High-Throughput Proteomic Profiling to Evaluate Differentiation Syndrome With Menin Inhibition.
Miriam B Garcia, Bofei Wang, Irtiza Sheikh...
https://pubmed.ncbi.nlm.nih.gov/41621809Actively Recruiting
Phase 1
Phase 2
Age: 30Days +
All Genders
ID04065399
A Phase 1/2, Open-label, Dose-Escalation and Dose-Expansion Cohort Study of SNDX-5613 in Patients With Relapsed/Refractory Leukemias, Including Those Harboring an MLL/KMT2A Gene Rearrangement or Nucleophosmin 1 (NPM1) Mutation
Led by Syndax Pharmaceuticals · Updated on 2026-03-18
447
Participants Needed
57
Research Sites
N/A
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are studying revumenib, an oral drug, in people with relapsed or refractory acute leukemias, including those with specific genetic mutations like MLL/KMT2A rearrangements and NPM1 mutations. The study aims to find the highest safe dose and the recommended dose for further studies, as well as to evaluate how well the drug works, its safety, and how well patients tolerate it. This trial includes both a Phase 1 dose-escalation part and a Phase 2 expansion part focused on different leukemia types. In Phase 1, participants receive oral revumenib in one of six groups based on other medications they may be taking, such as antifungal drugs or cobicistat. The study tests increasing doses to find the maximum tolerated dose and recommended dose for Phase 2. In Phase 2, participants are grouped into four cohorts based on their leukemia subtype and genetic mutations to assess the drug's effects and safety over time. Participants will be monitored closely with assessments including blood tests to measure drug levels, side effects, and responses like remission rates. The study tracks outcomes such as adverse events, transfusion independence, and survival over several years. Participants’ physical health and leukemia status will be regularly evaluated to understand the treatment's impact and safety throughout the trial and follow-up periods.
CONDITIONS
Brief 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
You may qualify if you...
- Participants must have active acute leukemia with at least 5% bone marrow blasts or reappearance of blasts in peripheral blood, or acute leukemia with KMT2A rearrangement, NUP98 rearrangement, or NPM1 mutation with detectable bone marrow disease.
- For Phase 1, participants must fit into one of six arms based on use of CYP3A4 inhibitors or cobicistat.
- For Phase 2, participants must have relapsed or refractory acute leukemia with specific genetic mutations as defined for each cohort.
- White blood cell count below 25,000/microliter at enrollment; cytoreduction allowed before enrollment.
- Male or female participants aged 30 days and older; for those receiving cobicistat, weight must be at least 35 kg; Cohort 2D participants must be at least 18 years old and weigh at least 40 kg.
- ECOG performance status 0-2 or Karnofsky/Lansky score at least 50.
- Prior treatment-related toxicities resolved to Grade 1 or lower, except for Grade 2 neuropathy or alopecia.
- Specific timing requirements for prior radiation, stem cell infusion, immunotherapy, antileukemia therapy, hematopoietic growth factors, biologics, and steroids.
- Adequate organ function.
- Willingness to use effective contraception if of childbearing potential from enrollment through 120 days after last dose.
You will not qualify if you...
- Diagnosis of active acute promyelocytic leukemia.
- Isolated extramedullary relapse (Phase 2 Cohorts 2A-2C only).
- Active central nervous system disease.
- Detectable HIV viral load within the past 6 months.
- Hepatitis B or C infection.
- Pregnant or nursing women.
- Recent serious cardiac events or conditions including myocardial infarction, unstable angina, congestive heart failure (Class II or higher), life-threatening arrhythmia, stroke, or QTc interval above 450 milliseconds.
- Gastrointestinal conditions affecting oral drug absorption or ingestion, including gastric bypass or gastroparesis.
- Cirrhosis with Child-Pugh score B or C.
- Signs or symptoms of graft-versus-host disease greater than Grade 0 within 4 weeks prior to enrollment.
- Concurrent malignancy within past 2 years except certain skin cancers and in situ carcinomas treated with curative therapy, or low-grade lymphoma that is asymptomatic without progression.
- Use of medications known or suspected to prolong QT/QTc interval, except permitted supportive therapies and specified azoles.
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
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Your Study Journey
Screening
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Treatment
Duration - Up to approximately 3 years depending on cohort and response
Participants receive oral revumenib as part of dose-escalation arms or indication-specific expansion cohorts to evaluate safety and efficacy.
Regular visits for treatment and monitoring during dosing
Follow-up
Duration - Up to approximately 2 years after treatment completion
Participants continue to be monitored for safety and long-term outcomes after treatment ends.
Periodic follow-up visits
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
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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Published Research Related To This Trial
Menin inhibition with revumenib for NPM1-mutated relapsed or refractory acute myeloid leukemia: the AUGMENT-101 study.
Martha L Arellano, Michael J Thirman, John F DiPersio...
https://pubmed.ncbi.nlm.nih.gov/40332046Menin Inhibition With Revumenib for KMT2A-Rearranged Relapsed or Refractory Acute Leukemia (AUGMENT-101).
Ghayas C Issa, Ibrahim Aldoss, Michael J Thirman...
https://pubmed.ncbi.nlm.nih.gov/39121437The menin inhibitor revumenib in KMT2A-rearranged or NPM1-mutant leukaemia.
Ghayas C Issa, Ibrahim Aldoss, John DiPersio...
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Jennifer A Jiménez, April A Apfelbaum, Allegra G Hawkins...
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