 2026 updated by: Syndax Pharmaceuticals

A Phase 1, Open-label, Dose-escalation, and Dose-expansion Study to Evaluate Safety, Tolerability, and Clinical Activity of SNDX-5613 in Combination With Intensive Chemotherapy in Participants With Newly Diagnosed Acute Myeloid Leukemias Harboring Alterations in Lysine-specific Methyltransferase 2A (KMT2A/MLL), Nucleophosmin 1 (NPM1), and Nucleoporin 98 (NUP98) Genes

The purpose of this study is to evaluate the safety, tolerability, pharmacokinetics, and clinical activity of SNDX-5613 in combination with intensive chemotherapy in participants with newly diagnosed acute myeloid leukemia (AML) harboring alterations in KMT2A, NPM1, or NUP98 genes.

Study Overview

Status

Status

Indicates the current recruitment status or the expanded access status.
Active, not recruiting

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

Detailed Description

The Dose Escalation portion of this study will identify the maximum tolerated dose, or if different, the recommended Phase 2 dose of SNDX-5613 to be used in combination with intensive chemotherapy and in maintenance monotherapy following intensive chemotherapy in participants with newly diagnosed AML harboring alterations in KMT2A, NPM1, or NUP98 genes.

In the Dose Expansion portion of the study, safety and preliminary efficacy of SNDX-5613 may be explored in expansion cohorts at tolerated dose levels.

In both Dose Escalation and Dose Expansion, the treatment period will consist of an induction phase (up to 2 cycles), a consolidation phase (up to 4 cycles and could include hematopoietic stem cell transplant for participants who are transplant eligible and have an available donor), and a maintenance monotherapy phase with SNDX-5613. The cycle duration will be 28 days.

Study Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Interventional

Enrollment (Actual)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
76

Phase

Phase

The stage of a clinical trial studying a drug or biological product, based on definitions developed by the U.S. Food and Drug Administration (FDA). The phase is based on the study's objective, the number of participants, and other characteristics. There are five phases: Early Phase 1 (formerly listed as Phase 0), Phase 1, Phase 2, Phase 3, and Phase 4. Not Applicable is used to describe trials without FDA-defined phases, including trials of devices or behavioral interventions.
  • Phase 1

Expanded Access

Expanded Access

A way for patients with serious diseases or conditions who cannot participate in a clinical trial to gain access to a medical product that has not been approved by the U.S. Food and Drug Administration (FDA). Also called compassionate use. There are different expanded access types.
No longer available

No longer available

  • Available: Expanded access is currently available for this investigational treatment, and patients who are not participants in the clinical study may be able to gain access to the drug, biologic, or medical device being studied.
  • No longer available: Expanded access was available for this intervention previously but is not currently available and will not be available in the future.
  • Temporarily not available: Expanded access is not currently available for this intervention but is expected to be available in the future.
  • Approved for marketing: The intervention has been approved by the U.S. Food and Drug Administration for use by the public.
 outside the clinical trial. See expanded access record.

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Contact

The name and contact information for the person who can answer enrollment questions for a clinical study. Each location where the study is being conducted may also have a specific contact, who may be better able to answer those questions.

Study Locations

  • Australia
      • Adelaide, Australia, SA 5000
        • Royal Adelaide Hospital
      • Melbourne, Australia, 3004
        • The Alfred Hospital, Victoria
      • Nedlands, Australia, 6009
        • Sir Charles Gairdner Hospital
    • Victoria
      • Epping, Victoria, Australia, 3076
        • Northern Hospital, Victoria
    • Western Australia
      • Perth, Western Australia, Australia, 6000
        • Royal Perth Hospital
  • Canada
    • Alberta
      • Edmonton, Alberta, Canada, T6G 2B7
        • University of Alberta Hospital
    • British Columbia
      • Vancouver, British Columbia, Canada, V5Z 1M9
        • Gordon and Leslie Diamond Health Care Center
    • Quebec
      • Montreal, Quebec, Canada, H3T 1E2
        • The Sir Mortimer B. Davis Jewish General Hospital
  • Netherlands
      • Utrecht, Netherlands, 3584
        • University Medical Center Utrecht
  • Spain
      • Barcelona, Spain, 8036
        • Hospital Clinic de Barcelona
      • Cáceres, Spain, 10003
        • Hospital San Pedro de Alcántara
      • Granada, Spain, 18014
        • Hospital Universitario Virgen De Las Nieves
      • Madrid, Spain, 28041
        • Hospital Universitario 12 De Octubre
      • Madrid, Spain, 28007
        • Hospital General Universitario Gregorio Maranon
      • Salamanca, Spain, 37007
        • Hospital Universitario de Salamanca
      • Seville, Spain, 41013
        • Hospital Universitario Virgen del Rocio - PPDS
      • Valencia, Spain, 46026
        • Universitat de València
    • Cantabria
      • Santander, Cantabria, Spain, 39008
        • Hospital Universitario Marques de Valdecilla
  • United Kingdom
      • Sutton, United Kingdom, SM2 5PT
        • The Royal Marsden NHS
    • London, City of
      • London, London, City of, United Kingdom, W12 0HS
        • Hammersmith Hospital
  • United States
    • Arizona
      • Goodyear, Arizona, United States, 85338
        • City Of Hope
    • California
      • Burbank, California, United States, 91505
        • UCLA Medical Hematology
      • Duarte, California, United States, 91010
        • City of Hope Medical Center
    • Florida
      • Orlando, Florida, United States, 32804
        • AdventHealth Blood & Marrow Transplant Center
      • Tampa, Florida, United States, 33606
        • Tampa General Hospital
    • Georgia
      • Atlanta, Georgia, United States, 30322
        • Emory Winship Cancer Institute
    • Illinois
      • Chicago, Illinois, United States, 60637
        • University of Chicago
      • Chicago, Illinois, United States, 60612
        • University of Illinois
    • Kentucky
      • Louisville, Kentucky, United States, 40207
        • Norton Cancer Institute, St. Matthews Campus
      • Louisville, Kentucky, United States, 40202
        • University of Louisville Health Brown Cancer Center
    • Massachusetts
      • Boston, Massachusetts, United States, 02215
        • Beth Israel Deaconess Medical Center
    • Michigan
      • Ann Arbor, Michigan, United States, 48109
        • University of Michigan
    • Minnesota
      • Minneapolis, Minnesota, United States, 55407
        • Allina Health Cancer Institute
    • Missouri
      • St Louis, Missouri, United States, 63110
        • Washington University School of Medicine
    • New York
      • Lake Success, New York, United States, 11042
        • Institution name: Northwell Health-Brany
      • New York, New York, United States, 10065
        • Memorial Sloan Kettering Cancer Center
      • Rochester, New York, United States, 14642
        • University of Rochester Medical Center
      • Syracuse, New York, United States, 13210
        • SUNY Upstate Medical University
    • North Carolina
      • Greenville, North Carolina, United States, 27834
        • East Carolina University
      • Winston-Salem, North Carolina, United States, 27157
        • Atrium Health Wake Forest Baptist Medical Center
    • Ohio
      • Cleveland, Ohio, United States, 44195
        • Cleveland Clinic Foundation
    • Oregon
      • Portland, Oregon, United States, 97239
        • Oregon Health and Science University- Center for Hematologic Malignancies
    • Pennsylvania
      • Pittsburgh, Pennsylvania, United States, 15232
        • UPMC Hillman Cancer Center
    • South Carolina
      • Charleston, South Carolina, United States, 29425
        • MUSC Hollings Cancer Center (HCC)
    • Texas
      • Dallas, Texas, United States, 75246
        • Baylor University Medical Center
      • Houston, Texas, United States, 77030
        • The University of Texas, MD Anderson Cancer Center
    • Utah
      • Salt Lake City, Utah, United States, 84143
        • LDS Hospital - Intermountain Healthcare
    • West Virginia
      • Morgantown, West Virginia, United States, 26506
        • West Virginia University

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

Ages Eligible for Study

  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Description

Inclusion Criteria:

  • Established, pathologically confirmed diagnosis of AML by World Health Organization 2022 criteria.
  • Previously untreated AML and eligible to receive intensive chemotherapy.
  • KMT2Ar, NPM1c, or NUP98r mutations identified by local laboratory prior to the first dose of SNDX-5613.
  • Eastern Cooperative Oncology Group performance status ≤2 and ≤1 if >65 years old .
  • Adequate liver, kidney, and cardiac function.

Exclusion Criteria:

  • Diagnosis of acute promyelocytic leukemia.
  • Clinically active central nervous system leukemia (blasts detected in cerebrospinal fluid, radiographic or clinical signs and symptoms).
  • Fridericia's corrected QT interval (QTcF) >450 milliseconds (average of triplicate), diagnosis or suspicion of Long QT syndrome or family history of Long QT syndrome.
  • Any gastrointestinal issue of the upper gastrointestinal tract that might affect oral drug absorption or ingestion.
  • Cirrhosis with a Child-Pugh score of B or C.
  • Any of the following within the 6 months prior to study entry: myocardial infarction, uncontrolled/unstable angina, congestive heart failure (New York Heart Association Classification Class ≥II), life-threatening, uncontrolled arrhythmia, cerebrovascular accident, or transient ischemic attack.
  • Hepatitis B, Hepatitis C, or HIV-positive with detectable viral load.
  • Documented active, uncontrolled infection.
  • Uncontrolled disseminated intravascular coagulation.
  • Lactating/breast feeding or pregnant.
  • Use of prohibited concomitant chemotherapy, radiation therapy, or immunotherapy.
  • Use of strong CYP3A4 inducers or inhibitors (except for Itraconazole, Ketoconazole, Posaconazole, or Voriconazole).

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Sequential Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm

Participant Group / Arm

A group or subgroup of participants in a clinical trial that receives a specific intervention/treatment, or no intervention, according to the trial's protocol.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
Experimental: SNDX-5613

Dose Escalation:

  • Induction: Sequential cohorts of escalating dose levels of SNDX-5613 with chemotherapy regimen.
  • Consolidation: Cohorts will receive high-dose cytarabine (HiDAC) chemotherapy followed by SNDX-5613.
  • Maintenance Monotherapy: Cohorts will receive SNDX-5613.

Dose Expansion:

  • Induction: SNDX-5613 at tolerated dose level with chemotherapy regimen.
  • Consolidation: Cohorts will receive SNDX-5613 with chemotherapy regimen and HiDAC.
  • Maintenance Monotherapy: Cohorts will receive SNDX-5613.
Drug: SNDX-5613
Participants will receive SNDX-5613 orally during Induction, Consolidation, and Maintenance until meeting criteria for discontinuation.
Drug: Chemotherapy Regimen
Induction: Participants will receive an intravenous (IV), 2-drug combination of cytarabine and either daunorubicin or idarubicin.
Drug: HiDAC
Consolidation: Participants will receive HiDAC IV.

What is the study measuring?

Primary Outcome Measures

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
Outcome Measure
Time Frame
Dose Escalation: Number of Participants with Dose-limiting Toxicities
Time Frame: Up to Day 42
Up to Day 42
Number of Participants with Treatment-emergent Adverse Events (TEAEs)
Time Frame: Day 1 through 30 days after final dose (up to approximately 3 years)
Day 1 through 30 days after final dose (up to approximately 3 years)

Secondary Outcome Measures

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
Outcome Measure
Time Frame
Maximum Plasma Concentration (Cmax) of SNDX-5613 and Relevant Metabolites
Time Frame: Predose through Day 15
Predose through Day 15
Area Under the Plasma Concentration Versus Time Curve From Time 0 to t (AUC0-t) of SNDX-5613 and Relevant Metabolites
Time Frame: Predose through Day 15
Predose through Day 15

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
Syndax Pharmaceuticals

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
May 21, 2024

Primary Completion (Estimated)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
February 1, 2027

Study Completion (Estimated)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
February 1, 2027

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
January 12, 2024

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
January 23, 2024

First Posted (Actual)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
January 26, 2024

Study Record Updates

Last Update Posted (Actual)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
June 30, 2026

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
June 29, 2026

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
June 1, 2026

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant numbers.
  • SNDX-5613-0708
  • 2024-514531-18-00 (Ctis)

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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