Risk-Stratified Therapy for Acute Myeloid Leukemia in Down Syndrome

Who is this study for? Infant to child patients with Down Syndrome and Acute Myeloid Leukemia or Myelodysplastic Syndrome
Status: Active, not recruiting
Location: See all (191) locations...
Intervention Type: Drug, Other
Study Type: Interventional
Study Phase: Phase 3
SUMMARY

This phase III trial studies response-based chemotherapy in treating newly diagnosed acute myeloid leukemia or myelodysplastic syndrome in younger patients with Down syndrome. Drugs used in chemotherapy work in different ways to stop the growth of cancer cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. Response-based chemotherapy separates patients into different risk groups and treats them according to how they respond to the first course of treatment (Induction I). Response-based treatment may be effective in treating acute myeloid leukemia or myelodysplastic syndrome in younger patients with Down syndrome while reducing the side effects.

Eligibility
Participation Requirements
Sex: All
Minimum Age: 3 months
Maximum Age: 3
Healthy Volunteers: No
View:

• 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

Locations
United States
Alaska
Providence Alaska Medical Center
Anchorage
Alabama
Children's Hospital of Alabama
Birmingham
Arkansas
Arkansas Children's Hospital
Little Rock
Arizona
Banner Children's at Desert
Mesa
Phoenix Childrens Hospital
Phoenix
California
Kaiser Permanente Downey Medical Center
Downey
Loma Linda University Medical Center
Loma Linda
Miller Children's and Women's Hospital Long Beach
Long Beach
Cedars Sinai Medical Center
Los Angeles
Children's Hospital Los Angeles
Los Angeles
Valley Children's Hospital
Madera
Kaiser Permanente-Oakland
Oakland
UCSF Benioff Children's Hospital Oakland
Oakland
Children's Hospital of Orange County
Orange
Lucile Packard Children's Hospital Stanford University
Palo Alto
Sutter Medical Center Sacramento
Sacramento
University of California Davis Comprehensive Cancer Center
Sacramento
Naval Medical Center -San Diego
San Diego
Rady Children's Hospital - San Diego
San Diego
UCSF Medical Center-Mission Bay
San Francisco
Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center
Torrance
Colorado
Children's Hospital Colorado
Aurora
Rocky Mountain Hospital for Children-Presbyterian Saint Luke's Medical Center
Denver
Connecticut
Connecticut Children's Medical Center
Hartford
Yale University
New Haven
Washington, D.c.
Children's National Medical Center
Washington
MedStar Georgetown University Hospital
Washington
Delaware
Alfred I duPont Hospital for Children
Wilmington
Florida
Broward Health Medical Center
Fort Lauderdale
Golisano Children's Hospital of Southwest Florida
Fort Myers
University of Florida Health Science Center - Gainesville
Gainesville
Memorial Regional Hospital/Joe DiMaggio Children's Hospital
Hollywood
Nemours Children's Clinic-Jacksonville
Jacksonville
Palms West Radiation Therapy
Loxahatchee Groves
Nicklaus Children's Hospital
Miami
University of Miami Miller School of Medicine-Sylvester Cancer Center
Miami
AdventHealth Orlando
Orlando
Arnold Palmer Hospital for Children
Orlando
Nemours Children's Hospital
Orlando
Nemours Children's Clinic - Pensacola
Pensacola
Sacred Heart Hospital
Pensacola
Johns Hopkins All Children's Hospital
Saint Petersburg
Saint Joseph's Hospital/Children's Hospital-Tampa
Tampa
Saint Mary's Hospital
West Palm Beach
Georgia
Children's Healthcare of Atlanta - Egleston
Atlanta
Memorial Health University Medical Center
Savannah
Hawaii
Kapiolani Medical Center for Women and Children
Honolulu
Iowa
Blank Children's Hospital
Des Moines
University of Iowa/Holden Comprehensive Cancer Center
Iowa City
Idaho
Saint Luke's Cancer Institute - Boise
Boise
Illinois
Lurie Children's Hospital-Chicago
Chicago
University of Chicago Comprehensive Cancer Center
Chicago
University of Illinois
Chicago
Advocate Children's Hospital-Oak Lawn
Oak Lawn
Advocate Children's Hospital-Park Ridge
Park Ridge
Saint Jude Midwest Affiliate
Peoria
Southern Illinois University School of Medicine
Springfield
Indiana
Ascension Saint Vincent Indianapolis Hospital
Indianapolis
Riley Hospital for Children
Indianapolis
Kentucky
University of Kentucky/Markey Cancer Center
Lexington
Norton Children's Hospital
Louisville
Louisiana
Children's Hospital New Orleans
New Orleans
Ochsner Medical Center Jefferson
New Orleans
Massachusetts
Dana-Farber Cancer Institute
Boston
Tufts Children's Hospital
Boston
Baystate Medical Center
Springfield
UMass Memorial Medical Center - University Campus
Worcester
Maryland
Johns Hopkins University/Sidney Kimmel Cancer Center
Baltimore
Sinai Hospital of Baltimore
Baltimore
Walter Reed National Military Medical Center
Bethesda
Maine
Eastern Maine Medical Center
Bangor
Michigan
C S Mott Children's Hospital
Ann Arbor
Ascension Saint John Hospital
Detroit
Wayne State University/Karmanos Cancer Institute
Detroit
Michigan State University Clinical Center
East Lansing
Hurley Medical Center
Flint
Helen DeVos Children's Hospital at Spectrum Health
Grand Rapids
Bronson Methodist Hospital
Kalamazoo
Beaumont Children's Hospital-Royal Oak
Royal Oak
Minnesota
Children's Hospitals and Clinics of Minnesota - Minneapolis
Minneapolis
University of Minnesota/Masonic Cancer Center
Minneapolis
Mayo Clinic in Rochester
Rochester
Missouri
Columbia Regional
Columbia
Children's Mercy Hospitals and Clinics
Kansas City
Mercy Hospital Saint Louis
Saint Louis
Washington University School of Medicine
Saint Louis
Mississippi
University of Mississippi Medical Center
Jackson
North Carolina
Mission Hospital
Asheville
UNC Lineberger Comprehensive Cancer Center
Chapel Hill
Carolinas Medical Center/Levine Cancer Institute
Charlotte
Novant Health Presbyterian Medical Center
Charlotte
Duke University Medical Center
Durham
East Carolina University
Greenville
Wake Forest University Health Sciences
Winston-salem
North Dakota
Sanford Broadway Medical Center
Fargo
New Hampshire
Dartmouth Hitchcock Medical Center
Lebanon
New Jersey
Hackensack University Medical Center
Hackensack
Morristown Medical Center
Morristown
Rutgers Cancer Institute of New Jersey-Robert Wood Johnson University Hospital
New Brunswick
Saint Joseph's Regional Medical Center
Paterson
New Mexico
Presbyterian Hospital
Albuquerque
University of New Mexico Cancer Center
Albuquerque
Nevada
Alliance for Childhood Diseases/Cure 4 the Kids Foundation
Las Vegas
Summerlin Hospital Medical Center
Las Vegas
Sunrise Hospital and Medical Center
Las Vegas
University Medical Center of Southern Nevada
Las Vegas
Renown Regional Medical Center
Reno
New York
Albany Medical Center
Albany
Montefiore Medical Center - Moses Campus
Bronx
Roswell Park Cancer Institute
Buffalo
NYU Winthrop Hospital
Mineola
Laura and Isaac Perlmutter Cancer Center at NYU Langone
New York
Mount Sinai Hospital
New York
NYP/Columbia University Medical Center/Herbert Irving Comprehensive Cancer Center
New York
NYP/Weill Cornell Medical Center
New York
University of Rochester
Rochester
Stony Brook University Medical Center
Stony Brook
State University of New York Upstate Medical University
Syracuse
New York Medical College
Valhalla
Ohio
Children's Hospital Medical Center of Akron
Akron
Cincinnati Children's Hospital Medical Center
Cincinnati
Cleveland Clinic Foundation
Cleveland
Rainbow Babies and Childrens Hospital
Cleveland
Nationwide Children's Hospital
Columbus
Dayton Children's Hospital
Dayton
ProMedica Toledo Hospital/Russell J Ebeid Children's Hospital
Toledo
Oklahoma
University of Oklahoma Health Sciences Center
Oklahoma City
Oregon
Legacy Emanuel Children's Hospital
Portland
Oregon Health and Science University
Portland
Pennsylvania
Geisinger Medical Center
Danville
Penn State Children's Hospital
Hershey
Children's Hospital of Philadelphia
Philadelphia
Saint Christopher's Hospital for Children
Philadelphia
Children's Hospital of Pittsburgh of UPMC
Pittsburgh
Rhode Island
Rhode Island Hospital
Providence
South Carolina
Medical University of South Carolina
Charleston
BI-LO Charities Children's Cancer Center
Greenville
South Dakota
Sanford USD Medical Center - Sioux Falls
Sioux Falls
Tennessee
T C Thompson Children's Hospital
Chattanooga
East Tennessee Childrens Hospital
Knoxville
Saint Jude Children's Research Hospital
Memphis
The Children's Hospital at TriStar Centennial
Nashville
Vanderbilt University/Ingram Cancer Center
Nashville
Texas
Texas Tech University Health Sciences Center-Amarillo
Amarillo
Dell Children's Medical Center of Central Texas
Austin
Driscoll Children's Hospital
Corpus Christi
Medical City Dallas Hospital
Dallas
UT Southwestern/Simmons Cancer Center-Dallas
Dallas
El Paso Children's Hospital
El Paso
Cook Children's Medical Center
Fort Worth
Baylor College of Medicine/Dan L Duncan Comprehensive Cancer Center
Houston
Covenant Children's Hospital
Lubbock
UMC Cancer Center / UMC Health System
Lubbock
Children's Hospital of San Antonio
San Antonio
Methodist Children's Hospital of South Texas
San Antonio
University of Texas Health Science Center at San Antonio
San Antonio
Utah
Primary Children's Hospital
Salt Lake City
Virginia
Inova Fairfax Hospital
Falls Church
Children's Hospital of The King's Daughters
Norfolk
Virginia Commonwealth University/Massey Cancer Center
Richmond
Vermont
University of Vermont and State Agricultural College
Burlington
Washington
Seattle Children's Hospital
Seattle
Providence Sacred Heart Medical Center and Children's Hospital
Spokane
Madigan Army Medical Center
Tacoma
Mary Bridge Children's Hospital and Health Center
Tacoma
Wisconsin
Saint Vincent Hospital Cancer Center Green Bay
Green Bay
University of Wisconsin Carbone Cancer Center
Madison
Children's Hospital of Wisconsin
Milwaukee
West Virginia
West Virginia University Charleston Division
Charleston
Other Locations
Australia
John Hunter Children's Hospital
Hunter Regional Mail Centre
Royal Children's Hospital
Parkville
Perth Children's Hospital
Perth
Princess Margaret Hospital for Children
Perth
Sydney Children's Hospital
Randwick
The Children's Hospital at Westmead
Westmead
Canada
IWK Health Centre
Halifax
McMaster Children's Hospital at Hamilton Health Sciences
Hamilton
Kingston Health Sciences Centre
Kingston
Children's Hospital
London
Centre Hospitalier Universitaire Sainte-Justine
Montreal
The Montreal Children's Hospital of the MUHC
Montreal
Children's Hospital of Eastern Ontario
Ottawa
Centre Hospitalier Universitaire de Quebec
Quebec
Hospital for Sick Children
Toronto
British Columbia Children's Hospital
Vancouver
CancerCare Manitoba
Winnipeg
New Zealand
Christchurch Hospital
Christchurch
Starship Children's Hospital
Grafton
Puerto Rico
San Jorge Children's Hospital
San Juan
University Pediatric Hospital
San Juan
Saudi Arabia
King Faisal Specialist Hospital and Research Centre
Riyadh
Time Frame
Start Date: November 23, 2015
Estimated Completion Date: June 30, 2024
Participants
Target number of participants: 312
Treatments
Experimental: Arm A (standard risk)
INDUCTION II: Patients receive cytarabine IV continuously over 96 hours, daunorubicin hydrochloride IV over 1-15 minutes, and thioguanine PO BID on days 1-4. Induction II continues for a minimum of 28 days.~INDUCTION III: Patients receive cytarabine, daunorubicin hydrochloride, and thioguanine as in Induction II. Induction III continues for a minimum of 28 days.~INTENSIFICATION I: Patients receive cytarabine IV continuously over 168 hours on days 1-7 and etoposide IV over 60-120 minutes on days 1-3. Intensification I continues for a minimum of 28 days.~INTENSIFICATION II: Patients receive cytarabine and etoposide as in Intensification I. Intensification II continues for a minimum of 28 days.~(This arm is closed to accrual and treatment with amendment #4A 01/07/2019)
Experimental: Arm B (high risk)
INDUCTION II: Patients receive high dose cytarabine IV over 1-3 hours Q12 hours on days 1-4 and mitoxantrone hydrochloride IV over 15-30 minutes on days 3-6. Induction II continues for a minimum of 28 days.~INTENSIFICATION I: Patients receive high dose cytarabine IV over 1-3 hours Q12 hours and etoposide IV over 90-120 minutes on days 1-5. Intensification I continues for a minimum of 28 days.~INTENSIFICATION II: Patients receive high dose cytarabine IV over 3 hours Q12 hours on days 1, 2, 8, and 9. Patients also receive asparaginase or asparaginase Erwinia chrysanthemi IM or IV over 30 minutes on days 2 and 9. Intensification II continues for a minimum of 28 days.
Authors
Samuel O. Anim, Craig Erker, Debra L. Friedman, Lisa L. Hartman, Angela R. Girvin, John F. Kuttesch, Joseph M. Wiley, Carola A. Arndt, Elyssa M. Rubin, Robin Y. Dulman, Haydar A. Frangoul, William G. Woods, Ashley E. Meyer, Michael J. Burke, Alexander Aledo, Nina S. Kadan-Lottick, Alissa Kahn, Kathryn L. Laurie, Marcio H. Malogolowkin, Jessica C. Hochberg, Douglas S. Hawkins, Mary L. Schmidt, Eduard H. Panosyan, Vinod K. Gidvani-Diaz, Julio C. Barredo, Afshan A. Ali, Gita V. Massey, Vlad C. Radulescu, Matthew A. Kutny, Jeffrey W. Taub, Eric J. Lowe, Karol H. Kerr, Gary D. Crouch, Nadine P. SantaCruz, Mariko Sato, Caroline Y. Hu, Peter M. Gordon, Ashok B. Raj, Jessica M. Valdez, Anne B. Warwick, Peter H. Shaw, Jessica Boklan, Keith J. August, Chana L. Glasser, Kishor M. Bhende, Lisa Gennarini, Stanton C. Goldman, Michael S. Isakoff, Kenneth G. Lucas, John J. Letterio, Justine M. Kahn, Kayelyn J. Wagner, Mukund G. Dole, Michael J. Kelly, David L. Becton, Laura E. Agresta, Adit L. Tal, Lars M. Wagner, Phillip E. Barnette, Howard M. Katzenstein, Rabi Hanna, Hardeo K. Panchoosingh, Timothy C. Griffin, Robin D. Hanson, Vonda L. Crouse, Vincent F. Giusti, Jeffrey E. Rubnitz, Ranjan Bista, Kenneth J. Cohen, Don E. Eslin, William B. Slayton, Lolie C. Yu, Kamnesh R. Pradhan, Iftikhar Hanif, Xiaxin Li, Aron Flagg, Catherine A. Long, Mark A. Ranalli, David S. Dickens, Erin K. Barr, Leo Mascarenhas, Ray C. Pais, Laura E. Hogan, Jamie L. Dargart, Sheri L. Spunt, Adonis N. Lorenzana, Jason Law, Kenneth M. Heym, Duncan S. Stearns, Andrew L. Pendleton, Bethany G. Sleckman, Shelton A. Viola, Barbara A. Gruner, Yoko T. Udaka, Lisa G. Roth, Craig A. Mullen, Jose M. Esquilin, Manoo G. Bhakta, Rajen Mody, Joseph C. Torkildson, Clare J. Twist, Craig Lotterman, Erlyn C. Smith, Aniket Saha, Burton E. Appel, Maria E. Echevarria, Eugenia Chang, Sandeep Batra, Benjamin J. Huang, Waitman K. Aumann, Thomas B. Russell, Meghann P. McManus, Richard A. Drachtman, Kenneth Caldwell, Luke D. Maese, James T. Badgett, Robert G. Irwin, Carla B. Golden, Douglas J. Scothorn, Jonathan L. Metts, Joanna G. Luty, Fouad M. Hajjar, Judy L. Felgenhauer, Kelly W. Maloney, Barbara A. Degar, Wade T. Kyono, Amy A. Smith, Emi H. Caywood, Robert J. Fallon, Elaine R. Morgan, William D. Roberts, Gregory P. Brandt, Gregory A. Hale, Marshall A. Schorin, Lisa M. McGregor, Robert M. Cooper, Nichole L. Bryant, Santosh S. Hanmod, Wendy L. Woods-Swafford, Susan G. Kreissman, Steven K. Bergstrom, Sara Chaffee, Laura A. Campbell, Jennifer J. Welch, Jagadeesh Ramdas, Philip M. Monteleone, Andrea R. Whitfield, Jeffrey A. Toretsky, Jay Michael S. Balagtas, Joel A. Kaplan, Jean M. Tersak, Gregory E. Halligan, Bassem I. Razzouk, Brenda J. Wittman, Robert J. Hayashi, Tamra L. Slone, Yung S. Yim, Samuel J. Milanovich, Alan K. Ikeda, Samantha L. Mallory, Enrique A. Escalon, Dana A. Obzut, Nkechi I. Mba, Janice F. Olson, Laura K. Gowans, Rebecca E. McFall, Narayana Gowda, Michael K. Richards, Steven J. Kuerbitz, Alice Lee, Lauren R. Weintraub, Mark E. Weinblatt, Jeffrey H. Schwartz, Jason M. Fixler, Sharon L. Gardner, Nobuko Hijiya, Jennifer J. Clark, Karen R. Rabin, Julie Kim, Richard Aplenc, Mary A. Bonilla, Jessica A. Bell, Jessica L. Heath, Albert Kheradpour, Jeffrey S. Dome, Melissa A. Forouhar, Mohamad H. Badawi, Pillai Pallavi Madhusoodhan, Steven L. Halpern, Maureen M. O'Brien, Stefanie R. Lowas, Melissa S. Singer, Farzana Pashankar, John J. Gregory, Dana B. Salzberg, Emad K. Salman, Stuart H. Gold, Amy C. Fowler, Luis A. Clavell, Kenneth B. De Santes, Christopher P. Keuker, Tanya R. Brown, Nyree O. Cole, Violet Shen, Elizabeth L. Hesketh, Karen S. Fernandez, Patrick A. Brown, Rebecca O. Clark, Jacqueline N. Casillas, Ashley Chopek, Sharon B. Abish, Laura Wheaton, Ayman A. El-Sheikh, Jordan R. Hansford, Lochie R. Teague, Carol A. Portwine, Yvan Samson, Anne-Marie R. Langevin, Donna L. Johnston, David B. Dix, Pedro A. De Alarcon, Shannon M. Cohn, Jason Berman, Kenneth B. DeSantes, Uma H. Athale, Jacqueline M. Kraveka, Joseph L. Lasky, Siobhan F. Cross, Johann Hitzler, Fataneh (Fae) Majlessipour, Jennifer J. Greene Welch, Shayna M. Zelcer, Rene Y. McNall-Knapp, David E. Kram, Kathleen J. Yost, Jaime M. Libes, Marianne B. Phillips, Draga Barbaric, Samer Zaid Kaylani, Mohamad M. Al-Rahawan, Hector M. Rodriguez-Cortes, Mariana P. Silva, Chibuzo C. O'Suoji, Bill H. Chang, Sarah E. Leary, Geoff D. Cuvelier, Anderson (Andy) B. Collier, Jennifer L. McNeer, Bruno Michon, Geoffrey B. McCowage, Conrad V. Fernandez, Kenneth Lieuw
Sponsors
Leads: Children's Oncology Group
Collaborators: National Cancer Institute (NCI)

This content was sourced from clinicaltrials.gov

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