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NCT06577441PHASE2Recruiting

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)

National Cancer Institute (NCI)

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

Inclusion Criteria: * 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 * 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.

Interventions

PROCEDURE

Biospecimen Collection

PROCEDURE

Bone Marrow Aspiration

PROCEDURE

Bone Marrow Biopsy

DRUG

Decitabine and Cedazuridine

DRUG

Enasidenib

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Conditions

Myelodysplastic Syndrome

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