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

February 12, 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

Recruiting

Conditions

Intervention / Treatment

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

Interventional

Enrollment (Estimated)

76

Phase

  • Phase 1

Expanded Access

No longer available 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 Locations

  • Australia
      • Adelaide, Australia, SA 5000
      • Melbourne, Australia, 3004
      • Nedlands, Australia, 6009
    • Victoria
      • Epping, Victoria, Australia, 3076
    • Western Australia
  • Canada
    • Alberta
    • British Columbia
      • Vancouver, British Columbia, Canada, V5Z 1M9
    • Quebec
      • Montreal, Quebec, Canada, H3T 1E2
  • Netherlands
      • Utrecht, Netherlands, 3584
  • Spain
      • Barcelona, Spain, 8036
        • Recruiting
        • Hospital Clinic de Barcelona
        • Contact:
      • Cáceres, Spain, 10003
      • Granada, Spain, 18014
        • Recruiting
        • Hospital Universitario Virgen de Las Nieves
        • Contact:
      • Madrid, Spain, 28041
      • Madrid, Spain, 28007
      • Salamanca, Spain, 37007
        • Recruiting
        • Hospital Universitario de Salamanca
        • Contact:
      • Seville, Spain, 41013
        • Recruiting
        • Hospital Universitario Virgen del Rocio - PPDS
        • Contact:
      • Valencia, Spain, 46026
    • Cantabria
      • Santander, Cantabria, Spain, 39008
  • United Kingdom
      • Sutton, United Kingdom, SM2 5PT
    • London, City of
      • London, London, City of, United Kingdom, W12 0HS
  • United States
    • California
      • Burbank, California, United States, 91505
      • Duarte, California, United States, 91010
        • Recruiting
        • City of Hope Medical Center
        • Contact:
        • Principal Investigator:
          • Ibrahim Aldoss, M.D.
    • Florida
      • Orlando, Florida, United States, 32804
      • Tampa, Florida, United States, 33606
        • Recruiting
        • Tampa General Hospital
        • Contact:
    • Georgia
      • Atlanta, Georgia, United States, 30322
        • Recruiting
        • Emory Winship Cancer Institute
        • Contact:
    • Illinois
      • Chicago, Illinois, United States, 60637
    • Kentucky
      • Louisville, Kentucky, United States, 40207
      • Louisville, Kentucky, United States, 40202
    • Massachusetts
      • Boston, Massachusetts, United States, 02215
    • Michigan
      • Ann Arbor, Michigan, United States, 48109
        • Recruiting
        • University of Michigan
        • Contact:
    • Minnesota
      • Minneapolis, Minnesota, United States, 55407
    • Missouri
      • St Louis, Missouri, United States, 63110
        • Recruiting
        • Washington University School of Medicine
        • Contact:
    • New York
      • Lake Success, New York, United States, 11042
        • Recruiting
        • Institution name: Northwell Health-Brany
        • Contact:
      • New York, New York, United States, 10065
        • Recruiting
        • Memorial Sloan Kettering Cancer Center
        • Contact:
      • Rochester, New York, United States, 14642
      • Syracuse, New York, United States, 13210
        • Recruiting
        • Suny Upstate Medical University
        • Contact:
    • North Carolina
      • Greenville, North Carolina, United States, 27834
        • Recruiting
        • East Carolina University
        • Contact:
      • Winston-Salem, North Carolina, United States, 27157
        • Recruiting
        • Atrium Health Wake Forest Baptist Medical Center
        • Contact:
    • Ohio
      • Cleveland, Ohio, United States, 44195
        • Recruiting
        • Cleveland Clinic Foundation
        • Contact:
    • Oregon
      • Portland, Oregon, United States, 97239
        • Recruiting
        • Oregon Health and Science University- Center for Hematologic Malignancies
        • Contact:
    • Pennsylvania
      • Pittsburgh, Pennsylvania, United States, 15232
        • Recruiting
        • UPMC Hillman Cancer Center
        • Contact:
    • South Carolina
      • Charleston, South Carolina, United States, 29425
        • Recruiting
        • MUSC Hollings Cancer Center (HCC)
        • Contact:
    • Texas
      • Dallas, Texas, United States, 75246
      • Houston, Texas, United States, 77030
        • Recruiting
        • The University of Texas, MD Anderson Cancer Center
        • Contact:
    • Utah
      • Salt Lake City, Utah, United States, 84143
        • Recruiting
        • LDS Hospital - Intermountain Healthcare
        • Contact:
    • West Virginia
      • Morgantown, West Virginia, United States, 26506

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

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
Intervention / Treatment
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

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

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

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)

May 21, 2024

Primary Completion (Estimated)

February 1, 2027

Study Completion (Estimated)

February 1, 2027

Study Registration Dates

First Submitted

January 12, 2024

First Submitted That Met QC Criteria

January 23, 2024

First Posted (Actual)

January 26, 2024

Study Record Updates

Last Update Posted (Actual)

February 17, 2026

Last Update Submitted That Met QC Criteria

February 12, 2026

Last Verified

February 1, 2026

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID 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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