New combo therapy aims to boost remission in rare blood cancer
NCT ID NCT06577441
First seen Jun 27, 2026 · Last updated Sep 18, 2026 · Updated 17 times
Summary
This study tests whether adding enasidenib to standard treatment (ASTX727) improves outcomes for people with a high-risk form of myelodysplastic syndrome (MDS) that has a specific gene mutation (IDH2). About 54 adults who have not had prior cancer therapy will be randomly assigned to receive either the standard drug alone or the combination. The main goal is to see if the combination leads to more complete remissions.
This is an AI summary of the original study and may miss details. Read our disclaimer.
Study facts
What this study's own registry entry says, in plain language.
- Phase
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Phase 2
Tests whether the treatment actually works, and watches for side effects, in a larger group.
- Participants
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About 54 people
The number the study aims to enrol. It can still change while the study runs.
- Started
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Jun 2025
- Expected to finish
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Mar 2027
An estimate. End dates often move.
- Lead sponsor
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A government research agency
The lead sponsor is the US National Institutes of Health.
Who can take part
This study's own entry requirements. Only the study team can say for certain whether you qualify.
- Ages
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18 years and older
- Sex
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Anyone
- Healthy volunteers
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Not accepted
This study is not open to healthy volunteers. The entry requirements below say who it is open to.
Show the full entry requirements Hide the full entry requirements
Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.
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.
Get updates
Get notified about this study
Sign up to get updates when this study changes or when new studies for Myelodysplastic syndrome are added.
Genom att skicka in godkänner du våra Användarvillkor
Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
How to take part
Only the study team decides who joins. These are the ways to reach them.
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The study's own enquiry address
This study publishes an address for enquiries. See it below .
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The places running it
142 sites in 2 countries. The list below names each one and where it is.
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The official record
ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.
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A doctor treating you
A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.
Contacts and locations
Show contact details
Enter your email to view the contact information for this study.
Genom att skicka in godkänner du våra Användarvillkor
Locations
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Alta Bates Summit Medical Center-Herrick Campus
ACTIVE_NOT_RECRUITINGBerkeley, California, 94704, United States
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Benefis Sletten Cancer Institute
RECRUITINGGreat Falls, Montana, 59405, United States
Contact Email: •••••@•••••
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Billings Clinic Cancer Center
RECRUITINGBillings, Montana, 59101, United States
Contact Email: •••••@•••••
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Bozeman Health Deaconess Hospital
RECRUITINGBozeman, Montana, 59715, United States
Contact Email: •••••@•••••
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Cancer Care Center of O'Fallon
RECRUITINGO'Fallon, Illinois, 62269, United States
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Cancer Care Specialists of Illinois - Decatur
RECRUITINGDecatur, Illinois, 62526, United States
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Cancer Hematology Centers - Flint
RECRUITINGFlint, Michigan, 48503, United States
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Carle Cancer Center
RECRUITINGUrbana, Illinois, 61801, United States
Contact Email: •••••@•••••
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Carle Physician Group-Effingham
RECRUITINGEffingham, Illinois, 62401, United States
Contact Email: •••••@•••••
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Carle Physician Group-Mattoon/Charleston
RECRUITINGMattoon, Illinois, 61938, United States
Contact Email: •••••@•••••
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Carle at The Riverfront
RECRUITINGDanville, Illinois, 61832, United States
Contact Email: •••••@•••••
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Centro Comprensivo de Cancer de UPR
RECRUITINGSan Juan, 00927, Puerto Rico
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Community Hospital of Anaconda
RECRUITINGAnaconda, Montana, 59711, United States
Contact Email: •••••@•••••
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Community Medical Center
RECRUITINGMissoula, Montana, 59804, United States
Contact Email: •••••@•••••
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Community Medical Center
RECRUITINGToms River, New Jersey, 08755, United States
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Cooperman Barnabas Medical Center
RECRUITINGLivingston, New Jersey, 07039, United States
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Crossroads Cancer Center
RECRUITINGEffingham, Illinois, 62401, United States
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Dartmouth Hitchcock Medical Center/Dartmouth Cancer Center
RECRUITINGLebanon, New Hampshire, 03756, United States
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Decatur Memorial Hospital
RECRUITINGDecatur, Illinois, 62526, United States
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Duke University Medical Center
RECRUITINGDurham, North Carolina, 27710, United States
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Duluth Clinic Ashland
RECRUITINGAshland, Wisconsin, 54806, United States
Contact Email: •••••@•••••
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East Carolina University
RECRUITINGGreenville, North Carolina, 27834, United States
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Edward Hines Jr VA Hospital
RECRUITINGHines, Illinois, 60141, United States
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Essentia Health - Deer River Clinic
RECRUITINGDeer River, Minnesota, 56636, United States
Contact Email: •••••@•••••
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Essentia Health Cancer Center
RECRUITINGDuluth, Minnesota, 55805, United States
Contact Email: •••••@•••••
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Essentia Health Cancer Center-South University Clinic
RECRUITINGFargo, North Dakota, 58103, United States
Contact Email: •••••@•••••
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Essentia Health Hibbing Clinic
RECRUITINGHibbing, Minnesota, 55746, United States
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Essentia Health Saint Joseph's Medical Center
RECRUITINGBrainerd, Minnesota, 56401, United States
Contact Email: •••••@•••••
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Essentia Health Sandstone
RECRUITINGSandstone, Minnesota, 55072, United States
Contact Email: •••••@•••••
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Essentia Health Virginia Clinic
RECRUITINGVirginia, Minnesota, 55792, United States
Contact Email: •••••@•••••
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Geisinger Medical Center
RECRUITINGDanville, Pennsylvania, 17822, United States
Contact Email: •••••@•••••
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Geisinger Wyoming Valley/Henry Cancer Center
RECRUITINGWilkes-Barre, Pennsylvania, 18711, United States
Contact Email: •••••@•••••
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Genesee Hematology Oncology PC
SUSPENDEDFlint, Michigan, 48503, United States
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Genesys Hurley Cancer Institute
RECRUITINGFlint, Michigan, 48503, United States
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Gundersen Lutheran Medical Center
RECRUITINGLa Crosse, Wisconsin, 54601, United States
Contact Email: •••••@•••••
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HSHS Saint Elizabeth's Hospital
RECRUITINGO'Fallon, Illinois, 62269, United States
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Hennepin County Medical Center
RECRUITINGMinneapolis, Minnesota, 55415, United States
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Huntsman Cancer Institute/University of Utah
RECRUITINGSalt Lake City, Utah, 84112, United States
Contact Email: •••••@•••••
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Illinois CancerCare - Washington
RECRUITINGWashington, Illinois, 61571, United States
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Illinois CancerCare-Bloomington
RECRUITINGBloomington, Illinois, 61704, United States
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Illinois CancerCare-Canton
RECRUITINGCanton, Illinois, 61520, United States
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Illinois CancerCare-Carthage
RECRUITINGCarthage, Illinois, 62321, United States
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Illinois CancerCare-Dixon
RECRUITINGDixon, Illinois, 61021, United States
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Illinois CancerCare-Eureka
RECRUITINGEureka, Illinois, 61530, United States
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Illinois CancerCare-Galesburg
RECRUITINGGalesburg, Illinois, 61401, United States
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Illinois CancerCare-Kewanee Clinic
RECRUITINGKewanee, Illinois, 61443, United States
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Illinois CancerCare-Macomb
RECRUITINGMacomb, Illinois, 61455, United States
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Illinois CancerCare-Ottawa Clinic
RECRUITINGOttawa, Illinois, 61350, United States
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Illinois CancerCare-Pekin
RECRUITINGPekin, Illinois, 61554, United States
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Illinois CancerCare-Peoria
RECRUITINGPeoria, Illinois, 61615, United States
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Illinois CancerCare-Peru
RECRUITINGPeru, Illinois, 61354, United States
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Illinois CancerCare-Princeton
RECRUITINGPrinceton, Illinois, 61356, United States
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Iowa Methodist Medical Center
RECRUITINGDes Moines, Iowa, 50309, United States
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Kootenai Clinic Cancer Services - Post Falls
RECRUITINGPost Falls, Idaho, 83854, United States
Contact Email: •••••@•••••
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Kootenai Clinic Cancer Services - Sandpoint
RECRUITINGSandpoint, Idaho, 83864, United States
Contact Email: •••••@•••••
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Kootenai Health - Coeur d'Alene
RECRUITINGCoeur d'Alene, Idaho, 83814, United States
Contact Email: •••••@•••••
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LSU Health Baton Rouge-North Clinic
RECRUITINGBaton Rouge, Louisiana, 70805, United States
Contact Email: •••••@•••••
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Logan Health Medical Center
RECRUITINGKalispell, Montana, 59901, United States
Contact Email: •••••@•••••
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Loyola University Medical Center
RECRUITINGMaywood, Illinois, 60153, United States
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Marshfield Medical Center - Weston
RECRUITINGWeston, Wisconsin, 54476, United States
Contact Email: •••••@•••••
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Marshfield Medical Center-River Region at Stevens Point
RECRUITINGStevens Point, Wisconsin, 54482, United States
Contact Email: •••••@•••••
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Medical College of Wisconsin
RECRUITINGMilwaukee, Wisconsin, 53226, United States
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Memorial Hospital West
RECRUITINGPembroke Pines, Florida, 33028, United States
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Mercy Medical Center - Des Moines
RECRUITINGDes Moines, Iowa, 50314, United States
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Miami Cancer Institute
RECRUITINGMiami, Florida, 33176, United States
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Mills Health Center
ACTIVE_NOT_RECRUITINGSan Mateo, California, 94401, United States
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Missouri Baptist Medical Center
RECRUITINGSt Louis, Missouri, 63131, United States
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Missouri Baptist Sullivan Hospital
RECRUITINGSullivan, Missouri, 63080, United States
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Monmouth Medical Center
RECRUITINGLong Branch, New Jersey, 07740, United States
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NorthShore University HealthSystem-Evanston Hospital
RECRUITINGEvanston, Illinois, 60201, United States
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NorthShore University HealthSystem-Glenbrook Hospital
RECRUITINGGlenview, Illinois, 60026, United States
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NorthShore University HealthSystem-Highland Park Hospital
RECRUITINGHighland Park, Illinois, 60035, United States
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Northwestern Medicine Cancer Center Delnor
RECRUITINGGeneva, Illinois, 60134, United States
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Northwestern Medicine Cancer Center Kishwaukee
RECRUITINGDeKalb, Illinois, 60115, United States
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Northwestern Medicine Cancer Center Warrenville
RECRUITINGWarrenville, Illinois, 60555, United States
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Northwestern Medicine Glenview Outpatient Center
RECRUITINGGlenview, Illinois, 60026, United States
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Northwestern Medicine Grayslake Outpatient Center
RECRUITINGGrayslake, Illinois, 60030, United States
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Northwestern Medicine Lake Forest Hospital
RECRUITINGLake Forest, Illinois, 60045, United States
Contact Email: •••••@•••••
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Northwestern Medicine Orland Park
RECRUITINGOrland Park, Illinois, 60462, United States
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Northwestern University
RECRUITINGChicago, Illinois, 60611, United States
Contact Email: •••••@•••••
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Ohio State University Comprehensive Cancer Center
RECRUITINGColumbus, Ohio, 43210, United States
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Our Lady of The Lake
RECRUITINGBaton Rouge, Louisiana, 70808, United States
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Our Lady of the Lake Physician Group
RECRUITINGBaton Rouge, Louisiana, 70808, United States
Contact Email: •••••@•••••
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Parkland Health Center - Farmington
RECRUITINGFarmington, Missouri, 63640, United States
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Phoebe Putney Memorial Hospital
RECRUITINGAlbany, Georgia, 31701, United States
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Prisma Health Cancer Institute - Butternut
RECRUITINGGreenville, South Carolina, 29605, United States
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Prisma Health Cancer Institute - Easley
RECRUITINGEasley, South Carolina, 29640, United States
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Prisma Health Cancer Institute - Eastside
RECRUITINGGreenville, South Carolina, 29615, United States
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Prisma Health Cancer Institute - Faris
RECRUITINGGreenville, South Carolina, 29605, United States
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Prisma Health Cancer Institute - Greer
RECRUITINGGreer, South Carolina, 29650, United States
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Prisma Health Cancer Institute - Seneca
RECRUITINGSeneca, South Carolina, 29672, United States
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Prisma Health Cancer Institute - Spartanburg
RECRUITINGBoiling Springs, South Carolina, 29316, United States
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Roswell Park Cancer Institute
RECRUITINGBuffalo, New York, 14263, United States
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Rutgers Cancer Institute of New Jersey
RECRUITINGNew Brunswick, New Jersey, 08903, United States
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Saint Alphonsus Cancer Care Center-Nampa
RECRUITINGNampa, Idaho, 83687, United States
Contact Email: •••••@•••••
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Saint Luke's Cancer Institute - Boise
RECRUITINGBoise, Idaho, 83712, United States
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Saint Luke's Cancer Institute - Fruitland
RECRUITINGFruitland, Idaho, 83619, United States
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Saint Luke's Cancer Institute - Meridian
RECRUITINGMeridian, Idaho, 83642, United States
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Saint Luke's Cancer Institute - Nampa
RECRUITINGNampa, Idaho, 83687, United States
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Saint Vincent Hospital Cancer Center Green Bay
RECRUITINGGreen Bay, Wisconsin, 54301, United States
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Saint Vincent Hospital Cancer Center at Saint Mary's
RECRUITINGGreen Bay, Wisconsin, 54303, United States
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Sainte Genevieve County Memorial Hospital
RECRUITINGSainte Genevieve, Missouri, 63670, United States
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San Juan City Hospital
RECRUITINGSan Juan, 00936, Puerto Rico
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Southern Illinois University School of Medicine
RECRUITINGSpringfield, Illinois, 62702, United States
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Springfield Clinic
RECRUITINGSpringfield, Illinois, 62702, United States
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Springfield Memorial Hospital
RECRUITINGSpringfield, Illinois, 62781, United States
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Stony Brook University Medical Center
RECRUITINGStony Brook, New York, 11794, United States
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Swedish Cancer Institute-Edmonds
RECRUITINGEdmonds, Washington, 98026, United States
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Swedish Cancer Institute-Issaquah
RECRUITINGIssaquah, Washington, 98029, United States
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Swedish Covenant Hospital
RECRUITINGChicago, Illinois, 60625, United States
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Swedish Medical Center-First Hill
RECRUITINGSeattle, Washington, 98122, United States
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The James Graham Brown Cancer Center at University of Louisville
SUSPENDEDLouisville, Kentucky, 40202, United States
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Trinity Health IHA Medical Group Hematology Oncology - Brighton
RECRUITINGBrighton, Michigan, 48114, United States
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Trinity Health IHA Medical Group Hematology Oncology - Canton
RECRUITINGCanton, Michigan, 48188, United States
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Trinity Health IHA Medical Group Hematology Oncology - Chelsea Hospital
RECRUITINGChelsea, Michigan, 48118, United States
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Trinity Health IHA Medical Group Hematology Oncology Ann Arbor Campus
RECRUITINGYpsilanti, Michigan, 48197, United States
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Trinity Health Saint Joseph Mercy Oakland Hospital
RECRUITINGPontiac, Michigan, 48341, United States
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Trinity Health Saint Mary Mercy Livonia Hospital
RECRUITINGLivonia, Michigan, 48154, United States
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Tufts Medical Center
RECRUITINGBoston, Massachusetts, 02111, United States
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UC Comprehensive Cancer Center at Silver Cross
RECRUITINGNew Lenox, Illinois, 60451, United States
Contact Email: •••••@•••••
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UC Irvine Health/Chao Family Comprehensive Cancer Center
RECRUITINGOrange, California, 92868, United States
Contact Email: •••••@•••••
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UCI Health - Chao Family Comprehensive Cancer Center and Ambulatory Care
RECRUITINGIrvine, California, 92612, United States
Contact Email: •••••@•••••
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UChicago Medicine Northwest Indiana
RECRUITINGCrown Point, Indiana, 46307, United States
Contact Email: •••••@•••••
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UF Health Cancer Institute - Gainesville
RECRUITINGGainesville, Florida, 32610, United States
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UI Health Care Mission Cancer and Blood - Ankeny Clinic
RECRUITINGAnkeny, Iowa, 50023, United States
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UI Health Care Mission Cancer and Blood - Des Moines Clinic
RECRUITINGDes Moines, Iowa, 50309, United States
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UI Health Care Mission Cancer and Blood - Laurel Clinic
RECRUITINGDes Moines, Iowa, 50314, United States
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UI Health Care Mission Cancer and Blood - Waukee Clinic
RECRUITINGWaukee, Iowa, 50263, United States
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UI Health Care Mission Cancer and Blood - West Des Moines Clinic
RECRUITINGClive, Iowa, 50325, United States
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UI Healthcare Mission Cancer and Blood - Pella
RECRUITINGPella, Iowa, 50219, United States
Contact Email: •••••@•••••
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UPMC Hillman Cancer Center
RECRUITINGPittsburgh, Pennsylvania, 15232, United States
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University of Chicago Comprehensive Cancer Center
RECRUITINGChicago, Illinois, 60637, United States
Contact Email: •••••@•••••
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University of Chicago Medicine-Orland Park
RECRUITINGOrland Park, Illinois, 60462, United States
Contact Email: •••••@•••••
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University of Iowa/Holden Comprehensive Cancer Center
RECRUITINGIowa City, Iowa, 52242, United States
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University of Kansas Cancer Center
RECRUITINGKansas City, Kansas, 66160, United States
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University of Kansas Clinical Research Center
RECRUITINGFairway, Kansas, 66205, United States
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University of Kansas Hospital-Westwood Cancer Center
RECRUITINGWestwood, Kansas, 66205, United States
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University of New Mexico Cancer Center
RECRUITINGAlbuquerque, New Mexico, 87106, United States
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University of Oklahoma Health Sciences Center
RECRUITINGOklahoma City, Oklahoma, 73104, United States
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University of Tennessee - Knoxville
RECRUITINGKnoxville, Tennessee, 37920, United States
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UofL Health Medical Center Northeast
RECRUITINGLouisville, Kentucky, 40245, United States
Contact Email: •••••@•••••
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VCU Massey Comprehensive Cancer Center
RECRUITINGRichmond, Virginia, 23298, United States
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Walter Reed National Military Medical Center
RECRUITINGBethesda, Maryland, 20889-5600, United States
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William S Middleton VA Medical Center
RECRUITINGMadison, Wisconsin, 53705, United States
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