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)
Start Date
6/12/2025
Completion Date
3/1/2027
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. 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: I. 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: I. 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). X. To estimate the median time to transformation of MDS to acute myeloid leukemia (AML). EXPLORATORY OBJECTIVES: I. 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.
Eligibility Criteria
Age Range: 18 years to No maximum
Interventions
Biospecimen Collection
Bone Marrow Aspiration
Bone Marrow Biopsy
Decitabine and Cedazuridine
Enasidenib
Conditions
Locations
Alta Bates Summit Medical Center-Herrick Campus
Berkeley, California 94704
United States
UCI Health - Chao Family Comprehensive Cancer Center and Ambulatory Care
Irvine, California 92612
United States
UC Irvine Health/Chao Family Comprehensive Cancer Center
Orange, California 92868
United States
Mills Health Center
San Mateo, California 94401
United States
UF Health Cancer Institute - Gainesville
Gainesville, Florida 32610
United States
Miami Cancer Institute
Miami, Florida 33176
United States
Memorial Hospital West
Pembroke Pines, Florida 33028
United States
Phoebe Putney Memorial Hospital
Albany, Georgia 31701
United States
Saint Luke's Cancer Institute - Boise
Boise, Idaho 83712
United States
Kootenai Health - Coeur d'Alene
Coeur d'Alene, Idaho 83814
United States
Saint Luke's Cancer Institute - Fruitland
Fruitland, Idaho 83619
United States
Saint Luke's Cancer Institute - Meridian
Meridian, Idaho 83642
United States
Saint Alphonsus Cancer Care Center-Nampa
Nampa, Idaho 83687
United States
Saint Luke's Cancer Institute - Nampa
Nampa, Idaho 83687
United States
Kootenai Clinic Cancer Services - Post Falls
Post Falls, Idaho 83854
United States
Kootenai Clinic Cancer Services - Sandpoint
Sandpoint, Idaho 83864
United States
Illinois CancerCare-Bloomington
Bloomington, Illinois 61704
United States
Illinois CancerCare-Canton
Canton, Illinois 61520
United States
Illinois CancerCare-Carthage
Carthage, Illinois 62321
United States
Northwestern University
Chicago, Illinois 60611
United States
Swedish Covenant Hospital
Chicago, Illinois 60625
United States
University of Chicago Comprehensive Cancer Center
Chicago, Illinois 60637
United States
Carle at The Riverfront
Danville, Illinois 61832
United States
Cancer Care Specialists of Illinois - Decatur
Decatur, Illinois 62526
United States
Decatur Memorial Hospital
Decatur, Illinois 62526
United States
Northwestern Medicine Cancer Center Kishwaukee
DeKalb, Illinois 60115
United States
Illinois CancerCare-Dixon
Dixon, Illinois 61021
United States
Carle Physician Group-Effingham
Effingham, Illinois 62401
United States
Crossroads Cancer Center
Effingham, Illinois 62401
United States
Illinois CancerCare-Eureka
Eureka, Illinois 61530
United States
NorthShore University HealthSystem-Evanston Hospital
Evanston, Illinois 60201
United States
Illinois CancerCare-Galesburg
Galesburg, Illinois 61401
United States
Northwestern Medicine Cancer Center Delnor
Geneva, Illinois 60134
United States
NorthShore University HealthSystem-Glenbrook Hospital
Glenview, Illinois 60026
United States
Northwestern Medicine Glenview Outpatient Center
Glenview, Illinois 60026
United States
Northwestern Medicine Grayslake Outpatient Center
Grayslake, Illinois 60030
United States
NorthShore University HealthSystem-Highland Park Hospital
Highland Park, Illinois 60035
United States
Illinois CancerCare-Kewanee Clinic
Kewanee, Illinois 61443
United States
Northwestern Medicine Lake Forest Hospital
Lake Forest, Illinois 60045
United States
Illinois CancerCare-Macomb
Macomb, Illinois 61455
United States
Carle Physician Group-Mattoon/Charleston
Mattoon, Illinois 61938
United States
Loyola University Medical Center
Maywood, Illinois 60153
United States
UC Comprehensive Cancer Center at Silver Cross
New Lenox, Illinois 60451
United States
Cancer Care Center of O'Fallon
O'Fallon, Illinois 62269
United States
HSHS Saint Elizabeth's Hospital
O'Fallon, Illinois 62269
United States
Northwestern Medicine Orland Park
Orland Park, Illinois 60462
United States
University of Chicago Medicine-Orland Park
Orland Park, Illinois 60462
United States
Illinois CancerCare-Ottawa Clinic
Ottawa, Illinois 61350
United States
Illinois CancerCare-Pekin
Pekin, Illinois 61554
United States
Illinois CancerCare-Peoria
Peoria, Illinois 61615
United States
Illinois CancerCare-Peru
Peru, Illinois 61354
United States
Illinois CancerCare-Princeton
Princeton, Illinois 61356
United States
Southern Illinois University School of Medicine
Springfield, Illinois 62702
United States
Springfield Clinic
Springfield, Illinois 62702
United States
Springfield Memorial Hospital
Springfield, Illinois 62781
United States
Carle Cancer Center
Urbana, Illinois 61801
United States
Northwestern Medicine Cancer Center Warrenville
Warrenville, Illinois 60555
United States
Illinois CancerCare - Washington
Washington, Illinois 61571
United States
UChicago Medicine Northwest Indiana
Crown Point, Indiana 46307
United States
UI Health Care Mission Cancer and Blood - Ankeny Clinic
Ankeny, Iowa 50023
United States
UI Health Care Mission Cancer and Blood - West Des Moines Clinic
Clive, Iowa 50325
United States
Iowa Methodist Medical Center
Des Moines, Iowa 50309
United States
UI Health Care Mission Cancer and Blood - Des Moines Clinic
Des Moines, Iowa 50309
United States
Mercy Medical Center - Des Moines
Des Moines, Iowa 50314
United States
UI Health Care Mission Cancer and Blood - Laurel Clinic
Des Moines, Iowa 50314
United States
University of Iowa/Holden Comprehensive Cancer Center
Iowa City, Iowa 52242
United States
UI Healthcare Mission Cancer and Blood - Pella
Pella, Iowa 50219
United States
UI Health Care Mission Cancer and Blood - Waukee Clinic
Waukee, Iowa 50263
United States
University of Kansas Clinical Research Center
Fairway, Kansas 66205
United States
University of Kansas Cancer Center
Kansas City, Kansas 66160
United States
University of Kansas Hospital-Westwood Cancer Center
Westwood, Kansas 66205
United States
The James Graham Brown Cancer Center at University of Louisville
Louisville, Kentucky 40202
United States
UofL Health Medical Center Northeast
Louisville, Kentucky 40245
United States
LSU Health Baton Rouge-North Clinic
Baton Rouge, Louisiana 70805
United States
Our Lady of the Lake Physician Group
Baton Rouge, Louisiana 70808
United States
Our Lady of The Lake
Baton Rouge, Louisiana 70808
United States
Walter Reed National Military Medical Center
Bethesda, Maryland 20889-5600
United States
Tufts Medical Center
Boston, Massachusetts 02111
United States
Trinity Health IHA Medical Group Hematology Oncology - Brighton
Brighton, Michigan 48114
United States
Trinity Health IHA Medical Group Hematology Oncology - Canton
Canton, Michigan 48188
United States
Trinity Health IHA Medical Group Hematology Oncology - Chelsea Hospital
Chelsea, Michigan 48118
United States
Cancer Hematology Centers - Flint
Flint, Michigan 48503
United States
Genesee Hematology Oncology PC
Flint, Michigan 48503
United States
Genesys Hurley Cancer Institute
Flint, Michigan 48503
United States
Trinity Health Saint Mary Mercy Livonia Hospital
Livonia, Michigan 48154
United States
Trinity Health Saint Joseph Mercy Oakland Hospital
Pontiac, Michigan 48341
United States
Trinity Health IHA Medical Group Hematology Oncology Ann Arbor Campus
Ypsilanti, Michigan 48197
United States
Essentia Health Saint Joseph's Medical Center
Brainerd, Minnesota 56401
United States
Essentia Health - Deer River Clinic
Deer River, Minnesota 56636
United States
Essentia Health Cancer Center
Duluth, Minnesota 55805
United States
Essentia Health Hibbing Clinic
Hibbing, Minnesota 55746
United States
Hennepin County Medical Center
Minneapolis, Minnesota 55415
United States
Essentia Health Sandstone
Sandstone, Minnesota 55072
United States
Essentia Health Virginia Clinic
Virginia, Minnesota 55792
United States
Parkland Health Center - Farmington
Farmington, Missouri 63640
United States
Sainte Genevieve County Memorial Hospital
Sainte Genevieve, Missouri 63670
United States
Missouri Baptist Medical Center
St Louis, Missouri 63131
United States
Missouri Baptist Sullivan Hospital
Sullivan, Missouri 63080
United States
BJC Outpatient Center at Sunset Hills
Sunset Hills, Missouri 63127
United States
Community Hospital of Anaconda
Anaconda, Montana 59711
United States
Billings Clinic Cancer Center
Billings, Montana 59101
United States
Bozeman Health Deaconess Hospital
Bozeman, Montana 59715
United States
Benefis Sletten Cancer Institute
Great Falls, Montana 59405
United States
Logan Health Medical Center
Kalispell, Montana 59901
United States
Community Medical Center
Missoula, Montana 59804
United States
Cooperman Barnabas Medical Center
Livingston, New Jersey 07039
United States
Monmouth Medical Center
Long Branch, New Jersey 07740
United States
Rutgers Cancer Institute of New Jersey
New Brunswick, New Jersey 08903
United States
Community Medical Center
Toms River, New Jersey 08755
United States
University of New Mexico Cancer Center
Albuquerque, New Mexico 87106
United States
Roswell Park Cancer Institute
Buffalo, New York 14263
United States
Stony Brook University Medical Center
Stony Brook, New York 11794
United States
Duke University Medical Center
Durham, North Carolina 27710
United States
East Carolina University
Greenville, North Carolina 27834
United States
Essentia Health Cancer Center-South University Clinic
Fargo, North Dakota 58103
United States
Ohio State University Comprehensive Cancer Center
Columbus, Ohio 43210
United States
University of Oklahoma Health Sciences Center
Oklahoma City, Oklahoma 73104
United States
Geisinger Medical Center
Danville, Pennsylvania 17822
United States
UPMC Hillman Cancer Center
Pittsburgh, Pennsylvania 15232
United States
Geisinger Wyoming Valley/Henry Cancer Center
Wilkes-Barre, Pennsylvania 18711
United States
Prisma Health Cancer Institute - Spartanburg
Boiling Springs, South Carolina 29316
United States
Prisma Health Cancer Institute - Easley
Easley, South Carolina 29640
United States
Prisma Health Cancer Institute - Butternut
Greenville, South Carolina 29605
United States
Prisma Health Cancer Institute - Faris
Greenville, South Carolina 29605
United States
Prisma Health Cancer Institute - Eastside
Greenville, South Carolina 29615
United States
Prisma Health Cancer Institute - Greer
Greer, South Carolina 29650
United States
Prisma Health Cancer Institute - Seneca
Seneca, South Carolina 29672
United States
University of Tennessee - Knoxville
Knoxville, Tennessee 37920
United States
Huntsman Cancer Institute/University of Utah
Salt Lake City, Utah 84112
United States
VCU Massey Comprehensive Cancer Center
Richmond, Virginia 23298
United States
Swedish Cancer Institute-Edmonds
Edmonds, Washington 98026
United States
Swedish Cancer Institute-Issaquah
Issaquah, Washington 98029
United States
Swedish Medical Center-First Hill
Seattle, Washington 98122
United States
Duluth Clinic Ashland
Ashland, Wisconsin 54806
United States
Saint Vincent Hospital Cancer Center Green Bay
Green Bay, Wisconsin 54301
United States
Saint Vincent Hospital Cancer Center at Saint Mary's
Green Bay, Wisconsin 54303
United States
Gundersen Lutheran Medical Center
La Crosse, Wisconsin 54601
United States
William S Middleton VA Medical Center
Madison, Wisconsin 53705
United States
Medical College of Wisconsin
Milwaukee, Wisconsin 53226
United States
Marshfield Medical Center-River Region at Stevens Point
Stevens Point, Wisconsin 54482
United States
Marshfield Medical Center - Weston
Weston, Wisconsin 54476
United States
Centro Comprensivo de Cancer de UPR
San Juan, 00927
Puerto Rico
San Juan City Hospital
San Juan, 00936
Puerto Rico