Actively Recruiting

Phase 1
Phase 2
Age: 30Days +
All Genders
NCT04065399

A Study of Revumenib in R/R Leukemias Including Those With an MLL/KMT2A Gene Rearrangement or NPM1 Mutation

Led by Syndax Pharmaceuticals · Updated on 2026-03-18

447

Participants Needed

57

Research Sites

423 weeks

Total Duration

On this page

AI-Summary

What this Trial Is About

Phase 1 dose escalation will determine the maximum tolerated dose (MTD) and recommended Phase 2 dose (RP2D) of revumenib in participants with acute leukemia. In Phase 2, participants will be enrolled in 4 indication-specific expansion cohorts to determine the efficacy, short- and long-term safety, and tolerability of revumenib.

CONDITIONS

Official Title

A Study of Revumenib in R/R Leukemias Including Those With an MLL/KMT2A Gene Rearrangement or NPM1 Mutation

Who Can Participate

Age: 30Days +
All Genders

Eligibility Criteria

Eligible

You may qualify if you...

  • Participants must have active acute leukemia with 5% or more bone marrow blasts or reappearance of blasts in peripheral blood, including those with KMT2A rearrangement, NUP98 rearrangement, or NPM1 mutation.
  • Participants in Phase 1 are assigned to one of six arms based on use of CYP3A4 inhibitors/inducers or fluconazole and may receive revumenib with or without cobicistat.
  • Participants in Phase 2 must have documented relapsed or refractory acute leukemia matching specific genetic groups: KMT2A ALL/MPAL, KMT2A AML, NPM1 mutated AML, or acute leukemia with mutations leading to HOX/MEIS upregulation.
  • White blood cell count must be below 25,000 per microliter at enrollment.
  • Participants must be male or female aged at least 30 days; those receiving revumenib with cobicistat must weigh at least 35 kg; Cohort 2D participants must be at least 18 years old and weigh at least 40 kg.
  • Eastern Cooperative Oncology Group (ECOG) performance status 0-2 or Karnofsky/Lansky score 50 or higher.
  • Prior treatment-related toxicities must be resolved to Grade 1 or lower, except for Grade 2 neuropathy or alopecia.
  • Adequate time intervals required since prior radiation therapy, stem cell transplant, immunotherapy, antileukemic therapy, hematopoietic growth factors, biologics, and steroids.
  • Adequate organ function.
  • Participants of childbearing potential must agree to use highly effective contraception during the study and for 120 days afterward.
Not Eligible

You will not qualify if you...

  • Participants with active acute promyelocytic leukemia.
  • Participants with isolated extramedullary relapse (Phase 2 Cohorts 2A-2C only).
  • Participants with active central nervous system disease.
  • Participants with detectable HIV viral load within the past 6 months.
  • Participants with hepatitis B or C.
  • Pregnant or nursing women.
  • Participants with recent serious cardiac disease or prolonged QT interval.
  • Participants with gastrointestinal conditions affecting oral drug absorption or cirrhosis with Child-Pugh B or C.
  • Participants with signs or symptoms of graft-versus-host disease greater than Grade 0 within 4 weeks before enrollment.
  • Participants with concurrent malignancies within 2 years except certain treated skin cancers or asymptomatic low-grade lymphoma.
  • Participants requiring medications known or suspected to prolong QT interval, except certain allowed supportive drugs and azoles in study arms.

AI-Screening

AI-Powered Screening

Complete this quick 3-step screening to check your eligibility

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Trial Site Locations

Total: 57 locations

1

City of Hope Comprehensive Cancer Center

Duarte, California, United States, 91010

Active, Not Recruiting

2

University Of California Care Medical Group - Norris Comprehensive Cancer Center And Hospital

Los Angeles, California, United States, 90033

Completed

3

Stanford Cancer Institute

Palo Alto, California, United States, 94305

Active, Not Recruiting

4

University of Colorado

Aurora, Colorado, United States, 80045

Actively Recruiting

5

Florida Cancer Specialists and Research Institute

Sarasota, Florida, United States, 34232

Active, Not Recruiting

6

Moffitt Cancer Center

Tampa, Florida, United States, 33162

Completed

7

Emory Winship Cancer Institute

Atlanta, Georgia, United States, 30322

Actively Recruiting

8

Children's Healthcare of Atlanta

Atlanta, Georgia, United States, 30329

Completed

9

The University of Chicago Medical Center

Chicago, Illinois, United States, 60637

Actively Recruiting

10

University of Iowa Hospital

Iowa City, Iowa, United States, 52246

Actively Recruiting

11

Dana Farber Cancer Institute

Boston, Massachusetts, United States, 02215

Active, Not Recruiting

12

Washington University in St. Louis School of Medicine

St Louis, Missouri, United States, 63110

Actively Recruiting

13

Hackensack University Medical Center

Hackensack, New Jersey, United States, 07601

Completed

14

Memorial Sloan Kettering Cancer Center

New York, New York, United States, 10065

Active, Not Recruiting

15

Montefiore Medical Center

New York, New York, United States, 10467

Actively Recruiting

16

Duke University Medical Center

Durham, North Carolina, United States, 27110

Actively Recruiting

17

University of Cincinnati

Cincinnati, Ohio, United States, 45267

Completed

18

Ohio State University

Columbus, Ohio, United States, 43201

Actively Recruiting

19

Oregon Health & Science University

Portland, Oregon, United States, 97239

Actively Recruiting

20

University of Pennsylvania

Philadelphia, Pennsylvania, United States, 19104

Actively Recruiting

21

The University of Texas MD Anderson Cancer Center

Houston, Texas, United States, 77030

Active, Not Recruiting

22

Huntsman Cancer Institute at the University of Utah

Salt Lake City, Utah, United States, 84112

Completed

23

Peter MacCallum Cancer Centre (PMCC)

Melbourne, Victoria, Australia, 3000

Active, Not Recruiting

24

Royal Melbourne Hospital (RMH)

Parkville, Victoria, Australia, 3050

Active, Not Recruiting

25

Alfred Hospital

Melbourne, Australia, 3004

Actively Recruiting

26

Sir Charles Gairdner Hospital

Nedlands, Australia, 6009

Actively Recruiting

27

Royal North Shore Hospital

Saint Leonards, Australia, 2065

Actively Recruiting

28

University Health Network

Toronto, Canada, M5G 2M9

Actively Recruiting

29

The Hospital for Sick Children

Toronto, Canada

Active, Not Recruiting

30

Hospital Saint-Louis - APHP

Paris, France, 75010

Actively Recruiting

31

Centre Hospitalier Universitaire (CHU) de Bordeaux

Pessac, France, 33604

Actively Recruiting

32

Centre Hospitalier Lyon Sud

Pierre-Bénite, France, 69495

Actively Recruiting

33

Institut Gustave Roussy-Gustave Roussy Cancer Center -DITEP

Villejuif, France, 94805

Actively Recruiting

34

University Hospital Of Ulm, Universitatsklinikum Ulm

Ulm, Baden-Wurttemberg, Germany, 89081

Actively Recruiting

35

Universitaetsklinikum Essen (AoR)

Essen, Germany, 45147

Withdrawn

36

Universitaetsmedizin Greifswald

Greifswald, Germany, 17475

Completed

37

Universitaetsmedizin Der Johannes

Gutenberg, Germany, 55131

Actively Recruiting

38

Universitaetsklinikum Hamburg-Eppendorf

Hamburg, Germany, 20246

Actively Recruiting

39

University of Leipzig

Leipzig, Germany, 04103

Actively Recruiting

40

Klinikum Nuernberg Nord

Nuremberg, Germany, 90419

Completed

41

Rambam Health Care Campus (RHCC)

Haifa, Israel, 3109601

Actively Recruiting

42

Shaare Zedek Medical Center

Jerusalem, Israel, 9103102

Actively Recruiting

43

Hadassah Medical Center- Ein Kerem

Jerusalem, Israel, 9112001

Actively Recruiting

44

Galilee Medical Center

Nahariya, Israel, 2210010

Actively Recruiting

45

Rabin Medical Center

Petah Tikva, Israel, 4941492

Actively Recruiting

46

Sheba Medical Center

Ramat Gan, Israel, 52621

Actively Recruiting

47

IRCCS Azienda Ospedaliero Universitaria di Bologna

Bologna, Italy, 40138

Actively Recruiting

48

Istituto Romagnolo Per Lo Studio dei tumori Dino Amadori

Meldola, Italy, 47014

Actively Recruiting

49

IRCCS-Istituto Europeo di Oncologia

Milan, Italy, 20141

Actively Recruiting

50

Universita Cattolica Fondazione Policlinico Agostino Gemelli

Roma, Italy, 00168

Actively Recruiting

51

S Bortolo Hospital AULSS 8 Berica

Vicenza, Italy, 36100

Actively Recruiting

52

Vilnius University Hospital Santaros Klinikos

Vilnius, Lithuania, 08661

Actively Recruiting

53

Princess Maxima Center for Pediatric Oncology

Utrecht, Netherlands, 3584 CS

Actively Recruiting

54

Hospital Centro Comprensivo de Cancer UPR

San Juan, Puerto Rico, 00935

Actively Recruiting

55

Institut Catala d'Oncologia (ICO) - Hospital Duran i Reynals

L'Hospitalet de Llobregat, Spain, 08908

Actively Recruiting

56

Hospital Universitario Virgen del Rocio

Seville, Spain, 41013

Actively Recruiting

57

Hospital Universitari i Politecnic La Fe de Valencia

Valencia, Spain, 46026

Actively Recruiting

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Research Team

S

Syndax Pharmaceuticals

CONTACT

How is the study designed?

Study Type

INTERVENTIONAL

Masking

NONE

Allocation

NA

Model

SEQUENTIAL

Primary Purpose

TREATMENT

Number of Arms

1

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