Can tailored chemotherapy tame leukemia in down syndrome?

NCT ID NCT02521493

Disease control Sponsor: Children's Oncology Group Source: ClinicalTrials.gov ↗

First seen Aug 19, 2026 · Last updated Aug 20, 2026 · Updated 1 time

Summary

This phase 3 trial is testing whether adjusting chemotherapy based on a child's early response can improve outcomes for acute myeloid leukemia (AML) or myelodysplastic syndrome in children with Down syndrome. After the first round of treatment, patients are placed into risk groups, and those at higher risk receive more intensive therapy while standard-risk patients get less, aiming to boost effectiveness and reduce side effects. The study enrolls children with Down syndrome who are newly diagnosed with these blood cancers.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance: chemotherapy (including asparaginase) tailored to response
What this could lead to: If successful, this approach could improve survival and reduce side effects for children with Down syndrome and AML, offering a more personalized treatment strategy.
What could go wrong: The trial is still in progress, and results are not yet known. Chemotherapy carries risks of infection, bleeding, and organ damage, and the response-based approach may not benefit all patients.

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

Phase 3

Large-scale testing in a bigger group. Usually the last step before a treatment can be approved.

Participants

280 people

The number who actually took part.

Started

Dec 2015

Expected to finish

Jun 2027

An estimate. End dates often move.

Lead sponsor

A research network

The lead sponsor is a research network or cooperative group.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

91 days to 3 years

Sex

Anyone

Healthy volunteers

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

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Inclusion Criteria: * Patients must have constitutional trisomy 21 (Down syndrome) or trisomy 21 mosaicism (by karyotype or fluorescence in situ hybridization \[FISH\]) * Patient has one of the following: * Patient has previously untreated de novo AML and meets the criteria for AML with \>= 20% bone marrow blasts as set out in the World Health Organization (WHO) Myeloid Neoplasm classification * Attempts to obtain bone marrow either by aspirate or biopsy must be made unless clinically prohibitive; in cases where it is clinically prohibitive, peripheral blood with an excess of 20% blasts and in which adequate flow cytometric and cytogenetics/FISH testing is feasible can be substituted for the marrow exam at diagnosis * Patient has cytopenias and/or bone marrow blasts but does not meet the criteria for the diagnosis of AML (WHO Myeloid Neoplasm classification) because of \< 20% marrow blasts and meets the criteria for a diagnosis of myelodysplastic syndrome (MDS) * For patients who do not meet criteria for AML or MDS as outlined above; patient has a history of transient myeloproliferative disorder (which may or may not have required chemotherapy intervention and: * Is \> 8 weeks since resolution of transient myeloproliferative disease (TMD) with \>= 5% blasts, OR * Has an increasing blast count (\>= 5%) in serial bone marrow aspirates performed at least 4 weeks apart * Children who have previously received chemotherapy, radiation therapy or any anti-leukemic therapy are not eligible for this protocol, with the exception of cytarabine for the treatment of TMD * There are no minimal organ function requirements for enrollment on this study * Note: Previous cardiac repair with sufficient cardiac function is not an exclusion criteria * Each patient's parents or legal guardians must sign a written informed consent * All institutional, Food and Drug Administration (FDA), and National Cancer Institute (NCI) requirements for human subjects research must be met Exclusion Criteria: * Patients with promyelocytic leukemia (French-American-British \[FAB\] M3) * Prior therapy * Patients =\< 30 days from the last dose of cytarabine used for treatment of TMD

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Conditions

The condition(s) this trial relates to.

acute myeloid leukemia Down syndrome myelodysplastic syndrome Myelodysplastic Syndromes myeloid leukemia associated with down syndrome Myeloproliferative Disorders myeloproliferative neoplasm

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Acute myeloid leukemia Down syndrome Myelodysplastic syndrome Myeloid leukemia associated with Down syndrome Myeloproliferative neoplasm

Contacts and locations

Locations

AdventHealth Orlando

Orlando, Florida, 32803, United States
Advocate Children's Hospital-Oak Lawn

Oak Lawn, Illinois, 60453, United States
Advocate Children's Hospital-Park Ridge

Park Ridge, Illinois, 60068, United States
Albany Medical Center

Albany, New York, 12208, United States
Alfred I duPont Hospital for Children

Wilmington, Delaware, 19803, United States
Alliance for Childhood Diseases/Cure 4 the Kids Foundation

Las Vegas, Nevada, 89135, United States
Arkansas Children's Hospital

Little Rock, Arkansas, 72202-3591, United States
Arnold Palmer Hospital for Children

Orlando, Florida, 32806, United States
Ascension Saint Vincent Indianapolis Hospital

Indianapolis, Indiana, 46260, United States
BI-LO Charities Children's Cancer Center

Greenville, South Carolina, 29605, United States
Banner Children's at Desert

Mesa, Arizona, 85202, United States
Baylor College of Medicine/Dan L Duncan Comprehensive Cancer Center

Houston, Texas, 77030, United States
Baystate Medical Center

Springfield, Massachusetts, 01199, United States
Blank Children's Hospital

Des Moines, Iowa, 50309, United States
British Columbia Children's Hospital

Vancouver, British Columbia, V6H 3V4, Canada
Bronson Methodist Hospital

Kalamazoo, Michigan, 49007, United States
Broward Health Medical Center

Fort Lauderdale, Florida, 33316, United States
C S Mott Children's Hospital

Ann Arbor, Michigan, 48109, United States
CHU de Quebec-Centre Hospitalier de l'Universite Laval (CHUL)

Québec, G1V 4G2, Canada
CancerCare Manitoba

Winnipeg, Manitoba, R3E 0V9, Canada
Carolinas Medical Center/Levine Cancer Institute

Charlotte, North Carolina, 28203, United States
Cedars-Sinai Medical Center

Los Angeles, California, 90048, United States
Centre Hospitalier Universitaire Sainte-Justine

Montreal, Quebec, H3T 1C5, Canada
Children's Healthcare of Atlanta - Arthur M Blank Hospital

Atlanta, Georgia, 30329, United States
Children's Hospital

London, Ontario, N6A 5W9, Canada
Children's Hospital Colorado

Aurora, Colorado, 80045, United States
Children's Hospital Los Angeles

Los Angeles, California, 90027, United States
Children's Hospital Medical Center of Akron

Akron, Ohio, 44308, United States
Children's Hospital New Orleans

New Orleans, Louisiana, 70118, United States
Children's Hospital of Alabama

Birmingham, Alabama, 35233, United States
Children's Hospital of Eastern Ontario

Ottawa, Ontario, K1H 8L1, Canada
Children's Hospital of Orange County

Orange, California, 92868, United States
Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, 19104, United States
Children's Hospital of Pittsburgh of UPMC

Pittsburgh, Pennsylvania, 15224, United States
Children's Hospital of San Antonio

San Antonio, Texas, 78207, United States
Children's Hospital of The King's Daughters

Norfolk, Virginia, 23507, United States
Children's Hospital of Wisconsin

Milwaukee, Wisconsin, 53226, United States
Children's Hospitals and Clinics of Minnesota - Minneapolis

Minneapolis, Minnesota, 55404, United States
Children's Mercy Hospitals and Clinics

Kansas City, Missouri, 64108, United States
Children's National Medical Center

Washington D.C., District of Columbia, 20010, United States
Christchurch Hospital

Christchurch, 8011, New Zealand
Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, 45229, United States
Cleveland Clinic Foundation

Cleveland, Ohio, 44195, United States
Connecticut Children's Medical Center

Hartford, Connecticut, 06106, United States
Cook Children's Medical Center

Fort Worth, Texas, 76104, United States
Corewell Health Children's

Royal Oak, Michigan, 48073, United States
Corewell Health Grand Rapids Hospitals - Helen DeVos Children's Hospital

Grand Rapids, Michigan, 49503, United States
Covenant Children's Hospital

Lubbock, Texas, 79410, United States
Dana-Farber Cancer Institute

Boston, Massachusetts, 02215, United States
Dartmouth Hitchcock Medical Center/Dartmouth Cancer Center

Lebanon, New Hampshire, 03756, United States
Dayton Children's Hospital

Dayton, Ohio, 45404, United States
Dell Children's Medical Center of Central Texas

Austin, Texas, 78723, United States
Driscoll Children's Hospital

Corpus Christi, Texas, 78411, United States
Duke University Medical Center

Durham, North Carolina, 27710, United States
East Carolina University

Greenville, North Carolina, 27834, United States
East Tennessee Childrens Hospital

Knoxville, Tennessee, 37916, United States
Eastern Maine Medical Center

Bangor, Maine, 04401, United States
El Paso Children's Hospital

El Paso, Texas, 79905, United States
Geisinger Medical Center

Danville, Pennsylvania, 17822, United States
Golisano Children's Hospital of Southwest Florida

Fort Myers, Florida, 33908, United States
Hackensack University Medical Center

Hackensack, New Jersey, 07601, United States
Henry Ford Health Saint John Hospital

Detroit, Michigan, 48236, United States
Hospital for Sick Children

Toronto, Ontario, M5G 1X8, Canada
Hurley Medical Center

Flint, Michigan, 48503, United States
IWK Health Centre

Halifax, Nova Scotia, B3K 6R8, Canada
Inova Fairfax Hospital

Falls Church, Virginia, 22042, United States
John Hunter Children's Hospital

Hunter Regional Mail Centre, New South Wales, 2310, Australia
Johns Hopkins All Children's Hospital

St. Petersburg, Florida, 33701, United States
Johns Hopkins University/Sidney Kimmel Cancer Center

Baltimore, Maryland, 21287, United States
Kaiser Permanente Downey Medical Center

Downey, California, 90242, United States
Kaiser Permanente-Oakland

Oakland, California, 94611, United States
Kapiolani Medical Center for Women and Children

Honolulu, Hawaii, 96826, United States
King Faisal Specialist Hospital and Research Centre

Riyadh, 11211, Saudi Arabia
Kingston Health Sciences Centre

Kingston, Ontario, K7L 2V7, Canada
Laura and Isaac Perlmutter Cancer Center at NYU Langone

New York, New York, 10016, United States
Legacy Emanuel Children's Hospital

Portland, Oregon, 97227, United States
Loma Linda University Medical Center

Loma Linda, California, 92354, United States
Lucile Packard Children's Hospital Stanford University

Palo Alto, California, 94304, United States
Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center

Torrance, California, 90502, United States
Lurie Children's Hospital-Chicago

Chicago, Illinois, 60611, United States
Madigan Army Medical Center

Tacoma, Washington, 98431, United States
Mary Bridge Children's Hospital and Health Center

Tacoma, Washington, 98405, United States
Mayo Clinic in Rochester

Rochester, Minnesota, 55905, United States
McMaster Children's Hospital at Hamilton Health Sciences

Hamilton, Ontario, L8N 3Z5, Canada
MedStar Georgetown University Hospital

Washington D.C., District of Columbia, 20007, United States
Medical City Dallas Hospital

Dallas, Texas, 75230, United States
Medical University of South Carolina

Charleston, South Carolina, 29425, United States
Memorial Health University Medical Center

Savannah, Georgia, 31404, United States
Memorial Regional Hospital/Joe DiMaggio Children's Hospital

Hollywood, Florida, 33021, United States
Mercy Hospital Saint Louis

St Louis, Missouri, 63141, United States
Methodist Children's Hospital of South Texas

San Antonio, Texas, 78229, United States
Michigan State University

East Lansing, Michigan, 48823, United States
Miller Children's and Women's Hospital Long Beach

Long Beach, California, 90806, United States
Mission Hospital

Asheville, North Carolina, 28801, United States
Montefiore Medical Center - Moses Campus

The Bronx, New York, 10467, United States
Morristown Medical Center

Morristown, New Jersey, 07960, United States
Mount Sinai Hospital

New York, New York, 10029, United States
NYP/Columbia University Medical Center/Herbert Irving Comprehensive Cancer Center

New York, New York, 10032, United States
NYP/Weill Cornell Medical Center

New York, New York, 10065, United States
NYU Langone Hospital - Long Island

Mineola, New York, 11501, United States
Nationwide Children's Hospital

Columbus, Ohio, 43205, United States
Naval Medical Center -San Diego

San Diego, California, 92134, United States
Nemours Children's Clinic - Pensacola

Pensacola, Florida, 32504, United States
Nemours Children's Clinic-Jacksonville

Jacksonville, Florida, 32207, United States
Nemours Children's Hospital

Orlando, Florida, 32827, United States
New York Medical College

Valhalla, New York, 10595, United States
Nicklaus Children's Hospital

Miami, Florida, 33155, United States
Norton Children's Hospital

Louisville, Kentucky, 40202, United States
Novant Health Presbyterian Medical Center

Charlotte, North Carolina, 28204, United States
OSF Children's Hospital of Illinois

Peoria, Illinois, 61637, United States
Ochsner Medical Center Jefferson

New Orleans, Louisiana, 70121, United States
Oregon Health and Science University

Portland, Oregon, 97239, United States
Palms West Radiation Therapy

Loxahatchee Groves, Florida, 33470, United States
Penn State Children's Hospital

Hershey, Pennsylvania, 17033, United States
Perth Children's Hospital

Perth, Western Australia, 6009, Australia
Phoenix Childrens Hospital

Phoenix, Arizona, 85016, United States
Presbyterian Hospital

Albuquerque, New Mexico, 87106, United States
Primary Children's Hospital

Salt Lake City, Utah, 84113, United States
Princess Margaret Hospital for Children

Perth, Western Australia, 6008, Australia
ProMedica Toledo Hospital/Russell J Ebeid Children's Hospital

Toledo, Ohio, 43606, United States
Providence Alaska Medical Center

Anchorage, Alaska, 99508, United States
Providence Sacred Heart Medical Center and Children's Hospital

Spokane, Washington, 99204, United States
Rady Children's Hospital - San Diego

San Diego, California, 92123, United States
Rainbow Babies and Childrens Hospital

Cleveland, Ohio, 44106, United States
Renown Regional Medical Center

Reno, Nevada, 89502, United States
Rhode Island Hospital

Providence, Rhode Island, 02903, United States
Riley Hospital for Children

Indianapolis, Indiana, 46202, United States
Rocky Mountain Hospital for Children-Presbyterian Saint Luke's Medical Center

Denver, Colorado, 80218, United States
Roswell Park Cancer Institute

Buffalo, New York, 14263, United States
Royal Children's Hospital

Parkville, Victoria, 3052, Australia
Rutgers Cancer Institute of New Jersey-Robert Wood Johnson University Hospital

New Brunswick, New Jersey, 08903, United States
Sacred Heart Hospital

Pensacola, Florida, 32504, United States
Saint Christopher's Hospital for Children

Philadelphia, Pennsylvania, 19134, United States
Saint Joseph's Hospital/Children's Hospital-Tampa

Tampa, Florida, 33607, United States
Saint Joseph's Regional Medical Center

Paterson, New Jersey, 07503, United States
Saint Jude Children's Research Hospital

Memphis, Tennessee, 38105, United States
Saint Luke's Cancer Institute - Boise

Boise, Idaho, 83712, United States
Saint Mary's Medical Center

West Palm Beach, Florida, 33407, United States
Saint Vincent Hospital Cancer Center Green Bay

Green Bay, Wisconsin, 54301, United States
San Jorge Children's Hospital

San Juan, 00912, Puerto Rico
Sanford Broadway Medical Center

Fargo, North Dakota, 58122, United States
Sanford USD Medical Center - Sioux Falls

Sioux Falls, South Dakota, 57117-5134, United States
Seattle Children's Hospital

Seattle, Washington, 98105, United States
Sinai Hospital of Baltimore

Baltimore, Maryland, 21215, United States
Southern Illinois University School of Medicine

Springfield, Illinois, 62702, United States
Starship Children's Hospital

Grafton, Auckland, 1145, New Zealand
State University of New York Upstate Medical University

Syracuse, New York, 13210, United States
Stony Brook University Medical Center

Stony Brook, New York, 11794, United States
Summerlin Hospital Medical Center

Las Vegas, Nevada, 89144, United States
Sunrise Hospital and Medical Center

Las Vegas, Nevada, 89109, United States
Sutter Medical Center Sacramento

Sacramento, California, 95816, United States
Sydney Children's Hospital

Randwick, New South Wales, 2031, Australia
T C Thompson Children's Hospital

Chattanooga, Tennessee, 37403, United States
Texas Tech University Health Sciences Center-Amarillo

Amarillo, Texas, 79106, United States
The Children's Hospital at TriStar Centennial

Nashville, Tennessee, 37203, United States
The Children's Hospital at Westmead

Westmead, New South Wales, 2145, Australia
The Montreal Children's Hospital of the MUHC

Montreal, Quebec, H3H 1P3, Canada
Tufts Children's Hospital

Boston, Massachusetts, 02111, United States
UCSF Benioff Children's Hospital Oakland

Oakland, California, 94609, United States
UCSF Medical Center-Mission Bay

San Francisco, California, 94158, United States
UF Health Cancer Institute - Gainesville

Gainesville, Florida, 32610, United States
UMC Cancer Center / UMC Health System

Lubbock, Texas, 79415, United States
UMass Memorial Medical Center - University Campus

Worcester, Massachusetts, 01655, United States
UNC Lineberger Comprehensive Cancer Center

Chapel Hill, North Carolina, 27599, United States
UT Southwestern/Simmons Cancer Center-Dallas

Dallas, Texas, 75390, United States
University Medical Center of Southern Nevada

Las Vegas, Nevada, 89102, United States
University Pediatric Hospital

San Juan, 00926, Puerto Rico
University of California Davis Comprehensive Cancer Center

Sacramento, California, 95817, United States
University of Chicago Comprehensive Cancer Center

Chicago, Illinois, 60637, United States
University of Illinois

Chicago, Illinois, 60612, United States
University of Iowa/Holden Comprehensive Cancer Center

Iowa City, Iowa, 52242, United States
University of Kentucky/Markey Cancer Center

Lexington, Kentucky, 40536, United States
University of Miami Miller School of Medicine-Sylvester Cancer Center

Miami, Florida, 33136, United States
University of Minnesota/Masonic Cancer Center

Minneapolis, Minnesota, 55455, United States
University of Mississippi Medical Center

Jackson, Mississippi, 39216, United States
University of Missouri Children's Hospital

Columbia, Missouri, 65212, United States
University of New Mexico Cancer Center

Albuquerque, New Mexico, 87106, United States
University of Oklahoma Health Sciences Center

Oklahoma City, Oklahoma, 73104, United States
University of Rochester

Rochester, New York, 14642, United States
University of Texas Health Science Center at San Antonio

San Antonio, Texas, 78229, United States
University of Vermont and State Agricultural College

Burlington, Vermont, 05405, United States
University of Wisconsin Carbone Cancer Center - University Hospital

Madison, Wisconsin, 53792, United States
VCU Massey Comprehensive Cancer Center

Richmond, Virginia, 23298, United States
Valley Children's Hospital

Madera, California, 93636, United States
Vanderbilt University/Ingram Cancer Center

Nashville, Tennessee, 37232, United States
Wake Forest University Health Sciences

Winston-Salem, North Carolina, 27157, United States
Walter Reed National Military Medical Center

Bethesda, Maryland, 20889-5600, United States
Washington University School of Medicine

St Louis, Missouri, 63110, United States
Wayne State University/Karmanos Cancer Institute

Detroit, Michigan, 48201, United States
West Virginia University Charleston Division

Charleston, West Virginia, 25304, United States
Yale University

New Haven, Connecticut, 06520, United States

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