Myelodysplastic SyndromePhase 2NCT06577441
Testing the Addition of an IDH2 Inhibitor, Enasidenib, to Usual Treatment (Cedazuridine-Decitabine) for Higher-Risk Myelodysplastic Syndrome (MDS) With IDH2 Mutation (A MyeloMATCH Treatment Trial)
A Phase 2 study testing Biospecimen Collection, Bone Marrow Aspiration and Bone Marrow Biopsy in people with Myelodysplastic Syndrome, run by National Cancer Institute (NCI) at 151 sites in 2 countries.
Trial summary
This phase II MyeloMATCH treatment trial compares the usual treatment of cedazuridine-decitabine (ASTX727) to the combination treatment of ASTX727 and enasidenib in treating patients with higher-risk, IDH2-mutated myelodysplastic syndrome (MDS). ASTX727 is a combination of two drugs, decitabine and cedazuridine. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Myelodysplastic Syndrome
- What is being tested?
- Biospecimen Collection, Bone Marrow Aspiration and Bone Marrow Biopsy
- Type of treatment
- Pill or oral medicine, Surgery or procedure
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 54 participants
- Where
- 151 sitesin 2 countries
- Study dates
- 12 Jun 2025to 1 Mar 2027
- Phase
- Phase 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 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- GENERAL MYLEOMATCH REGISTRATION CRITERIA:
- Patients must be registered to the Master Screening and Reassessment Protocol (MYELOMATCH) and assigned to this protocol by the MATCHBox Treatment Verification Team.
- Participants must not have received prior anti-cancer therapy for AML or MDS.
- Note: Hydroxyurea to control the white blood cell count (WBC) is allowed.
- Note: Prior erythroid stimulating agent (ESA) is not considered prior therapy for the purposes of eligibility.
- Participants must not be currently receiving any cytarabine-containing therapy other than up to 1 g/m^2 of cytarabine, which is allowed for urgent cytoreduction. The use of prior hydroxyurea, all-trans retinoic acid (ATRA), BCR-ABL directed tyrosine kinase inhibitor, erythropoiesis-stimulating agent, thrombopoietin receptor agonist and lenalidomide is allowed
Show all 33Show fewer
- REGISTRATION ELIGIBILITY CRITERIA (STEP 1): Patients must have a morphologically-confirmed diagnosis of MDS with a Revised International Prognostic Scoring System (IPSS-R) score ≥ 4.
- REGISTRATION ELIGIBILITY CRITERIA (STEP 1): Patients must have a detectable pathogenic IDH2 mutation based on the National Cancer Institute (NCI) Myeloid Panel.
- REGISTRATION ELIGIBILITY CRITERIA (STEP 1): No prior treatment with deoxyribonucleic acid (DNA) methyltransferase inhibitors (ASTX727, azacitidine, or decitabine).
- REGISTRATION ELIGIBILITY CRITERIA (STEP 1): Prior treatment with growth factors (ESA, granulocyte colony-stimulating factor [g-CSF], thrombopoietin [TPO] agonist) or luspatercept is allowed.
- REGISTRATION ELIGIBILITY CRITERIA (STEP 1): Prior treatment with lenalidomide with a maximum limit of 1 month of exposure is allowed.
- REGISTRATION ELIGIBILITY CRITERIA (STEP 1): Patients with therapy-related MDS are allowed.
- REGISTRATION ELIGIBILITY CRITERIA (STEP 1): Age ≥ 18 years.
- REGISTRATION ELIGIBILITY CRITERIA (STEP 1): Eastern Cooperative Oncology Group (ECOG) performance status ≤ 2.
- REGISTRATION ELIGIBILITY CRITERIA (STEP 1): Total bilirubin ≤ 1.5 x upper limit of normal (ULN)
- Unless elevated due to Gilbert's syndrome. In patients with Gilbert's syndrome, if the total bilirubin is ≤ 3.0 x ULN, then they are eligible for enrollment.
- REGISTRATION ELIGIBILITY CRITERIA (STEP 1): Aspartate aminotransferase (AST) (serum glutamic-oxaloacetic transaminase [SGOT])/alanine aminotransferase (ALT) (serum glutamic-pyruvic transaminase [SGPT]) ≤ 3.0 x upper limit of normal (ULN).
- REGISTRATION ELIGIBILITY CRITERIA (STEP 1): Creatinine clearance ≥ 30 mL/min
- To be calculated using Cockroft Gault formula.
- REGISTRATION ELIGIBILITY CRITERIA (STEP 1): Not pregnant and not nursing, because this study involves: an agent that has known genotoxic, mutagenic and teratogenic effects.
- Therefore, for women of childbearing potential only, a negative pregnancy test done as part of screening lab work prior to registration is required.
- REGISTRATION ELIGIBILITY CRITERIA (STEP 1): Patients with a prior or concurrent malignancy whose natural history or treatment does not have the potential to interfere with the safety or efficacy assessment of the investigational regimen are eligible for this trial.
- REGISTRATION ELIGIBILITY CRITERIA (STEP 1): HIV-infected patients on effective anti-retroviral therapy with undetectable viral load within 6 months prior to registration are eligible for this trial.
- REGISTRATION ELIGIBILITY CRITERIA (STEP 1): For patients with evidence of chronic hepatitis B virus (HBV) infection, the HBV viral load must be undetectable on suppressive therapy, if indicated.
- REGISTRATION ELIGIBILITY CRITERIA (STEP 1): Patients with a history of hepatitis C virus (HCV) infection must have been treated and cured. For patients with HCV infection who are currently on treatment, they are eligible if they have an undetectable HCV viral load.
- RE-REGISTRATION ELIGIBILITY CRITERIA (STEP 2): Patients on the ASTX727 monotherapy arm (Regimen 1) that do not achieve a CR (complete response), CRL (CR with limited count recovery), or CRh (CR with partial count recovery) after completing 6 cycles of study treatment.
- RE-REGISTRATION ELIGIBILITY CRITERIA (STEP 2): ECOG performance status ≤ 2.
- RE-REGISTRATION ELIGIBILITY CRITERIA (STEP 2): Total bilirubin ≤ 1.5 x upper limit of normal (ULN).
- Unless elevated due to Gilbert's syndrome. In patients with Gilbert's syndrome if the total bilirubin is ≤ 3.0 x ULN, then they are eligible for enrollment.
- RE-REGISTRATION ELIGIBILITY CRITERIA (STEP 2): AST (SGOT)/ALT (SGPT) ≤ 3.0 x upper limit of normal (ULN)
- RE-REGISTRATION ELIGIBILITY CRITERIA (STEP 2): Creatinine clearance ≥ 30 mL/min
- To be calculated using Cockroft Gault formula.
- RE-REGISTRATION ELIGIBILITY CRITERIA (STEP 2): Not pregnant and not nursing, because this study involves: an agent that has known genotoxic, mutagenic and teratogenic effects.
Who cannot join
None listed.
Trial locations and contacts
151 sites in 2 countries. Contact the study team or a site near you to ask about taking part.
151 sites in 2 countries
United States149 sites, 145 recruiting
- Recruiting
Phoebe Putney Memorial Hospital
Albany, Georgia, 31701
Site Public Contact, 229-312-0405, ga_cares@augusta.edu
Vamsi Kota
- Recruiting
University of New Mexico Cancer Center
Albuquerque, New Mexico, 87106
Site Public Contact, 505-925-0348, HSC-ClinicalTrialInfo@salud.unm.edu
Charles Foucar
- Recruiting
Community Hospital of Anaconda
Anaconda, Montana, 59711
Site Public Contact, 406-969-6060, mccinfo@mtcancer.org
John M. Schallenkamp
- Recruiting
UI Health Care Mission Cancer and Blood - Ankeny Clinic
Ankeny, Iowa, 50023
Site Public Contact, 515-241-3305
Seema Harichand-Herdt
- Recruiting
Duluth Clinic Ashland
Ashland, Wisconsin, 54806
Site Public Contact, 218-786-3308, CancerTrials@EssentiaHealth.org
Bret E. Friday
- Recruiting
LSU Health Baton Rouge-North Clinic
Baton Rouge, Louisiana, 70805
Site Public Contact, 225-765-7659, research@ololrmc.com
Harry G. Sequeira Gross
- Recruiting
Our Lady of the Lake Physician Group
Baton Rouge, Louisiana, 70808
Site Public Contact, 225-765-7659, research@ololrmc.com
Harry G. Sequeira Gross
- Recruiting
Our Lady of The Lake
Baton Rouge, Louisiana, 70808
Site Public Contact, 225-765-7659
Harry G. Sequeira Gross
- Active, not recruiting
Alta Bates Summit Medical Center-Herrick Campus
Berkeley, California, 94704
- Recruiting
Walter Reed National Military Medical Center
Bethesda, Maryland, 20889-5600
Site Public Contact, 301-319-2100
Ryan Jones
- Recruiting
Billings Clinic Cancer Center
Billings, Montana, 59101
Site Public Contact, 800-996-2663, research@billingsclinic.org
John M. Schallenkamp
- Recruiting
Illinois CancerCare-Bloomington
Bloomington, Illinois, 61704
Site Public Contact, 309-243-3605, andersonj@illinoiscancercare.com
Bryan A. Faller
- Recruiting
Prisma Health Cancer Institute - Spartanburg
Boiling Springs, South Carolina, 29316
Site Public Contact, 864-522-4317, Kim.Williams3@prismahealth.org
Suzanne R. Fanning
- Recruiting
Saint Luke's Cancer Institute - Boise
Boise, Idaho, 83712
Site Public Contact, 208-381-2774, eslinget@slhs.org
Charles W. Drescher
- Recruiting
Tufts Medical Center
Boston, Massachusetts, 02111
Site Public Contact, 617-636-5000, ContactUsCancerCenter@TuftsMedicalCenter.org
Andreas K. Klein
- Recruiting
Bozeman Health Deaconess Hospital
Bozeman, Montana, 59715
Site Public Contact, 406-969-6060, mccinfo@mtcancer.org
John M. Schallenkamp
- Recruiting
Essentia Health Saint Joseph's Medical Center
Brainerd, Minnesota, 56401
Site Public Contact, 218-786-3308, CancerTrials@EssentiaHealth.org
Bret E. Friday
- Recruiting
Trinity Health IHA Medical Group Hematology Oncology - Brighton
Brighton, Michigan, 48114
Site Public Contact, 734-712-7251, MCRCwebsitecontactform@stjoeshealth.org
Elie G. Dib
- Recruiting
Roswell Park Cancer Institute
Buffalo, New York, 14263
Site Public Contact, 800-767-9355, askroswell@roswellpark.org
Eunice S. Wang
- Recruiting
Illinois CancerCare-Canton
Canton, Illinois, 61520
Site Public Contact, 309-243-3605, andersonj@illinoiscancercare.com
Bryan A. Faller
- Recruiting
Trinity Health IHA Medical Group Hematology Oncology - Canton
Canton, Michigan, 48188
Site Public Contact, 734-712-7251, MCRCwebsitecontactform@stjoeshealth.org
Elie G. Dib
- Recruiting
Illinois CancerCare-Carthage
Carthage, Illinois, 62321
Site Public Contact, 309-243-3605, andersonj@illinoiscancercare.com
Bryan A. Faller
- Recruiting
Trinity Health IHA Medical Group Hematology Oncology - Chelsea Hospital
Chelsea, Michigan, 48118
Site Public Contact, 734-712-7251, MCRCwebsitecontactform@stjoeshealth.org
Elie G. Dib
- Recruiting
Northwestern University
Chicago, Illinois, 60611
Site Public Contact, 312-695-1301, cancer@northwestern.edu
Yasmin Abaza
- Recruiting
Swedish Covenant Hospital
Chicago, Illinois, 60625
Site Public Contact, 773-878-8200
Robert M. Eisner
- Recruiting
University of Chicago Comprehensive Cancer Center
Chicago, Illinois, 60637
Site Public Contact, 773-702-8222, cancerclinicaltrials@bsd.uchicago.edu
Anand A. Patel
- Recruiting
UI Health Care Mission Cancer and Blood - West Des Moines Clinic
Clive, Iowa, 50325
Site Public Contact, 515-241-3305
Seema Harichand-Herdt
- Recruiting
Kootenai Health - Coeur d'Alene
Coeur d'Alene, Idaho, 83814
Site Public Contact, 406-969-6060, mccinfo@mtcancer.org
John M. Schallenkamp
- Recruiting
Ohio State University Comprehensive Cancer Center
Columbus, Ohio, 43210
Site Public Contact, 800-293-5066, Jamesline@osumc.edu
Jayanshu Jain
- Recruiting
UChicago Medicine Northwest Indiana
Crown Point, Indiana, 46307
Site Public Contact, 855-702-8222, cancerclinicaltrials@bsd.uchicago.edu
Anand A. Patel
- Recruiting
Carle at The Riverfront
Danville, Illinois, 61832
Site Public Contact, 800-446-5532, Research@Carle.com
Vamsi K. Vasireddy
- Recruiting
Geisinger Medical Center
Danville, Pennsylvania, 17822
Site Public Contact, 570-271-5251, cancerresearch@geisinger.edu
Joseph J. Vadakara
- Recruiting
Cancer Care Specialists of Illinois - Decatur
Decatur, Illinois, 62526
Site Public Contact, 217-876-4762, morganthaler.jodi@mhsil.com
Bryan A. Faller
- Recruiting
Decatur Memorial Hospital
Decatur, Illinois, 62526
Site Public Contact, 217-876-4762, morganthaler.jodi@mhsil.com
Bryan A. Faller
- Recruiting
Essentia Health - Deer River Clinic
Deer River, Minnesota, 56636
Site Public Contact, 218-786-3308, CancerTrials@EssentiaHealth.org
Bret E. Friday
- Recruiting
Northwestern Medicine Cancer Center Kishwaukee
DeKalb, Illinois, 60115
Site Public Contact, 630-352-5360, Donald.Smith3@nm.org
Yasmin Abaza
- Recruiting
Iowa Methodist Medical Center
Des Moines, Iowa, 50309
Site Public Contact, 515-241-6727
Seema Harichand-Herdt
- Recruiting
UI Health Care Mission Cancer and Blood - Des Moines Clinic
Des Moines, Iowa, 50309
Site Public Contact, 515-241-3305
Seema Harichand-Herdt
- Recruiting
Mercy Medical Center - Des Moines
Des Moines, Iowa, 50314
Site Public Contact, 515-241-3305
Richard L. Deming
- Recruiting
UI Health Care Mission Cancer and Blood - Laurel Clinic
Des Moines, Iowa, 50314
Site Public Contact, 515-241-3305
Seema Harichand-Herdt
- Recruiting
Illinois CancerCare-Dixon
Dixon, Illinois, 61021
Site Public Contact, 815-285-7800
Bryan A. Faller
- Recruiting
Essentia Health Cancer Center
Duluth, Minnesota, 55805
Site Public Contact, 218-786-3308, CancerTrials@EssentiaHealth.org
Bret E. Friday
- Recruiting
Duke University Medical Center
Durham, North Carolina, 27710
Site Public Contact, 888-275-3853
Harry P. Erba
- Recruiting
Prisma Health Cancer Institute - Easley
Easley, South Carolina, 29640
Site Public Contact, 864-522-4317, Kim.Williams3@prismahealth.org
Suzanne R. Fanning
- Recruiting
Fairview Southdale Hospital
Edina, Minnesota, 55435
Site Public Contact, 952-993-1517, mmcorc@healthpartners.com
Yan Ji
- Recruiting
Minnesota Oncology - Edina
Edina, Minnesota, 55435
Site Public Contact, 952-993-1517, mmcorc@healthpartners.com
Yan Ji
- Recruiting
Swedish Cancer Institute-Edmonds
Edmonds, Washington, 98026
Site Public Contact, 206-215-2343, PCRC-NCORP@Swedish.org
Charles W. Drescher
- Recruiting
Carle Physician Group-Effingham
Effingham, Illinois, 62401
Site Public Contact, 800-446-5532, Research@carle.com
Vamsi K. Vasireddy
- Recruiting
Crossroads Cancer Center
Effingham, Illinois, 62401
Site Public Contact, 217-876-4762, morganthaler.jodi@mhsil.com
Bryan A. Faller
- Recruiting
Illinois CancerCare-Eureka
Eureka, Illinois, 61530
Site Public Contact, 309-243-3605, andersonj@illinoiscancercare.com
Bryan A. Faller
- Recruiting
NorthShore University HealthSystem-Evanston Hospital
Evanston, Illinois, 60201
Site Public Contact, 847-570-2109
Robert M. Eisner
- Recruiting
University of Kansas Clinical Research Center
Fairway, Kansas, 66205
Site Public Contact, 913-588-3671, KUCC_Navigation@kumc.edu
Jesus Gonzalez Lugo
- Recruiting
Essentia Health Cancer Center-South University Clinic
Fargo, North Dakota, 58103
Site Public Contact, 218-786-3308, CancerTrials@EssentiaHealth.org
Bret E. Friday
- Recruiting
Parkland Health Center - Farmington
Farmington, Missouri, 63640
Site Public Contact, 314-996-5569
Bryan A. Faller
- Recruiting
Cancer Hematology Centers - Flint
Flint, Michigan, 48503
Site Public Contact, 810-762-8038, wstrong@ghci.org
Elie G. Dib
- Suspended
Genesee Hematology Oncology PC
Flint, Michigan, 48503
- Recruiting
Genesys Hurley Cancer Institute
Flint, Michigan, 48503
Site Public Contact, 810-762-8038, wstrong@ghci.org
Elie G. Dib
- Recruiting
Saint Luke's Cancer Institute - Fruitland
Fruitland, Idaho, 83619
Site Public Contact, 208-381-2774, eslinget@slhs.org
Charles W. Drescher
- Recruiting
UF Health Cancer Institute - Gainesville
Gainesville, Florida, 32610
Site Public Contact, 352-273-8010
Dina G. Khalaf
- Recruiting
Illinois CancerCare-Galesburg
Galesburg, Illinois, 61401
Site Public Contact, 309-243-3605, andersonj@illinoiscancercare.com
Bryan A. Faller
- Recruiting
Northwestern Medicine Cancer Center Delnor
Geneva, Illinois, 60134
Site Public Contact, 630-352-5360, Donald.Smith3@nm.org
Yasmin Abaza
- Recruiting
NorthShore University HealthSystem-Glenbrook Hospital
Glenview, Illinois, 60026
Site Public Contact, 847-570-2109
Robert M. Eisner
- Recruiting
Northwestern Medicine Glenview Outpatient Center
Glenview, Illinois, 60026
Site Public Contact, 312-695-1102
Yasmin Abaza
- Recruiting
Northwestern Medicine Grayslake Outpatient Center
Grayslake, Illinois, 60030
Site Public Contact, 312-695-1102
Yasmin Abaza
- Recruiting
Benefis Sletten Cancer Institute
Great Falls, Montana, 59405
Site Public Contact, 406-969-6060, mccinfo@mtcancer.org
John M. Schallenkamp
- Recruiting
Saint Vincent Hospital Cancer Center Green Bay
Green Bay, Wisconsin, 54301
Site Public Contact, 920-433-8889, WI_research_admin@hshs.org
Anthony J. Jaslowski
- Recruiting
Saint Vincent Hospital Cancer Center at Saint Mary's
Green Bay, Wisconsin, 54303
Site Public Contact, 920-433-8889, wi_research_admin@hshs.org
Anthony J. Jaslowski
- Recruiting
East Carolina University
Greenville, North Carolina, 27834
Site Public Contact, 252-744-1015, eubankss@ecu.edu
Darla K. Liles
- Recruiting
Prisma Health Cancer Institute - Butternut
Greenville, South Carolina, 29605
Site Public Contact, 864-522-4317, Kim.Williams3@prismahealth.org
Suzanne R. Fanning
- Recruiting
Prisma Health Cancer Institute - Faris
Greenville, South Carolina, 29605
Site Public Contact, 864-522-4317, Kim.Williams3@prismahealth.org
Suzanne R. Fanning
- Recruiting
Prisma Health Cancer Institute - Eastside
Greenville, South Carolina, 29615
Site Public Contact, 864-522-4317, Kim.Williams3@prismahealth.org
Suzanne R. Fanning
- Recruiting
Prisma Health Cancer Institute - Greer
Greer, South Carolina, 29650
Site Public Contact, 864-522-4317, Kim.Williams3@prismahealth.org
Suzanne R. Fanning
- Recruiting
Essentia Health Hibbing Clinic
Hibbing, Minnesota, 55746
Site Public Contact, 218-786-3308
Bret E. Friday
- Recruiting
NorthShore University HealthSystem-Highland Park Hospital
Highland Park, Illinois, 60035
Site Public Contact, 847-570-2109
Robert M. Eisner
- Recruiting
Edward Hines Jr VA Hospital
Hines, Illinois, 60141
Site Public Contact, 708-202-8387
Jorgena Kosti-Schwartz
- Recruiting
University of Iowa/Holden Comprehensive Cancer Center
Iowa City, Iowa, 52242
Site Public Contact, 800-237-1225
Prajwal Dhakal
- Recruiting
UCI Health - Chao Family Comprehensive Cancer Center and Ambulatory Care
Irvine, California, 92612
Site Public Contact, 877-827-8839, ucstudy@uci.edu
Deepa Jeyakumar
- Recruiting
Swedish Cancer Institute-Issaquah
Issaquah, Washington, 98029
Site Public Contact, 206-215-2343, PCRC-NCORP@Swedish.org
Charles W. Drescher
- Recruiting
Logan Health Medical Center
Kalispell, Montana, 59901
Site Public Contact, 406-969-6060, mccinfo@mtcancer.org
John M. Schallenkamp
- Recruiting
University of Kansas Cancer Center
Kansas City, Kansas, 66160
Site Public Contact, 913-588-3671, KUCC_Navigation@kumc.edu
Jesus Gonzalez Lugo
- Recruiting
Illinois CancerCare-Kewanee Clinic
Kewanee, Illinois, 61443
Site Public Contact, 309-243-3605, andersonj@illinoiscancercare.com
Bryan A. Faller
- Suspended
University of Tennessee - Knoxville
Knoxville, Tennessee, 37920
- Recruiting
Gundersen Lutheran Medical Center
La Crosse, Wisconsin, 54601
Site Public Contact, 608-775-2385, cancerctr@gundersenhealth.org
Michael O. Ojelabi
- Recruiting
Northwestern Medicine Lake Forest Hospital
Lake Forest, Illinois, 60045
Site Public Contact, cancertrials@northwestern.edu
Yasmin Abaza
- Recruiting
Dartmouth Hitchcock Medical Center/Dartmouth Cancer Center
Lebanon, New Hampshire, 03756
Site Public Contact, 800-639-6918, cancer.research.nurse@dartmouth.edu
Patrick W. Mellors
- Recruiting
Cooperman Barnabas Medical Center
Livingston, New Jersey, 07039
Site Public Contact, 973-322-5200
Neil D. Palmisiano
- Recruiting
Trinity Health Saint Mary Mercy Livonia Hospital
Livonia, Michigan, 48154
Site Public Contact, 734-712-7251, MCRCwebsitecontactform@stjoeshealth.org
Elie G. Dib
- Recruiting
Monmouth Medical Center
Long Branch, New Jersey, 07740
Site Public Contact, 732-923-6564, mary.danish@rwjbh.org
Neil D. Palmisiano
- Recruiting
The James Graham Brown Cancer Center at University of Louisville
Louisville, Kentucky, 40202
Site Public Contact, 502-562-3429
Mohamed M. Hegazi
- Recruiting
UofL Health Medical Center Northeast
Louisville, Kentucky, 40245
Site Public Contact, 502-852-2755, ctoinfo@louisville.edu
Mohamed M. Hegazi
- Recruiting
Illinois CancerCare-Macomb
Macomb, Illinois, 61455
Site Public Contact, 309-243-3605, andersonj@illinoiscancercare.com
Bryan A. Faller
- Recruiting
William S Middleton VA Medical Center
Madison, Wisconsin, 53705
Site Public Contact, 608-256-1901 ext. 17007
Christopher D. Fletcher
- Recruiting
Carle Physician Group-Mattoon/Charleston
Mattoon, Illinois, 61938
Site Public Contact, 800-446-5532, Research@carle.com
Vamsi K. Vasireddy
- Recruiting
Loyola University Medical Center
Maywood, Illinois, 60153
Site Public Contact, 708-226-4357
Stephanie B. Tsai
- Recruiting
Saint Luke's Cancer Institute - Meridian
Meridian, Idaho, 83642
Site Public Contact, 208-381-2774, eslinget@slhs.org
Charles W. Drescher
- Recruiting
Miami Cancer Institute
Miami, Florida, 33176
Site Public Contact, 786-596-2000
Firas El Chaer
- Recruiting
Medical College of Wisconsin
Milwaukee, Wisconsin, 53226
Site Public Contact, 414-805-3666
Guru Subramanian Guru Murthy
- Recruiting
Abbott-Northwestern Hospital
Minneapolis, Minnesota, 55407
Site Public Contact, 952-993-1517, mmcorc@healthpartners.com
Yan Ji
- Recruiting
Hennepin County Medical Center
Minneapolis, Minnesota, 55415
Site Public Contact, 952-993-1517, mmcorc@healthpartners.com
Yan Ji
- Recruiting
Community Medical Center
Missoula, Montana, 59804
Site Public Contact, 406-969-6060, mccinfo@mtcancer.org
John M. Schallenkamp
- Recruiting
Saint Alphonsus Cancer Care Center-Nampa
Nampa, Idaho, 83687
Site Public Contact, 406-969-6060, mccinfo@mtcancer.org
Elie G. Dib
- Recruiting
Saint Luke's Cancer Institute - Nampa
Nampa, Idaho, 83687
Site Public Contact, 208-381-2774, eslinget@slhs.org
Charles W. Drescher
- Recruiting
Rutgers Cancer Institute of New Jersey
New Brunswick, New Jersey, 08903
Site Public Contact, 732-235-7356
Neil D. Palmisiano
- Recruiting
UC Comprehensive Cancer Center at Silver Cross
New Lenox, Illinois, 60451
Site Public Contact, 773-702-8222, cancerclinicaltrials@bsd.uchicago.edu
Anand A. Patel
- Recruiting
Cancer Care Center of O'Fallon
O'Fallon, Illinois, 62269
Site Public Contact, 217-876-4762, morganthaler.jodi@mhsil.com
Bryan A. Faller
- Recruiting
HSHS Saint Elizabeth's Hospital
O'Fallon, Illinois, 62269
Site Public Contact, 217-876-4762, morganthaler.jodi@mhsil.com
Bryan A. Faller
- Recruiting
University of Oklahoma Health Sciences Center
Oklahoma City, Oklahoma, 73104
Site Public Contact, 405-271-8777, ou-clinical-trials@ouhsc.edu
Zimu Gong
- Recruiting
UC Irvine Health/Chao Family Comprehensive Cancer Center
Orange, California, 92868
Site Public Contact, 877-827-8839, ucstudy@uci.edu
Deepa Jeyakumar
- Recruiting
Northwestern Medicine Orland Park
Orland Park, Illinois, 60462
Site Public Contact, nctnprogram_rhlccc@northwestern.edu
Yasmin Abaza
- Recruiting
University of Chicago Medicine-Orland Park
Orland Park, Illinois, 60462
Site Public Contact, 773-702-8222, cancerclinicaltrials@bsd.uchicago.edu
Anand A. Patel
- Recruiting
Illinois CancerCare-Ottawa Clinic
Ottawa, Illinois, 61350
Site Public Contact, 309-243-3605, andersonj@illinoiscancercare.com
Bryan A. Faller
- Recruiting
Illinois CancerCare-Pekin
Pekin, Illinois, 61554
Site Public Contact, 309-243-3605, andersonj@illinoiscancercare.com
Bryan A. Faller
- Recruiting
UI Healthcare Mission Cancer and Blood - Pella
Pella, Iowa, 50219
Site Public Contact, 515-282-2921, trials@missioncancer.com
Seema Harichand-Herdt
- Recruiting
Memorial Hospital West
Pembroke Pines, Florida, 33028
Site Public Contact, 954-265-4325
Fernando M. Vargas Madueno
- Recruiting
Illinois CancerCare-Peoria
Peoria, Illinois, 61615
Site Public Contact, 309-243-3605, andersonj@illinoiscancercare.com
Bryan A. Faller
- Recruiting
Illinois CancerCare-Peru
Peru, Illinois, 61354
Site Public Contact, 309-243-3605, andersonj@illinoiscancercare.com
Bryan A. Faller
- Recruiting
UPMC Hillman Cancer Center
Pittsburgh, Pennsylvania, 15232
Site Public Contact, 412-647-8073
Annie P. Im
- Recruiting
Trinity Health Saint Joseph Mercy Oakland Hospital
Pontiac, Michigan, 48341
Site Public Contact, 734-712-7251, MCRCwebsitecontactform@stjoeshealth.org
Elie G. Dib
- Recruiting
Kootenai Clinic Cancer Services - Post Falls
Post Falls, Idaho, 83854
Site Public Contact, 406-969-6060, mccinfo@mtcancer.org
John M. Schallenkamp
- Recruiting
Illinois CancerCare-Princeton
Princeton, Illinois, 61356
Site Public Contact, 309-243-3605, andersonj@illinoiscancercare.com
Bryan A. Faller
- Recruiting
VCU Massey Comprehensive Cancer Center
Richmond, Virginia, 23298
Site Public Contact, 804-628-6430, CTOclinops@vcu.edu
Keri R. Maher
- Recruiting
Park Nicollet Clinic - Saint Louis Park
Saint Louis Park, Minnesota, 55416
Site Public Contact, 952-993-1517, mmcorc@healthpartners.com
Yan Ji
- Recruiting
Regions Hospital
Saint Paul, Minnesota, 55101
Site Public Contact, 952-993-1517, mmcorc@healthpartners.com
Yan Ji
- Recruiting
United Hospital
Saint Paul, Minnesota, 55102
Site Public Contact, 952-993-1517, mmcorc@healthpartners.com
Yan Ji
- Recruiting
Sainte Genevieve County Memorial Hospital
Sainte Genevieve, Missouri, 63670
Site Public Contact, 314-996-5569
Bryan A. Faller
- Recruiting
Huntsman Cancer Institute/University of Utah
Salt Lake City, Utah, 84112
Site Public Contact, 888-424-2100, cancerinfo@hci.utah.edu
Paul J. Shami
- Active, not recruiting
Mills Health Center
San Mateo, California, 94401
- Recruiting
Kootenai Clinic Cancer Services - Sandpoint
Sandpoint, Idaho, 83864
Site Public Contact, 406-969-6060, mccinfo@mtcancer.org
John M. Schallenkamp
- Recruiting
Essentia Health Sandstone
Sandstone, Minnesota, 55072
Site Public Contact, 218-786-3308, CancerTrials@EssentiaHealth.org
Bret E. Friday
- Recruiting
Swedish Medical Center-First Hill
Seattle, Washington, 98122
Site Public Contact, 206-215-2343, PCRC-NCORP@Swedish.org
Charles W. Drescher
- Recruiting
Prisma Health Cancer Institute - Seneca
Seneca, South Carolina, 29672
Site Public Contact, 864-522-4317, Kim.Williams3@prismahealth.org
Suzanne R. Fanning
- Recruiting
Southern Illinois University School of Medicine
Springfield, Illinois, 62702
Site Public Contact, 217-545-7929
Bryan A. Faller
- Recruiting
Springfield Clinic
Springfield, Illinois, 62702
Site Public Contact, 800-444-7541
Bryan A. Faller
- Recruiting
Springfield Memorial Hospital
Springfield, Illinois, 62781
Site Public Contact, 217-528-7541, pallante.beth@mhsil.com
Bryan A. Faller
- Recruiting
Missouri Baptist Medical Center
St Louis, Missouri, 63131
Site Public Contact, 314-996-5569
Bryan A. Faller
- Recruiting
Marshfield Medical Center-River Region at Stevens Point
Stevens Point, Wisconsin, 54482
Site Public Contact, 800-782-8581, oncology.clinical.trials@marshfieldresearch.org
Adedayo A. Onitilo
- Recruiting
Stony Brook University Medical Center
Stony Brook, New York, 11794
Site Public Contact, 800-862-2215
Suhu Liu
- Recruiting
Saint Vincent Hospital Cancer Center at Sturgeon Bay
Sturgeon Bay, Wisconsin, 54235-1495
Site Public Contact, 920-433-8889, wi_research_admin@hshs.org
Anthony J. Jaslowski
- Recruiting
Missouri Baptist Sullivan Hospital
Sullivan, Missouri, 63080
Site Public Contact, 314-996-5569
Bryan A. Faller
- Recruiting
Community Medical Center
Toms River, New Jersey, 08755
Site Public Contact, 732-557-8294, Lennette.Gonzales@rwjbh.org
Neil D. Palmisiano
- Recruiting
Carle Cancer Center
Urbana, Illinois, 61801
Site Public Contact, 800-446-5532, Research@carle.com
Vamsi K. Vasireddy
- Recruiting
Essentia Health Virginia Clinic
Virginia, Minnesota, 55792
Site Public Contact, 218-786-3308, CancerTrials@EssentiaHealth.org
Bret E. Friday
- Recruiting
Northwestern Medicine Cancer Center Warrenville
Warrenville, Illinois, 60555
Site Public Contact, 630-352-5360, Donald.Smith3@nm.org
Yasmin Abaza
- Recruiting
Illinois CancerCare - Washington
Washington, Illinois, 61571
Site Public Contact, 309-243-3605, andersonj@illinoiscancercare.com
Bryan A. Faller
- Recruiting
UI Health Care Mission Cancer and Blood - Waukee Clinic
Waukee, Iowa, 50263
Site Public Contact, 515-241-3305
Seema Harichand-Herdt
- Recruiting
Marshfield Medical Center - Weston
Weston, Wisconsin, 54476
Site Public Contact, 800-782-8581, oncology.clinical.trials@marshfieldresearch.org
Adedayo A. Onitilo
- Recruiting
University of Kansas Hospital-Westwood Cancer Center
Westwood, Kansas, 66205
Site Public Contact, 913-588-3671, KUCC_Navigation@kumc.edu
Jesus Gonzalez Lugo
- Recruiting
Geisinger Wyoming Valley/Henry Cancer Center
Wilkes-Barre, Pennsylvania, 18711
Site Public Contact, 570-271-5251, HemonCCTrials@geisinger.edu
Joseph J. Vadakara
- Recruiting
Trinity Health IHA Medical Group Hematology Oncology Ann Arbor Campus
Ypsilanti, Michigan, 48197
Site Public Contact, 734-712-7251, MCRCwebsitecontactform@stjoeshealth.org
Elie G. Dib
Puerto Rico2 sites, 2 recruiting
- Recruiting
Centro Comprensivo de Cancer de UPR
San Juan, 00927
Site Public Contact, 888-823-5923, ctsucontact@westat.com
Luis J. Santos Reyes
- Recruiting
San Juan City Hospital
San Juan, 00936
Site Public Contact, 787-763-1296
Luis J. Santos Reyes
About this study
Official title: A Randomized Phase II Trial of Enasidenib-Based Therapies Versus Cedazuridine-Decitabine in Higher-Risk IDH2-Mutated Myelodysplastic Syndrome: A MyeloMATCH Sub-Study
This phase II MyeloMATCH treatment trial compares the usual treatment of cedazuridine-decitabine (ASTX727) to the combination treatment of ASTX727 and enasidenib in treating patients with higher-risk, IDH2-mutated myelodysplastic syndrome (MDS). ASTX727 is a combination of two drugs, decitabine and cedazuridine. Cedazuridine is in a class of medications called cytidine deaminase inhibitors. It prevents the breakdown of decitabine, making it more available in the body so that decitabine will have a greater effect. Decitabine is in a class of medications called hypomethylation agents. It works by helping the bone marrow produce normal blood cells and by killing abnormal cells in the bone marrow. Enasidenib is an enzyme inhibitor that may stop the growth of cells by blocking some of the enzymes needed for cell growth. Giving ASTX727 in combination with enasidenib may be effective in treating patients with higher-risk IDH2-mutated MDS.
Read the detailed description
PRIMARY OBJECTIVE:
- To compare the complete remission (CR) rate of enasidenib + decitabine and cedazuridine (ASTX727) and ASTX727 monotherapy in patients with higher-risk IDH2-mutated MDS using International Working Group 2023 (IWG2023) response criteria.
SECONDARY OBJECTIVES:
- To estimate the median event-free survival (EFS) at designated time point(s) for each treatment arm.
II. To estimate the median overall survival (OS) at designated time point(s) for each treatment arm.
III. To estimate the frequency and severity of toxicities with each regimen in this patient population.
IV. To estimate the median time to response for each treatment arm. V. To estimate the median duration of response for each treatment arm. VI. To estimate the IDH2 variant allele frequency (VAF) reduction for each treatment arm.
VII. To estimate the rate of allogeneic hematopoietic cell transplantation for each treatment arm.
VIII. To compare rates of partial response (PR), CR with limited count recovery (CRL), CR with partial count recovery (CRh), and hematologic improvement (HI) using IWG 2023 response criteria between treatment arms.
IX. To compare the measurable residual disease (MRD) kinetics by flow cytometry and next generation sequencing (NGS) at designated time point(s) at the end of cycle 4 \& 6 and to assess any correlation with clinical outcomes (e.g. CR, EFS, OS).
- To estimate the median time to transformation of MDS to acute myeloid leukemia (AML).
EXPLORATORY OBJECTIVES:
- To estimate CR rate, median EFS, and median OS in patients treated with ASTX727 monotherapy that crossover to the treatment arm with ASTX727 + enasidenib after 6 cycles if CR is not achieved.
II. To estimate CR rate, median EFS, and median OS for patients based on Molecular International Prognostic Scoring System (IPSS-M) prognostic risk score at diagnosis, stratified for score level.
III. To estimate concordance between centrally-performed molecular studies and cytogenetics to those done locally.
OUTLINE: Patients are randomized to 1 of 2 regimens.
REGIMEN 1: Patients receive ASTX727 orally (PO) once daily (QD) on days 1-5 of each cycle. Cycles repeat every 28 days in the absence of disease progression or unacceptable toxicity. Patients who do not achieve a CR, CRL, or CRh at the end of cycle 6 may cross-over to Regimen 2. Patients who experience CR, PR, or stable disease (SD) any time after 4 cycles of treatment may be reassessed in order to go to a higher myeloMATCH tier assignment or to Tier Advancement Pathway (TAP). Patients also undergo bone marrow biopsy and aspiration throughout the study. Patients may also undergo optional buccal swab on study, and/or optional additional bone marrow aspiration and blood sample collection on study and at disease progression.
REGIMEN 2: Patients receive ASTX727 PO QD on days 1-5 and enasidenib PO QD on days 1-28 of each cycle. Cycles repeat every 28 days in the absence of disease progression or unacceptable toxicity. Patients who experience CR, PR, or SD any time after 4 cycles of treatment may be reassessed in order to go to a higher myeloMATCH tier assignment or to TAP. Patients also undergo bone marrow biopsy and aspiration throughout the study. Patients may also undergo optional buccal swab on study, and/or optional additional bone marrow aspiration and blood sample collection on study and at disease progression.
After completion of study treatment, patients are followed up every 6 months for up to 5 years after registration.
- Conditions
- Myelodysplastic Syndrome
- Being tested
- Biospecimen Collection, Bone Marrow Aspiration, Bone Marrow Biopsy, Decitabine and Cedazuridine, Enasidenib
- Medical subject headings
- Myelodysplastic Syndromes
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Biospecimen CollectionProcedure or surgery
Also called Biological Sample Collection, Biospecimen Collected, Sample Collection, Specimen Collection
Undergo buccal swab and blood sample collection
Bone Marrow AspirationProcedure or surgery
Undergo bone marrow aspiration
Bone Marrow BiopsyProcedure or surgery
Also called Biopsy of Bone Marrow, Biopsy, Bone Marrow
Undergo bone marrow biopsy
Decitabine and CedazuridineDrug
Also called ASTX 727, ASTX-727, ASTX727, C-DEC
Given PO
EnasidenibDrug
Also called AG 221, AG-221, AG221, CC-90007 Free Base
Given PO
| Study group | Role | What they receive |
|---|---|---|
| Regimen 1 (ASTX727) | Active comparator | Patients receive ASTX727 PO QD on days 1-5 of each cycle. Cycles repeat every 28 days in the absence of disease progression or unacceptable toxicity. Patients who do not achieve a CR, CRL, or CRh at the end of cycle 6 may cross-over to Regimen 2. Patients who experience CR, PR, or SD any time after 4 cycles of treatment may be reassessed in order to go to a higher myeloMATCH tier assignment or to TAP. Patients also undergo bone marrow biopsy and aspiration throughout the study. Patients may also undergo optional buccal swab on study, and/or optional additional bone marrow aspiration and blood sample collection on study and at disease progression. Receives: Procedure: Biospecimen Collection; Procedure: Bone Marrow Aspiration; Procedure: Bone Marrow Biopsy; Drug: Decitabine and Cedazuridine |
| Regimen 2 (ASTX727, enasidenib) | Experimental | Patients receive ASTX727 PO QD on days 1-5 and enasidenib PO QD on days 1-28 of each cycle. Cycles repeat every 28 days in the absence of disease progression or unacceptable toxicity. Patients who experience CR, PR, or SD any time after 4 cycles of treatment may be reassessed in order to go to a higher myeloMATCH tier assignment or to TAP. Patients also undergo bone marrow biopsy and aspiration throughout the study. Patients may also undergo optional buccal swab on study, and/or optional additional bone marrow aspiration and blood sample collection on study and at disease progression. Receives: Procedure: Biospecimen Collection; Procedure: Bone Marrow Aspiration; Procedure: Bone Marrow Biopsy; Drug: Decitabine and Cedazuridine; Drug: Enasidenib |
What is being measured
The primary outcome is the main question the study is designed to answer.
Complete response (CR) rate
Will be assessed using the International Working Group 2023 (IWG2023) criteria. Will compare the CR rate between the two treatment arms to determine if patients treated with enasidenib + ASTX727 have a statistically significantly higher CR rate than patients treated with the ASTX727 monotherapy.
Measured: Up to 4 cycles of treatment
Secondary outcomes (10)
Event-free survival (EFS)
Sensitivity analyses will be conducted. Will use the methods of Kaplan-Meier as well as Cox regression models.
Measured: Time from randomization to either a failure to achieve a CR, CR with limited count recovery (CRL), or CR with partial count recovery (CRh) after four cycles of treatment, relapse, or death due to any cause, assessed up to 18 months
Overall survival (OS)
Will use the methods of Kaplan-Meier as well as Cox regression models.
Measured: Time from randomization to death due to any cause, assessed up to 18 months
Time to response
Will be assessed only among patients who achieve a response (CR, CRL, or CRh). Will utilize the methods of Kaplan-Meier as well as Cox regression models.
Measured: Time from randomization to documented response, assessed up to 5 years
Duration of response
Will be assessed only among patients who achieve a response (CR, CRL, or CRh). Will use the methods of Kaplan-Meier as well as Cox regression models.
Measured: From patient first achieves a response until either progression or death, assessed up to 5 years
Incidence of adverse events
Will be determined using the Common Terminology Criteria for Adverse Events version 5 criteria. The maximum grade for each type of toxicity will be recorded for each patient, and frequency tables will be reviewed to determine toxicity patterns. Will review all adverse event data that is graded as 3, 4, or 5 and classified as either "unrelated" or "unlikely to be related" to study treatment in the event of an actual relationship developing. The incidence of severe (grade 3+) adverse events or toxicities will be described for each treatment arm, but will also be compared between the arms. Fisher's exact tests will be used to quantitatively compare the incidence of severe as well as specific toxicities of interest and will graphically assess differences in maximum grades observed for toxicities.
Measured: Up to 4 weeks after completion of study treatment
Change in IDH2 variant allele frequency (VAF)
Will report the percent change of VAF along with a 95% confidence interval.
Measured: Baseline to end of cycle 4 and 6
Allogeneic stem cell transplantation rate
Will report along with a 95% confidence interval for each treatment arm.
Measured: Up to 5 years
Measurable residual disease rate
Will be measured by flow cytometry and next generation sequencing. Will be compared and used to determine association with clinical outcomes such as CR, EFS, or OS.
Measured: At the end of cycle 4 and 6
Cytogenetic and molecular features
Cytogenetic and molecular classifications including Molecular International Prognostic Scoring System will be compared and used to determined association with clinical outcomes such as CR, EFS, and OS.
Measured: Up to 5 years
Time to transformation of myelodysplastic syndrome to acute myeloid leukemia (AML)
Will utilize the methods of Kaplan-Meier as well as Cox regression models.
Measured: From randomization to transformation to AML or death from any causes, assessed up to 5 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted29 Aug 2024
- Study start12 Jun 2025
- Primary completion1 Mar 2027 (estimated)
- Study completion1 Mar 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Myelodysplastic SyndromeStudies of the same condition, open and under way.
- More trials run by National Cancer Institute (NCI)The sponsor's other registered studies.
- 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 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 54 participants (estimated)
Sponsor and investigators
- Lead sponsor
- National Cancer Institute (NCI)
- Sponsor type
- US National Institutes of Health
- Responsible party
- Sponsor
- Investigators
- Anand A Patel, Principal investigator, Alliance for Clinical Trials in Oncology
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06577441
- Sponsor's protocol ID
- NCI-2024-07049
- Other IDs
- NCI-2024-07049, MM1MDS-A01, MM1MDS-A01, U10CA180821
- Status last verified
- Aug 2026
- First submitted
- 28 Aug 2024
- Last updated
- 4 Nov 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Yes: NCI is committed to sharing data in accordance with NIH policy. For more details on how clinical trial data is shared, access the link to the NIH data sharing policy page.
Study at a glance
From the registry record, last updated by the sponsor on 4 Nov 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Randomized Phase II Trial of Enasidenib-Based Therapies Versus Cedazuridine-Decitabine in Higher-Risk IDH2-Mutated Myelodysplastic Syndrome: A MyeloMATCH Sub-Study
- ClinicalTrials.gov ID
- NCT06577441
- Acronym / sponsor ID
- NCI-2024-07049
- Conditions
- Myelodysplastic Syndrome
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 54 participants (estimated)
- Lead sponsor
- National Cancer Institute (NCI)US National Institutes of Health
- Study start
- 12 Jun 2025 (actual)
- Primary completion
- 1 Mar 2027 (estimated)
- Study completion
- 1 Mar 2027 (estimated)
- First posted
- 29 Aug 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.
This phase II MyeloMATCH treatment trial compares the usual treatment of cedazuridine-decitabine (ASTX727) to the combination treatment of ASTX727 and enasidenib in treating patients with higher-risk, IDH2-mutated myelodysplastic syndrome (MDS). ASTX727 is a combination of two drugs, decitabine and cedazuridine. Cedazuridine is in a class of medications called cytidine deaminase inhibitors. It prevents the breakdown of decitabine, making it more available in the body so that decitabine will have a greater effect. Decitabine is in a class of medications called hypomethylation agents. It works by helping the bone marrow produce normal blood cells and by killing abnormal cells in the bone marrow. Enasidenib is an enzyme inhibitor that may stop the growth of cells by blocking some of the enzymes needed for cell growth. Giving ASTX727 in combination with enasidenib may be effective in treating patients with higher-risk IDH2-mutated MDS.
Detailed description
PRIMARY OBJECTIVE:
- To compare the complete remission (CR) rate of enasidenib + decitabine and cedazuridine (ASTX727) and ASTX727 monotherapy in patients with higher-risk IDH2-mutated MDS using International Working Group 2023 (IWG2023) response criteria.
SECONDARY OBJECTIVES:
- To estimate the median event-free survival (EFS) at designated time point(s) for each treatment arm.
II. To estimate the median overall survival (OS) at designated time point(s) for each treatment arm.
III. To estimate the frequency and severity of toxicities with each regimen in this patient population.
IV. To estimate the median time to response for each treatment arm. V. To estimate the median duration of response for each treatment arm. VI. To estimate the IDH2 variant allele frequency (VAF) reduction for each treatment arm.
VII. To estimate the rate of allogeneic hematopoietic cell transplantation for each treatment arm.
VIII. To compare rates of partial response (PR), CR with limited count recovery (CRL), CR with partial count recovery (CRh), and hematologic improvement (HI) using IWG 2023 response criteria between treatment arms.
IX. To compare the measurable residual disease (MRD) kinetics by flow cytometry and next generation sequencing (NGS) at designated time point(s) at the end of cycle 4 \& 6 and to assess any correlation with clinical outcomes (e.g. CR, EFS, OS).
- To estimate the median time to transformation of MDS to acute myeloid leukemia (AML).
EXPLORATORY OBJECTIVES:
- To estimate CR rate, median EFS, and median OS in patients treated with ASTX727 monotherapy that crossover to the treatment arm with ASTX727 + enasidenib after 6 cycles if CR is not achieved.
II. To estimate CR rate, median EFS, and median OS for patients based on Molecular International Prognostic Scoring System (IPSS-M) prognostic risk score at diagnosis, stratified for score level.
III. To estimate concordance between centrally-performed molecular studies and cytogenetics to those done locally.
OUTLINE: Patients are randomized to 1 of 2 regimens.
REGIMEN 1: Patients receive ASTX727 orally (PO) once daily (QD) on days 1-5 of each cycle. Cycles repeat every 28 days in the absence of disease progression or unacceptable toxicity. Patients who do not achieve a CR, CRL, or CRh at the end of cycle 6 may cross-over to Regimen 2. Patients who experience CR, PR, or stable disease (SD) any time after 4 cycles of treatment may be reassessed in order to go to a higher myeloMATCH tier assignment or to Tier Advancement Pathway (TAP). Patients also undergo bone marrow biopsy and aspiration throughout the study. Patients may also undergo optional buccal swab on study, and/or optional additional bone marrow aspiration and blood sample collection on study and at disease progression.
REGIMEN 2: Patients receive ASTX727 PO QD on days 1-5 and enasidenib PO QD on days 1-28 of each cycle. Cycles repeat every 28 days in the absence of disease progression or unacceptable toxicity. Patients who experience CR, PR, or SD any time after 4 cycles of treatment may be reassessed in order to go to a higher myeloMATCH tier assignment or to TAP. Patients also undergo bone marrow biopsy and aspiration throughout the study. Patients may also undergo optional buccal swab on study, and/or optional additional bone marrow aspiration and blood sample collection on study and at disease progression.
After completion of study treatment, patients are followed up every 6 months for up to 5 years after registration.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Regimen 1 (ASTX727) | Active comparator | Procedure: Biospecimen Collection, Procedure: Bone Marrow Aspiration, Procedure: Bone Marrow Biopsy, Drug: Decitabine and CedazuridinePatients receive ASTX727 PO QD on days 1-5 of each cycle. Cycles repeat every 28 days in the absence of disease progression or unacceptable toxicity. Patients who do not achieve a CR, CRL, or CRh at the end of cycle 6 may cross-over to Regimen 2. Patients who experience CR, PR, or SD any time after 4 cycles of treatment may be reassessed in order to go to a higher myeloMATCH tier assignment or to TAP. Patients also undergo bone marrow biopsy and aspiration throughout the study. Patients may also undergo optional buccal swab on study, and/or optional additional bone marrow aspiration and blood sample collection on study and at disease progression. |
| Regimen 2 (ASTX727, enasidenib) | Experimental | Procedure: Biospecimen Collection, Procedure: Bone Marrow Aspiration, Procedure: Bone Marrow Biopsy, Drug: Decitabine and Cedazuridine, Drug: EnasidenibPatients receive ASTX727 PO QD on days 1-5 and enasidenib PO QD on days 1-28 of each cycle. Cycles repeat every 28 days in the absence of disease progression or unacceptable toxicity. Patients who experience CR, PR, or SD any time after 4 cycles of treatment may be reassessed in order to go to a higher myeloMATCH tier assignment or to TAP. Patients also undergo bone marrow biopsy and aspiration throughout the study. Patients may also undergo optional buccal swab on study, and/or optional additional bone marrow aspiration and blood sample collection on study and at disease progression. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (33 listed)
- GENERAL MYLEOMATCH REGISTRATION CRITERIA:
- REGISTRATION ELIGIBILITY CRITERIA (STEP 1): Patients must have a morphologically-confirmed diagnosis of MDS with a Revised International Prognostic Scoring System (IPSS-R) score ≥ 4.
- REGISTRATION ELIGIBILITY CRITERIA (STEP 1): Patients must have a detectable pathogenic IDH2 mutation based on the National Cancer Institute (NCI) Myeloid Panel.
- REGISTRATION ELIGIBILITY CRITERIA (STEP 1): No prior treatment with deoxyribonucleic acid (DNA) methyltransferase inhibitors (ASTX727, azacitidine, or decitabine).
- REGISTRATION ELIGIBILITY CRITERIA (STEP 1): Prior treatment with growth factors (ESA, granulocyte colony-stimulating factor [g-CSF], thrombopoietin [TPO] agonist) or luspatercept is allowed.
Key exclusion criteria (0 listed)
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 |
|---|---|---|
| Biospecimen CollectionAlso: Biological Sample Collection, Biospecimen Collected, Sample Collection, Specimen CollectionUndergo buccal swab and blood sample collection | Procedure or surgery | Regimen 1 (ASTX727), Regimen 2 (ASTX727, enasidenib) |
| Bone Marrow AspirationUndergo bone marrow aspiration | Procedure or surgery | Regimen 1 (ASTX727), Regimen 2 (ASTX727, enasidenib) |
| Bone Marrow BiopsyAlso: Biopsy of Bone Marrow, Biopsy, Bone MarrowUndergo bone marrow biopsy | Procedure or surgery | Regimen 1 (ASTX727), Regimen 2 (ASTX727, enasidenib) |
| Decitabine and CedazuridineAlso: ASTX 727, ASTX-727, ASTX727, C-DECGiven PO | Drug | Regimen 1 (ASTX727), Regimen 2 (ASTX727, enasidenib) |
| EnasidenibAlso: AG 221, AG-221, AG221, CC-90007 Free BaseGiven PO | Drug | Regimen 2 (ASTX727, enasidenib) |
Primary outcome
Complete response (CR) rate
Time frame: Up to 4 cycles of treatment
Will be assessed using the International Working Group 2023 (IWG2023) criteria. Will compare the CR rate between the two treatment arms to determine if patients treated with enasidenib + ASTX727 have a statistically significantly higher CR rate than patients treated with the ASTX727 monotherapy.
10 secondary outcomes
Event-free survival (EFS)
Time frame: Time from randomization to either a failure to achieve a CR, CR with limited count recovery (CRL), or CR with partial count recovery (CRh) after four cycles of treatment, relapse, or death due to any cause, assessed up to 18 months
Overall survival (OS)
Time frame: Time from randomization to death due to any cause, assessed up to 18 months
Time to response
Time frame: Time from randomization to documented response, assessed up to 5 years
Duration of response
Time frame: From patient first achieves a response until either progression or death, assessed up to 5 years
Incidence of adverse events
Time frame: Up to 4 weeks after completion of study treatment
Change in IDH2 variant allele frequency (VAF)
Time frame: Baseline to end of cycle 4 and 6
Allogeneic stem cell transplantation rate
Time frame: Up to 5 years
Measurable residual disease rate
Time frame: At the end of cycle 4 and 6
Cytogenetic and molecular features
Time frame: Up to 5 years
Time to transformation of myelodysplastic syndrome to acute myeloid leukemia (AML)
Time frame: From randomization to transformation to AML or death from any causes, assessed up to 5 years
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 4 Nov 2026.
- Individual participant dataSharing planned: Yes. NCI is committed to sharing data in accordance with NIH policy. For more details on how clinical trial data is shared, access the link to the NIH data sharing policy page.
Related trials for Myelodysplastic Syndrome
RecruitingPhase 2
Testing New Therapies for Patients With Acute Myeloid Leukemia Who Are Newly Diagnosed and Have Not Yet Started Treatment (A MyeloMATCH Treatment Trial)
Acute Myeloid Leukemia, Acute Myeloid Leukemia Arising From Previous Myelodysplastic/Myeloproliferative Neoplasm
Active, not recruitingPhase 1/2
Omacetaxine + Azacitidine in Untreated Patients With High Grade MDS
High Grade Myelodysplastic Syndromes
RecruitingPhase 1/2
Pacritinib, a Kinase Inhibitor of CSF1R, IRAK1, JAK2, and FLT3, in Adults and Pediatric Participants 12 Years of Age or Older With Myelodysplastic Syndromes or Myelodysplastic/Myeloproliferative Neoplasms
Myelodysplastic Syndromes
Active, not recruitingPhase 2
Clofarabine and Melphalan Before Donor Stem Cell Transplant in Treating Patients With Myelodysplasia, Acute Leukemia in Remission, or Chronic Myelomonocytic Leukemia
Adult Acute Lymphoblastic Leukemia in Remission, Acute Myeloid Leukemia Arising From Previous Myelodysplastic Syndrome
Not yet recruitingPhase 1
ATRN-119 in Combination With Decitabine in Patients With TP53-Mutated AML or Higher-Risk MDS
Acute Myeloid Leukemia, High-risk Myelodysplastic Syndrome
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.
UCI Health - Chao Family Comprehensive Cancer Center and Ambulatory Care
Irvine, United States
UC Irvine Health/Chao Family Comprehensive Cancer Center
Orange, United States
Anand A Patel principal investigator
Alliance for Clinical Trials in Oncology
151 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Biospecimen Collection, Bone Marrow Aspiration and Bone Marrow Biopsy in people with Myelodysplastic Syndrome, run by National Cancer Institute (NCI) at 151 sites in 2 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 0 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 4 Nov 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 151 sites in United States, Puerto Rico. 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 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?
Each site lists its own contact: choose the nearest one under Trial locations. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Complete response (CR) rate (time frame: Up to 4 cycles of treatment). 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 4 Nov 2026.