Acute Myeloid LeukemiasPhase 1NCT06226571
A Study of Revumenib in Combination With Intensive Chemotherapy in Participants With Acute Myeloid Leukemias
A Phase 1 study testing Revumenib, Chemotherapy Regimen and HiDAC in people with Acute Myeloid Leukemias, run by Syndax Pharmaceuticals at 48 sites in 6 countries.
Trial summary
The purpose of this study is to evaluate the safety, tolerability, pharmacokinetics, and clinical activity of revumenib in combination with intensive chemotherapy in participants with newly diagnosed acute myeloid leukemia (AML) harboring alterations in KMT2A, NPM1, or NUP98 genes. Read more about this study
- Who is it for?
- Ages 18 to 75 years
- Conditions the study is for
- Acute Myeloid Leukemias
- What is being tested?
- Revumenib, Chemotherapy Regimen and HiDAC
- Type of treatment
- Pill or oral medicine, Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 96 participants
- Where
- 48 sitesin 6 countries
- Study dates
- 21 May 2024to Feb 2027
- Phase
- Phase 1
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 75 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Established, pathologically confirmed diagnosis of AML by World Health Organization 2022 criteria.
- Newly diagnosed and previously untreated AML and eligible to receive intensive chemotherapy.
- KMT2Ar, NPM1m, or NUP98r mutations identified by local laboratory prior to the first dose of revumenib.
- Eastern Cooperative Oncology Group performance status ≤2 and ≤1 if >65 years old.
- Adequate liver, kidney, and cardiac function.
Who cannot join
- 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.
Show all 12Show fewer
- 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).
Trial locations and contacts
48 sites in 6 countries.
48 sites in 6 countries
United States28 sites
University of Michigan
Ann Arbor, Michigan, 48109
Emory Winship Cancer Institute
Atlanta, Georgia, 30322
Beth Israel Deaconess Medical Center
Boston, Massachusetts, 02215
UCLA Medical Hematology
Burbank, California, 91505
MUSC Hollings Cancer Center (HCC)
Charleston, South Carolina, 29425
University of Illinois
Chicago, Illinois, 60612
University of Chicago
Chicago, Illinois, 60637
Cleveland Clinic Foundation
Cleveland, Ohio, 44195
Baylor University Medical Center
Dallas, Texas, 75246
City of Hope Medical Center
Duarte, California, 91010
City of Hope
Goodyear, Arizona, 85338
East Carolina University
Greenville, North Carolina, 27834
The University of Texas, MD Anderson Cancer Center
Houston, Texas, 77030
Institution name: Northwell Health-Brany
Lake Success, New York, 11042
University of Louisville Health Brown Cancer Center
Louisville, Kentucky, 40202
Norton Cancer Institute, St. Matthews Campus
Louisville, Kentucky, 40207
Allina Health Cancer Institute
Minneapolis, Minnesota, 55407
West Virginia University
Morgantown, West Virginia, 26506
Memorial Sloan Kettering Cancer Center
New York, New York, 10065
AdventHealth Blood & Marrow Transplant Center
Orlando, Florida, 32804
UPMC Hillman Cancer Center
Pittsburgh, Pennsylvania, 15232
Oregon Health and Science University- Center for Hematologic Malignancies
Portland, Oregon, 97239
University of Rochester Medical Center
Rochester, New York, 14642
LDS Hospital - Intermountain Healthcare
Salt Lake City, Utah, 84143
Washington University School of Medicine
St Louis, Missouri, 63110
SUNY Upstate Medical University
Syracuse, New York, 13210
Tampa General Hospital
Tampa, Florida, 33606
Atrium Health Wake Forest Baptist Medical Center
Winston-Salem, North Carolina, 27157
Spain9 sites
Hospital Clinic de Barcelona
Barcelona, 8036
Hospital San Pedro de Alcantara
Cáceres, 10003
Hospital Universitario Virgen de Las Nieves
Granada, 18014
Hospital General Universitario Gregorio Marañon
Madrid, 28007
Hospital Universitario 12 de Octubre
Madrid, 28041
Hospital Universitario De Salamanca
Salamanca, 37007
Hospital Universitario Marques de Valdecilla
Santander, Cantabria, 39008
Hospital Universitario Virgen del Rocio - PPDS
Seville, 41013
Universitat de Valencia
Valencia, 46026
Australia5 sites
Royal Adelaide Hospital
Adelaide, SA 5000
Northern Hospital, Victoria
Epping, Victoria, 3076
The Alfred Hospital, Victoria
Melbourne, 3004
Sir Charles Gairdner Hospital
Nedlands, 6009
Royal Perth Hospital
Perth, Western Australia, 6000
Canada3 sites
University of Alberta Hospital
Edmonton, Alberta, T6G 2B7
The Sir Mortimer B. Davis Jewish General Hospital
Montreal, Quebec, H3T 1E2
Gordon and Leslie Diamond Health Care Center
Vancouver, British Columbia, V5Z 1M9
United Kingdom2 sites
Hammersmith Hospital
London, London, City of, W12 0HS
The Royal Marsden NHS
Sutton, SM2 5PT
About this study
Official title: A Phase 1, Open-label, Dose-escalation, and Dose-expansion Study to Evaluate Safety, Tolerability, and Clinical Activity of Revumenib 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 revumenib in combination with intensive chemotherapy in participants with newly diagnosed acute myeloid leukemia (AML) harboring alterations in KMT2A, NPM1, or NUP98 genes.
Read the detailed description
The Dose Escalation portion of this study will identify the maximum tolerated dose, or if different, the recommended Phase 2 dose of revumenib 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 revumenib 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 revumenib. The cycle duration will be 28 days.
- Conditions
- Acute Myeloid Leukemias
- Being tested
- Revumenib, Chemotherapy Regimen, HiDAC
- Keywords
- SNDX-5613, Lysine-specific Methyltransferase 2A, KMT2A/MLL, Nucleophosmin 1, NPM1, Nucleoporin 98, NUP98, AML, revumenib
- Medical subject headings
- Leukemia, Myeloid, Acute
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
RevumenibDrug
Also called SNDX-5613
Participants will receive revumenib orally during Induction, Consolidation, and Maintenance until meeting criteria for discontinuation.
Chemotherapy RegimenDrug
Induction: Participants will receive an intravenous (IV), 2-drug combination of cytarabine and either daunorubicin or idarubicin.
HiDACDrug
Consolidation: Participants will receive HiDAC IV.
| Study group | Role | What they receive |
|---|---|---|
| Revumenib | Experimental | Dose Escalation:
* Induction: Sequential cohorts of escalating dose levels of revumenib with chemotherapy regimen.
* Consolidation: Cohorts will receive high-dose cytarabine (HiDAC) chemotherapy followed by revumenib.
* Maintenance Monotherapy: Cohorts will receive revumenib
Dose Expansion:
* Induction: revumenib at tolerated dose level with chemotherapy regimen.
* Consolidation: Cohorts will receive revumenib with chemotherapy regimen and HiDAC.
* Maintenance Monotherapy: Cohorts will receive revumenib. Receives: Drug: Revumenib; Drug: Chemotherapy Regimen; Drug: HiDAC |
What is being measured
The primary outcome is the main question the study is designed to answer.
Dose Escalation: Number of Participants with Dose-limiting Toxicities
Measured: Up to Day 42
Number of Participants with Treatment-emergent Adverse Events (TEAEs)
Measured: Day 1 through 30 days after final dose (up to approximately 3 years)
Secondary outcomes (2)
Maximum Plasma Concentration (Cmax) of Revumenib and its Major Metabolite
Measured: Predose through Day 15
Area Under the Plasma Concentration Versus Time Curve From Time 0 to t (AUC0-t) of Revumenib and its Major Metabolite
Measured: Predose through Day 15
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted26 Jan 2024
- Study start21 May 2024
- Primary completionFeb 2027 (estimated)
- Study completionFeb 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Acute Myeloid LeukemiasStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Sequential
- Masking
- None (open label)
- Enrolment
- 96 participants (actual)
Sponsor and investigators
- Lead sponsor
- Syndax Pharmaceuticals
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06226571
- Sponsor's protocol ID
- SNDX-5613-0708
- Other IDs
- 2024-514531-18-00
- Status last verified
- Sep 2026
- First submitted
- 12 Jan 2024
- Last updated
- 16 Sep 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
- Expanded access
- Available
Study at a glance
From the registry record, last updated by the sponsor on 16 Sep 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 1, Open-label, Dose-escalation, and Dose-expansion Study to Evaluate Safety, Tolerability, and Clinical Activity of Revumenib 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
- ClinicalTrials.gov ID
- NCT06226571
- Acronym / sponsor ID
- SNDX-5613-0708
- Conditions
- Acute Myeloid Leukemias
- Keywords
- SNDX-5613, Lysine-specific Methyltransferase 2A, KMT2A/MLL, Nucleophosmin 1, NPM1, Nucleoporin 98, NUP98, AML
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 96 participants (actual)
- Lead sponsor
- Syndax PharmaceuticalsIndustry (pharma, biotech, devices)
- Study start
- 21 May 2024 (actual)
- Primary completion
- Feb 2027 (estimated)
- Study completion
- Feb 2027 (estimated)
- First posted
- 26 Jan 2024 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The purpose of this study is to evaluate the safety, tolerability, pharmacokinetics, and clinical activity of revumenib in combination with intensive chemotherapy in participants with newly diagnosed acute myeloid leukemia (AML) harboring alterations in KMT2A, NPM1, or NUP98 genes.
Detailed description
The Dose Escalation portion of this study will identify the maximum tolerated dose, or if different, the recommended Phase 2 dose of revumenib 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 revumenib 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 revumenib. The cycle duration will be 28 days.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Revumenib | Experimental | Drug: Revumenib, Drug: Chemotherapy Regimen, Drug: HiDACDose Escalation: * Induction: Sequential cohorts of escalating dose levels of revumenib with chemotherapy regimen. * Consolidation: Cohorts will receive high-dose cytarabine (HiDAC) chemotherapy followed by revumenib. * Maintenance Monotherapy: Cohorts will receive revumenib Dose Expansion: * Induction: revumenib at tolerated dose level with chemotherapy regimen. * Consolidation: Cohorts will receive revumenib with chemotherapy regimen and HiDAC. * Maintenance Monotherapy: Cohorts will receive revumenib. |
Who may qualify
- Ages
- 18 to 75 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (5 listed)
- Established, pathologically confirmed diagnosis of AML by World Health Organization 2022 criteria.
- Newly diagnosed and previously untreated AML and eligible to receive intensive chemotherapy.
- KMT2Ar, NPM1m, or NUP98r mutations identified by local laboratory prior to the first dose of revumenib.
- Eastern Cooperative Oncology Group performance status ≤2 and ≤1 if >65 years old.
- Adequate liver, kidney, and cardiac function.
Key exclusion criteria (12 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| RevumenibAlso: SNDX-5613Participants will receive revumenib orally during Induction, Consolidation, and Maintenance until meeting criteria for discontinuation. | Drug | Revumenib |
| Chemotherapy RegimenInduction: Participants will receive an intravenous (IV), 2-drug combination of cytarabine and either daunorubicin or idarubicin. | Drug | Revumenib |
| HiDACConsolidation: Participants will receive HiDAC IV. | Drug | Revumenib |
Primary outcome
Dose Escalation: Number of Participants with Dose-limiting Toxicities
Time frame: 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)
2 secondary outcomes
Maximum Plasma Concentration (Cmax) of Revumenib and its Major Metabolite
Time frame: Predose through Day 15
Area Under the Plasma Concentration Versus Time Curve From Time 0 to t (AUC0-t) of Revumenib and its Major Metabolite
Time frame: Predose through Day 15
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 16 Sep 2026.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
48 sites in 6 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Revumenib, Chemotherapy Regimen and HiDAC in people with Acute Myeloid Leukemias, run by Syndax Pharmaceuticals at 48 sites in 6 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 12 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: under way, not looking for participants, as of the sponsor's last update on 16 Sep 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 48 sites in United States, Spain, Australia, Canada, United Kingdom and 1 more country. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Dose Escalation: Number of Participants with Dose-limiting Toxicities (time frame: Up to Day 42). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 16 Sep 2026.