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AzTrials

Acute Myeloid LeukemiaPhase 1/2AUGMENT-101NCT04065399

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

A Phase 1/2 study testing revumenib and cobicistat in people with Acute Myeloid Leukemia and Acute Lymphoblastic Leukemia, run by Syndax Pharmaceuticals at 57 sites in 11 countries.

Recruiting

Looking for participants now. Contact the study team or a site below to ask about taking part.

Sites and contacts
Official record
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Trial summary

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… Read more about this study

Who is it for?
Ages 30 days and older
What is being tested?
revumenib and cobicistat
Type of treatment
Pill or oral medicine
Could I receive a placebo?
No, everyone receives an active treatment
How many people
447 participants
Where
57 sitesin 11 countries
Study dates
5 Nov 2019to 15 Dec 2027
Phase
Phase 1/2
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 30 days and older
  • All sexes
  • No healthy volunteers
  • Child, Adult, Older adult

Who may be able to join

  • Participants must have active acute leukemia (bone marrow blasts ≥5% or reappearance of blasts in peripheral blood) as defined by the National Comprehensive Cancer Network (NCCN) in the NCCN Clinical Practice Guidelines in Oncology (NCCN Guidelines®) for Acute Lymphoblastic Leukemia (Version 1.2020) and Acute Myeloid Leukemia (Version 3.2020), or acute leukemia harboring KMT2A rearrangement, NUP98 rearrangement, or NPM1 mutation that have detectable disease in the bone marrow.
  • Phase 1:
  • Arm A: Participants not receiving any strong CYP3A4 inhibitor/inducers or fluconazole.
  • Arm B: Participants receiving itraconazole, ketoconazole, posaconazole, or voriconazole (strong CYP3A4 inhibitors) for antifungal prophylaxis.
  • Arm C: Participants receiving revumenib in combination with cobicistat.
  • Arm D: Participants receiving fluconazole (moderate CYP3A4 inhibitor).
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  • Arm E: Participants not receiving any weak, moderate, or strong CYP3A4 inhibitors/inducers.
  • Arm F: Participants receiving isavuconazole (moderate CYP3A4 inhibitor) for antifungal prophylaxis.
  • Phase 2:
  • Documented R/R active acute leukemia (bone marrow blasts ≥5% or reappearance of blasts in peripheral blood) as defined by the NCCN Guidelines® for Acute Lymphoblastic Leukemia (Version 1.2020) and Acute Myeloid Leukemia (Version 3.2020).
  • Cohort 2A: Documented R/R ALL/MPAL with KMT2A rearrangement.
  • Cohort 2B: Documented R/R AML with KMT2A rearrangement.
  • Cohort 2C: Documented R/R AML with NPM1m.
  • Cohort 2D: Documented R/R acute leukemia with a genetic mutation expected to lead to HOX/MEIS upregulation (for example, KMT2Ar, NPM1m, and NUP98r), including participants who are MRD-positive by multiparametric flow cytometry or molecular methods only, and including participants with isolated extramedullary disease.
  • White blood cell count below 25,000/ microliter at time of enrollment. Participants may receive cytoreduction prior to enrollment per protocol-specified criteria.
  • Male or female participants aged ≥30 days old. Participants intended to receive SNDX-5613 in combination with cobicistat must weigh ≥35 kilograms (kg). Participants in Cohort 2D must be ≥18 years of age and have a body weight ≥40 kg.
  • Eastern Cooperative Oncology Group (ECOG) performance status score 0-2 or Karnofsky/Lansky score ≥50.
  • Any prior treatment-related toxicities resolved to ≤Grade 1 prior to enrollment, with the exception of ≤Grade 2 neuropathy or alopecia.
  • Phase 1 and Phase 2 Cohorts 2A-2C only:
  • Radiation Therapy: At least 60 days from prior total body irradiation (TBI), craniospinal radiation and/or ≥50% radiation of the pelvis, or at least 14 days from local palliative radiation therapy (small port).
  • Stem Cell Infusion: At least 60 days must have elapsed from hematopoietic stem cell transplant and at least 4 weeks must have elapsed from donor lymphocyte infusion.
  • Immunotherapy: At least 42 days since prior immunotherapy, including tumor vaccines, and at least 21 days since receipt of chimeric antigen receptor therapy or other modified T or NK cell therapy.
  • Antileukemia Therapy: At least 14 days, or 5 half-lives, whichever is shorter, since the completion of antileukemic therapy.
  • Hematopoietic Growth Factors: At least 7 days since the completion of therapy with short-acting hematopoietic growth factors and 14 days with long-acting growth factors.
  • Biologics: At least 90 days, or 5 half-lives, whichever is shorter, since the completion of therapy with an antineoplastic biologic agent.
  • Steroids: At least 7 days since systemic glucocorticoid therapy, unless receiving physiologic dosing or cytoreductive therapy.
  • Phase 2 Cohort 2D only:
  • At least 14 days since any other investigational or commercially available antileukemic therapy, with the following exceptions:
  • Cytoreductive therapy with hydroxyurea, low-dose cytarabine (20 mg/square meter (m^2)/day subcutaneously [SC] for 10 days) or low-dose etoposide (up to 200 mg/day orally for 10 days) may be administered concurrently with SNDX-5613.
  • Intrathecal chemotherapy for CNS prophylaxis is permitted at the treating physician's discretion.
  • Steroids at physiologic dosing (equivalent to ≤10 mg prednisone daily for participants ≥18 years or ≤10 mg/m^2/day for participants <18 years) or for cytoreductive therapy.
  • Adequate organ function.
  • If of childbearing potential, willing to use a highly effective method of contraception from the time of enrollment through 120 days following the last study drug dose.

Who cannot join

  • Participants meeting any of the following criteria are not eligible for study participation:
  • Diagnosis of active acute promyelocytic leukemia.
  • Isolated extramedullary relapse (Phase 2 Cohorts 2A-2C only).
  • Active central nervous system disease (cytologic, such as any blasts on cytospin, or radiographic).
  • Detectable human immunodeficiency virus (HIV) viral load within the previous 6 months. Participants with a known history of HIV 1/2 antibodies must have viral load testing prior to study enrollment.
  • Hepatitis B or C.
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  • Pregnant or nursing women.
  • Cardiac Disease:
  • 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.
  • Corrected QT interval (QTc) >450 milliseconds.
  • Gastrointestinal Disease:
  • any gastrointestinal issue of the upper GI tract that might affect oral drug absorption or ingestion (that is, gastric bypass and gastroparesis).
  • Cirrhosis with a Child-Pugh score of B or C.
  • Graft-Versus-Host Disease (GVHD): Signs or symptoms of acute or chronic GVHD >Grade 0 within 4 weeks of enrollment. All transplant participants must have been off all systemic immunosuppressive therapy and calcineurin inhibitors for at least 4 weeks prior to enrollment. Participants may be on physiological doses of steroids.
  • Concurrent malignancy in the previous 2 years with the exception of basal cell carcinoma of the skin, squamous cell carcinoma of the skin, or carcinoma in situ (for example, breast carcinoma, cervical cancer in situ, melanoma in situ) treated with potentially curative therapy, or concurrent low-grade lymphoma, that is asymptomatic and lacks bulky disease and shows no evidence of progression, and for which the participant is not receiving any systemic therapy or radiation.
  • In Phase 1 and Phase 2: Participants requiring the concurrent use of medications known or suspected to prolong the QT/QTc interval, with the exception of drugs with low risk of QT/QTc prolongation that are used as standard supportive therapies (for example, diphenhydramine, famotidine, ondansetron, Bactrim) and the azoles permitted in the relevant arms of Phase 1 and in Phase 2.
  • Note: Other protocol defined inclusion/exclusion criteria may apply.

Trial locations and contacts

57 sites in 11 countries. Contact the study team or a site near you to ask about taking part.

Syndax Pharmaceuticals contact

781-419-1400

clinicaltrials@syndax.com

57 sites in 11 countries

United States22 sites, 10 recruiting

  • Emory Winship Cancer Institute

    Atlanta, Georgia, 30322

    Feranmi Arowolo, feranmi.arowolo@emory.edu

    Martha Arellano

    Recruiting

  • Children's Healthcare of Atlanta

    Atlanta, Georgia, 30329

    Completed

  • University of Colorado

    Aurora, Colorado, 80045

    Katelyn Anttila, Katelyn.Anttila@ucdenver.edu

    Christine McMahon

    Recruiting

  • Dana Farber Cancer Institute

    Boston, Massachusetts, 02215

    Active, not recruiting

  • The University of Chicago Medical Center

    Chicago, Illinois, 60637

    Bukola Rinola, 773-795-2430, Bukola.Rinola@bsd.uchicago.edu

    Michael Thirman

    Recruiting

  • University of Cincinnati

    Cincinnati, Ohio, 45267

    Completed

  • Ohio State University

    Columbus, Ohio, 43201

    Mandana Moghaddam, 614-685-2932, Mandana.Moghaddam@osumc.edu

    James Blachly

    Recruiting

  • City of Hope Comprehensive Cancer Center

    Duarte, California, 91010

    Active, not recruiting

  • Duke University Medical Center

    Durham, North Carolina, 27110

    Linda Brown, linda.brown@duke.edu

    Brittany DePriest

    Recruiting

  • Hackensack University Medical Center

    Hackensack, New Jersey, 07601

    Completed

  • The University of Texas MD Anderson Cancer Center

    Houston, Texas, 77030

    Active, not recruiting

  • University of Iowa Hospital

    Iowa City, Iowa, 52246

    Chris Stamy, 319-467-5320, chris-stamy@uiowa.edu

    Michael Halyko

    Recruiting

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

    Los Angeles, California, 90033

    Completed

  • Memorial Sloan Kettering Cancer Center

    New York, New York, 10065

    Active, not recruiting

  • Montefiore Medical Center

    New York, New York, 10467

    Aradhika Dhawan, aradhika.dhawan@einsteinmed.edu

    Ioannis Mantzaris

    Recruiting

  • Stanford Cancer Institute

    Palo Alto, California, 94305

    Active, not recruiting

  • University of Pennsylvania

    Philadelphia, Pennsylvania, 19104

    Robin E Blauser, BSN RN, 215-662-2870, Robin.Blauser@pennmedicine.upenn.edu

    Thomas Greenwood, Thomas.Greenwood@Pennmedicine.upenn.edu

    Recruiting

  • Oregon Health & Science University

    Portland, Oregon, 97239

    Tess Messan, 503-494-5466, Allentes@ohsu.edu

    Elie Traer, MD

    Recruiting

  • Huntsman Cancer Institute at the University of Utah

    Salt Lake City, Utah, 84112

    Completed

  • Florida Cancer Specialists and Research Institute

    Sarasota, Florida, 34232

    Active, not recruiting

  • Washington University in St. Louis School of Medicine

    St Louis, Missouri, 63110

    Morgan Swanner, swanner@wustl.edu

    John DiPersio

    Recruiting

  • Moffitt Cancer Center

    Tampa, Florida, 33162

    Completed

Other trials in United States

Germany7 sites, 4 recruiting

  • Universitaetsklinikum Essen (AoR)

    Essen, 45147

    Withdrawn

  • Universitaetsmedizin Greifswald

    Greifswald, 17475

    Completed

  • Universitaetsmedizin Der Johannes

    Gutenberg, 55131

    m3.uct-studien@unimedizin-mainz.de

    Recruiting

  • Universitaetsklinikum Hamburg-Eppendorf

    Hamburg, 20246

    Petra Kuehne, p.kuehne@uke.de

    Franziska Westendorf

    Recruiting

  • University of Leipzig

    Leipzig, 04103

    Karolin Neelsen, karolin.neelsen@medizin.uni-leipzig.de

    Madlen Jentzsch

    Recruiting

  • Klinikum Nuernberg Nord

    Nuremberg, 90419

    Completed

  • University Hospital Of Ulm, Universitatsklinikum Ulm

    Ulm, Baden-Wurttemberg, 89081

    Christiane Rudolph, +49 (0)731 500 45903, Christiane.Rudolph@uniklinik-ulm.de

    Hartmut Doehner

    Recruiting

Other trials in Germany

Israel6 sites, 6 recruiting

  • Rambam Health Care Campus (RHCC)

    Haifa, 3109601

    Lareen Bouban, l_boban@rambam.health.gov.il

    Avraham Frisch

    Recruiting

  • Shaare Zedek Medical Center

    Jerusalem, 9103102

    Razan Ewisat, razanew@szmc.org.il

    Yishai Ofran

    Recruiting

  • Hadassah Medical Center- Ein Kerem

    Jerusalem, 9112001

    Leah Greenfeld

    leahgr@hadassah.org.il

    Recruiting

  • Galilee Medical Center

    Nahariya, 2210010

    Lana Saada, LanaS@gmc.gov.il

    Galia Stemer

    Recruiting

  • Rabin Medical Center

    Petah Tikva, 4941492

    Shani Gillows, shanigil@clalit.org.il

    Ofir Wolach

    Recruiting

  • Sheba Medical Center

    Ramat Gan, 52621

    Yuval Carmel, Yuval.Carmel@sheba.health.gov.il

    Irina Armitai

    Recruiting

Other trials in Israel

Australia5 sites, 3 recruiting

  • Peter MacCallum Cancer Centre (PMCC)

    Melbourne, Victoria, 3000

    Active, not recruiting

  • Alfred Hospital

    Melbourne, 3004

    Shaun Fleming, MD

    Recruiting

  • Sir Charles Gairdner Hospital

    Nedlands, 6009

    Carolyn Grove, MD

    Recruiting

  • Royal Melbourne Hospital (RMH)

    Parkville, Victoria, 3050

    Active, not recruiting

  • Royal North Shore Hospital

    Saint Leonards, 2065

    Matthew Greenwood, MD

    Recruiting

Other trials in Australia

Italy5 sites, 5 recruiting

  • IRCCS Azienda Ospedaliero Universitaria di Bologna

    Bologna, 40138

    Riccardo Mennuti, riccardo.mennuti@aosp.bo.it

    Cristina Papayannidis

    Recruiting

  • Istituto Romagnolo Per Lo Studio dei tumori Dino Amadori

    Meldola, 47014

    Fabiana Mammoli, fabiana.mammoli@irst.emr.it

    Maria Giannini

    Recruiting

  • IRCCS-Istituto Europeo di Oncologia

    Milan, 20141

    Marialuisa Catalano, Marialuisa.Catalano@ieo.it

    Enrico Derenzini

    Recruiting

  • Universita Cattolica Fondazione Policlinico Agostino Gemelli

    Roma, 00168

    Gulia De Santis, giulia.desantis@policlinicogemelli.it

    Patrizia Chiusolo

    Recruiting

  • S Bortolo Hospital AULSS 8 Berica

    Vicenza, 36100

    Irene Mutterle, irene.mutterle@aulss.veneto.it

    Giorgia Scotton

    Recruiting

Other trials in Italy

France4 sites, 4 recruiting

  • Hospital Saint-Louis - APHP

    Paris, 75010

    Marion Le Duff, marion.leduff@aphp.fr

    Emmanuel Raffoux

    Recruiting

  • Centre Hospitalier Universitaire (CHU) de Bordeaux

    Pessac, 33604

    Thomas Grenier, thomas.grenier@chu-bordeaux.fr

    Arnaud Pigneux

    Recruiting

  • Centre Hospitalier Lyon Sud

    Pierre-Bénite, 69495

    James EAV, ext-james.eav@chu-lyon.fr

    Mael Heiblig

    Recruiting

  • Institut Gustave Roussy-Gustave Roussy Cancer Center -DITEP

    Villejuif, 94805

    Laetitia PIREYRE, Laetitia.PIREYRE@gustaveroussy.fr

    Stephanie De Botton

    Recruiting

Other trials in France

Spain3 sites, 3 recruiting

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

    L'Hospitalet de Llobregat, 08908

    Alvaro Gonzalez, amayo@idibell.cat

    Montserrat Arnan Sangerman

    Recruiting

  • Hospital Universitario Virgen del Rocio

    Seville, 41013

    Laura More, lauramorahuvr@gmail.com

    Eduardo Rodriguez-Arboli

    Recruiting

  • Hospital Universitari i Politecnic La Fe de Valencia

    Valencia, 46026

    Alvaro Fernandez-Pardo, alvaro_fernandez@iislafe.es

    Pau Montesinos Fernandez

    Recruiting

Other trials in Spain

Canada2 sites, 1 recruiting

  • University Health Network

    Toronto, M5G 2M9

    Allison Mahon, Allison.Mahon@uhn.ca

    Andre Schuh, MD

    Recruiting

  • The Hospital for Sick Children

    Toronto

    Active, not recruiting

Other trials in Canada

Lithuania1 site, 1 recruiting

  • Vilnius University Hospital Santaros Klinikos

    Vilnius, 08661

    Busevicius Mingaudas, mingaudas.busevicius@santa.lt

    Andrius Zucenka

    Recruiting

Other trials in Lithuania

Netherlands1 site, 1 recruiting

  • Princess Maxima Center for Pediatric Oncology

    Utrecht, 3584 CS

    Secretary Trial and Data Centrum, tdcsecretary@prinsesmaximacentrum.nl

    Michael Zwaan

    Not yet recruiting

Other trials in Netherlands

Puerto Rico1 site, 1 recruiting

  • Hospital Centro Comprensivo de Cancer UPR

    San Juan, 00935

    Coral Del Mar Lázaga Ocasio Ocasio, clazaga@cccupr.org

    Alexis Cruz-Chacon

    Recruiting

Other trials in Puerto Rico

About this study

Official title: 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

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.

Read the detailed description

Phase 1: Oral revumenib; sequential cohorts of escalating dose levels of revumenib to identify the MTD and RP2D. Participants will be enrolled in one of six dose-escalation arms:

Arm A: Participants not receiving any strong cytochrome P450 3A4 (CYP3A4) inhibitor/inducers or fluconazole.

Arm B: Participants receiving itraconazole, ketoconazole, posaconazole, or voriconazole (strong CYP3A4 inhibitors) for antifungal prophylaxis.

Arm C: Participants receiving revumenib and cobicistat.

Arm D: Participants receiving fluconazole (moderate CYP3A4 inhibitor) for antifungal prophylaxis.

Arm E: Participants not receiving any weak, moderate, or strong CYP3A4 inhibitors/inducers.

Arm F: Participants receiving isavuconazole (moderate CYP3A4 inhibitor) for antifungal prophylaxis.

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:

  • Cohort 2A: Participants with KMT2Ar acute lymphoblastic leukemia (ALL)/mixed phenotype acute leukemia (MPAL)
  • Cohort 2B: Participants with KMT2A AML
  • Cohort 2C: Participants with NPM1m AML
  • Cohort 2D: Participants with acute leukemia (including KMT2Ar, NPM1m, NUP98r and other acute leukemias expected to have HOX/MEIS upregulation)
Being tested
revumenib, cobicistat
Keywords
AML, ALL, MPAL, MLAL, ALAL, relapsed leukemia, refractory leukemia, acute leukemia, KMT2A, NPM1
Medical subject headings
Leukemia, Myeloid, Acute, Precursor Cell Lymphoblastic Leukemia-Lymphoma, Leukemia, Biphenotypic, Acute, Leukemia

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • revumenibDrug

    Also called SNDX-5613

    revumenib orally

  • cobicistatDrug

    Phase 1 Arm C participants will receive 150 mg cobicistat daily.

Study groups in NCT04065399
Study groupRoleWhat they receive
RevumenibExperimentalPhase 1: Oral revumenib; sequential cohorts of escalating dose levels of revumenib. Participants will be enrolled in 1 of 6 dose-escalation arms: * Arm A: Participants not receiving any strong CYP3A4 inhibitor/inducers or fluconazole * Arm B: Participants receiving any strong CYP3A4 inhibitors for antifungal prophylaxis * Arm C: Participants receiving revumenib and cobicistat * Arm D: Participants receiving fluconazole for antifungal prophylaxis * Arm E: Participants not receiving any weak, moderate, or strong CYP3A4 inhibitors/inducers * Arm F: Participants receiving isavuconazole for antifungal prophylaxis Phase 2: Oral revumenib; 4 indication-specific expansion cohorts will be enrolled: * Cohort 2A: Participants with KMT2Ar ALL/MPAL * Cohort 2B: Participants with KMT2Ar AML * Cohort 2C: Participants with NPM1m AML * Cohort 2D: Participants with acute leukemia (including KMT2Ar, NPM1m, NUP98r and other acute leukemias expected to have HOX/MEIS upregulation)
Receives: Drug: revumenib; Drug: cobicistat

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Number of participants with dose-limiting toxicities (DLTs) (Phase 1)

    Assessed by the NCI CTCAE version 5.0 (Phase 1)

    Measured: Approximately 1 year

  2. Number of participants with treatment-emergent adverse events (TEAEs) (Phase 1)

    Assessed by the NCI CTCAE version 5.0 (Phase 1)

    Measured: Approximately 1 year

  3. Cmax (Phase 1)

    Maximum plasma concentration (Cmax) of revumenib and relevant metabolites (Phase 1)

    Measured: Approximately 1 year

  4. Tmax (Phase 1)

    Time to observed maximum plasma concentration of revumenib and relevant metabolites (Phase 1)

    Measured: Approximately 1 year

  5. AUC0-t (Phase 1)

    Area under the plasma concentration-time curve from time 0 to time of last measurable concentration (AUC0-t) of revumenib and relevant metabolites (Phase 1)

    Measured: Approximately 1 year

  6. CR+CRh rate (Phase 2 [Cohorts 2A-2C])

    To assess the complete remission (CR) and complete remission with partial hematologic recovery (CRh) rate (Phase 2 [Cohorts 2A-2C])

    Measured: Approximately 3 years

  7. Number of participants with TEAEs (Phase 2 [Cohorts 2A-2C])

    Assessed by the NCI CTCAE version 5.0 (Phase 2 [Cohorts 2A-2C])

    Measured: Approximately 3 years

  8. Cmax (Phase 2 [Cohort 2D])

    Cmax of revumenib (Phase 2 [Cohort 2D])

    Measured: Approximately 3 years

  9. AUC0-tau (Phase 2 [Cohort 2D])

    Area under the plasma concentration-time curve from time 0 to the end of the dosing interval (AUC0-tau) of revumenib (Phase 2 [Cohort 2D])

    Measured: Approximately 3 years

Secondary outcomes (11)

  1. Transfusion independence (Phase 2 [Cohorts 2A-2C])

    Transfusion independence is defined as any transfusion-free period lasting for at least 56 consecutive days

    Measured: Approximately 3 years

  2. CRc rate (Phase 2 [Cohorts 2A-2C])

    To assess the composite definition of complete remission (CRc) rate (Phase 2 [Cohorts 2A-2C])

    Measured: Approximately 3 years

  3. ORR (CRc+ morphological leukemia-free state [MLFS] + partial remission [PR]) (Phase 2 [Cohorts 2A-2C])

    To assess the overall response rate (ORR) of revumenib (Phase 2 [Cohorts 2A-2C])

    Measured: Approximately 3 years

  4. TTR (Phase 2 [Cohorts 2A-2C])

    To assess the time to response (TTR) of revumenib (Phase 2 [Cohorts 2A-2C])

    Measured: Approximately 34 months

  5. DOR (Phase 2 [Cohorts 2A-2C])

    To assess the duration of response (DOR) of revumenib (Phase 2 [Cohorts 2A-2C])

    Measured: Approximately 3 years

  6. EFS (Phase 2 [Cohorts 2A-2C])

    To assess the event free survival (EFS) of revumenib (Phase 2 [Cohorts 2A-2C])

    Measured: Approximately 3 years

  7. OS (Phase 2 [Cohorts 2A-2C])

    To assess overall survival (OS) of revumenib (Phase 2 [Cohorts 2A-2C])

    Measured: Approximately 5 years

  8. Cmax (Phase 2 [Cohorts 2A-2C])

    Cmax of revumenib and relevant metabolites (Phase 2 [Cohorts 2A-2C])

    Measured: Approximately 3 years

  9. Tmax (Phase 2 [Cohorts 2A-2C])

    Tmax of revumenib and relevant metabolites (Phase 2 [Cohorts 2A-2C])

    Measured: Approximately 3 years

  10. AUC0-t (Phase 2 [Cohorts 2A-2C])

    AUC0-t of revumenib and relevant metabolites (Phase 2 [Cohorts 2A-2C])

    Measured: Approximately 3 years

  11. Number of participants with TEAEs (Phase 2 [Cohort 2D])

    Assessed by the NCI CTCAE version 5.0 (Phase 2 [Cohort 2D]))

    Measured: Approximately 3 years

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted22 Aug 2019
  2. Study start5 Nov 2019
  3. Primary completion15 Dec 2027 (estimated)
  4. Study completion15 Dec 2027 (estimated)

Resources

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 1/2
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Sequential
Model description
Phase 1 will employ an accelerated titration design. The dose escalation will follow a modified Fibonacci sequence.
Masking
None (open label)
Enrolment
447 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT04065399
Sponsor's protocol ID
SNDX-5613-0700
Other IDs
2020-004104-34
Acronym
AUGMENT-101
Status last verified
Oct 2026
First submitted
16 Aug 2019
Last updated
9 Oct 2026
Results posted
No
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
No
Expanded access
Available

Publications

  • Garcia MB, Wang B, Sheikh I, El Hajjar G, McCall D, Nunez C, Gibson A, Lorenzi PL, Issa GC, Cuglievan B, Abbas HA. High-Throughput Proteomic Profiling to Evaluate Differentiation Syndrome With Menin Inhibition. Mol Cell Proteomics. 2026 Mar;25(3):101522. doi: 10.1016/j.mcpro.2026.101522. Epub 2026 Jan 30.

    PubMed 41621809

  • Arellano ML, Thirman MJ, DiPersio JF, Heiblig M, Stein EM, Schuh AC, Zucenka A, de Botton S, Grove CS, Mannis GN, Papayannidis C, Perl AE, Issa GC, Aldoss I, Bajel A, Dickens DS, Kuhn MWM, Mantzaris I, Raffoux E, Traer E, Amitai I, Dohner H, Greco C, Kovacsovics T, McMahon CM, Montesinos P, Pigneux A, Shami PJ, Stone RM, Wolach O, Harpel JG, Chudnovsky Y, Yu L, Bagley RG, Smith AR, Blachly JS. Menin inhibition with revumenib for NPM1-mutated relapsed or refractory acute myeloid leukemia: the AUGMENT-101 study. Blood. 2025 Aug 28;146(9):1065-1077. doi: 10.1182/blood.2025028357.

    PubMed 40332046

  • Issa GC, Aldoss I, Thirman MJ, DiPersio J, Arellano M, Blachly JS, Mannis GN, Perl A, Dickens DS, McMahon CM, Traer E, Zwaan CM, Grove CS, Stone R, Shami PJ, Mantzaris I, Greenwood M, Shukla N, Cuglievan B, Kovacsovics T, Gu Y, Bagley RG, Madigan K, Chudnovsky Y, Nguyen HV, McNeer N, Stein EM. Menin Inhibition With Revumenib for KMT2A-Rearranged Relapsed or Refractory Acute Leukemia (AUGMENT-101). J Clin Oncol. 2025 Jan;43(1):75-84. doi: 10.1200/JCO.24.00826. Epub 2024 Aug 9.

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Questions about this trial

What is this clinical trial studying?

A Phase 1/2 study testing revumenib and cobicistat in people with Acute Myeloid Leukemia and Acute Lymphoblastic Leukemia, run by Syndax Pharmaceuticals at 57 sites in 11 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 33 inclusion and 17 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: looking for participants now, as of the sponsor's last update on 9 Oct 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 57 sites in United States, Germany, Israel, Australia, Italy and 6 more countries. 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 combined 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.

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

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?

Contact Syndax Pharmaceuticals (781-419-1400, clinicaltrials@syndax.com), or a site near the patient. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Number of participants with dose-limiting toxicities (DLTs) (Phase 1) (time frame: Approximately 1 year). The study lists 9 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT04065399, which also lists 6 linked publications.

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 9 Oct 2026.

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