Acute Myeloid Leukemia Clinical Trial

Response-Based Chemotherapy in Treating Newly Diagnosed Acute Myeloid Leukemia or Myelodysplastic Syndrome in Younger Patients With Down Syndrome

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.

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Full Description

PRIMARY OBJECTIVES:

I. To determine the 2-year event-free-survival (EFS) for children with standard risk Down syndrome (DS) acute myeloid leukemia (AML) (minimal residual disease [MRD]-negative after one cycle of induction therapy) after elimination of high dose (HD) Ara-C (cytarabine) from the treatment regimen.

II. To determine the 2-year EFS for children with high risk DS AML (MRD-positive after one cycle of induction therapy) after intensification of treatment equivalent to that used for high risk AML in children without DS.

EXPLORATORY OBJECTIVES:

I. To compare the feasibility and analytical characteristics of flow cytometry, polymerase chain reaction (PCR) and targeted error-corrected sequencing of GATA binding protein 1 (globin transcription factor 1) (GATA1) mutations as methods to detect MRD in DS AML.

II. To establish a DS AML cell bank of viably frozen bone marrow samples collected at the end of induction and corresponding non-tumor deoxyribonucleic acid (DNA) samples collected at end of Induction 1.

OUTLINE:

INDUCTION I: Patients receive cytarabine intrathecally (IT) on day 1 and intravenously (IV) continuously over 96 hours, daunorubicin hydrochloride IV over 1-15 minutes, and thioguanine orally (PO) twice daily (BID) on days 1-4. Induction I continues for a minimum of 28 days.

Patients are assigned to 1 of 2 treatment arms based on their MRD status after completion of Induction I.

ARM A (STANDARD RISK) (Closed to accrual and treatment with amendment #4A 01/07/2019):

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.

ARM B (HIGH RISK):

INDUCTION II: Patients receive high dose cytarabine IV over 1-3 hours every 12 (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 (E. carotovora) intramuscularly (IM) or IV over 30 minutes on days 2 and 9. Intensification II continues for a minimum of 28 days.

After completion of study treatment, patients are followed up at 1 month, monthly for 12 months, every 3 months for 12 months, every 6 months for 3 years, annually for 10 years, and in case of relapse.

View Eligibility Criteria

Eligibility Criteria

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

Study is for people with:

Acute Myeloid Leukemia

Phase:

Phase 3

Estimated Enrollment:

312

Study ID:

NCT02521493

Recruitment Status:

Active, not recruiting

Sponsor:

Children's Oncology Group

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There are 189 Locations for this study

See Locations Near You

Children's Hospital of Alabama
Birmingham Alabama, 35233, United States
Providence Alaska Medical Center
Anchorage Alaska, 99508, United States
Banner Children's at Desert
Mesa Arizona, 85202, United States
Phoenix Childrens Hospital
Phoenix Arizona, 85016, United States
Arkansas Children's Hospital
Little Rock Arkansas, 72202, United States
Kaiser Permanente Downey Medical Center
Downey California, 90242, United States
Loma Linda University Medical Center
Loma Linda California, 92354, United States
Miller Children's and Women's Hospital Long Beach
Long Beach California, 90806, United States
Children's Hospital Los Angeles
Los Angeles California, 90027, United States
Cedars Sinai Medical Center
Los Angeles California, 90048, United States
Valley Children's Hospital
Madera California, 93636, United States
UCSF Benioff Children's Hospital Oakland
Oakland California, 94609, United States
Kaiser Permanente-Oakland
Oakland California, 94611, United States
Children's Hospital of Orange County
Orange California, 92868, United States
Lucile Packard Children's Hospital Stanford University
Palo Alto California, 94304, United States
Sutter Medical Center Sacramento
Sacramento California, 95816, United States
University of California Davis Comprehensive Cancer Center
Sacramento California, 95817, United States
Rady Children's Hospital - San Diego
San Diego California, 92123, United States
Naval Medical Center -San Diego
San Diego California, 92134, United States
UCSF Medical Center-Mission Bay
San Francisco California, 94158, United States
Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center
Torrance California, 90502, United States
Children's Hospital Colorado
Aurora Colorado, 80045, United States
Rocky Mountain Hospital for Children-Presbyterian Saint Luke's Medical Center
Denver Colorado, 80218, United States
Connecticut Children's Medical Center
Hartford Connecticut, 06106, United States
Yale University
New Haven Connecticut, 06520, United States
Alfred I duPont Hospital for Children
Wilmington Delaware, 19803, United States
MedStar Georgetown University Hospital
Washington District of Columbia, 20007, United States
Children's National Medical Center
Washington District of Columbia, 20010, United States
Broward Health Medical Center
Fort Lauderdale Florida, 33316, United States
Golisano Children's Hospital of Southwest Florida
Fort Myers Florida, 33908, United States
University of Florida Health Science Center - Gainesville
Gainesville Florida, 32610, United States
Memorial Regional Hospital/Joe DiMaggio Children's Hospital
Hollywood Florida, 33021, United States
Nemours Children's Clinic-Jacksonville
Jacksonville Florida, 32207, United States
Palms West Radiation Therapy
Loxahatchee Groves Florida, 33470, United States
University of Miami Miller School of Medicine-Sylvester Cancer Center
Miami Florida, 33136, United States
Nicklaus Children's Hospital
Miami Florida, 33155, United States
AdventHealth Orlando
Orlando Florida, 32803, United States
Arnold Palmer Hospital for Children
Orlando Florida, 32806, United States
Nemours Children's Hospital
Orlando Florida, 32827, United States
Nemours Children's Clinic - Pensacola
Pensacola Florida, 32504, United States
Sacred Heart Hospital
Pensacola Florida, 32504, United States
Johns Hopkins All Children's Hospital
Saint Petersburg Florida, 33701, United States
Saint Joseph's Hospital/Children's Hospital-Tampa
Tampa Florida, 33607, United States
Saint Mary's Hospital
West Palm Beach Florida, 33407, United States
Children's Healthcare of Atlanta - Egleston
Atlanta Georgia, 30322, United States
Memorial Health University Medical Center
Savannah Georgia, 31404, United States
Kapiolani Medical Center for Women and Children
Honolulu Hawaii, 96826, United States
Saint Luke's Cancer Institute - Boise
Boise Idaho, 83712, United States
Lurie Children's Hospital-Chicago
Chicago Illinois, 60611, United States
University of Illinois
Chicago Illinois, 60612, United States
University of Chicago Comprehensive Cancer Center
Chicago Illinois, 60637, 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
Saint Jude Midwest Affiliate
Peoria Illinois, 61637, United States
Southern Illinois University School of Medicine
Springfield Illinois, 62702, United States
Riley Hospital for Children
Indianapolis Indiana, 46202, United States
Ascension Saint Vincent Indianapolis Hospital
Indianapolis Indiana, 46260, United States
Blank Children's Hospital
Des Moines Iowa, 50309, 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
Norton Children's Hospital
Louisville Kentucky, 40202, United States
Children's Hospital New Orleans
New Orleans Louisiana, 70118, United States
Ochsner Medical Center Jefferson
New Orleans Louisiana, 70121, United States
Eastern Maine Medical Center
Bangor Maine, 04401, United States
Sinai Hospital of Baltimore
Baltimore Maryland, 21215, United States
Johns Hopkins University/Sidney Kimmel Cancer Center
Baltimore Maryland, 21287, United States
Walter Reed National Military Medical Center
Bethesda Maryland, 20889, United States
Tufts Children's Hospital
Boston Massachusetts, 02111, United States
Dana-Farber Cancer Institute
Boston Massachusetts, 02215, United States
Baystate Medical Center
Springfield Massachusetts, 01199, United States
UMass Memorial Medical Center - University Campus
Worcester Massachusetts, 01655, United States
C S Mott Children's Hospital
Ann Arbor Michigan, 48109, United States
Wayne State University/Karmanos Cancer Institute
Detroit Michigan, 48201, United States
Ascension Saint John Hospital
Detroit Michigan, 48236, United States
Michigan State University Clinical Center
East Lansing Michigan, 48824, United States
Hurley Medical Center
Flint Michigan, 48503, United States
Helen DeVos Children's Hospital at Spectrum Health
Grand Rapids Michigan, 49503, United States
Bronson Methodist Hospital
Kalamazoo Michigan, 49007, United States
Beaumont Children's Hospital-Royal Oak
Royal Oak Michigan, 48073, United States
Children's Hospitals and Clinics of Minnesota - Minneapolis
Minneapolis Minnesota, 55404, United States
University of Minnesota/Masonic Cancer Center
Minneapolis Minnesota, 55455, United States
Mayo Clinic in Rochester
Rochester Minnesota, 55905, United States
University of Mississippi Medical Center
Jackson Mississippi, 39216, United States
Columbia Regional
Columbia Missouri, 65201, United States
Children's Mercy Hospitals and Clinics
Kansas City Missouri, 64108, United States
Washington University School of Medicine
Saint Louis Missouri, 63110, United States
Mercy Hospital Saint Louis
Saint Louis Missouri, 63141, United States
University Medical Center of Southern Nevada
Las Vegas Nevada, 89102, United States
Sunrise Hospital and Medical Center
Las Vegas Nevada, 89109, United States
Alliance for Childhood Diseases/Cure 4 the Kids Foundation
Las Vegas Nevada, 89135, United States
Summerlin Hospital Medical Center
Las Vegas Nevada, 89144, United States
Renown Regional Medical Center
Reno Nevada, 89502, United States
Dartmouth Hitchcock Medical Center/Dartmouth Cancer Center
Lebanon New Hampshire, 03756, United States
Hackensack University Medical Center
Hackensack New Jersey, 07601, United States
Morristown Medical Center
Morristown New Jersey, 07960, United States
Rutgers Cancer Institute of New Jersey-Robert Wood Johnson University Hospital
New Brunswick New Jersey, 08903, United States
Saint Joseph's Regional Medical Center
Paterson New Jersey, 07503, United States
University of New Mexico Cancer Center
Albuquerque New Mexico, 87102, United States
Presbyterian Hospital
Albuquerque New Mexico, 87106, United States
Albany Medical Center
Albany New York, 12208, United States
Montefiore Medical Center - Moses Campus
Bronx New York, 10467, United States
Roswell Park Cancer Institute
Buffalo New York, 14263, United States
NYU Winthrop Hospital
Mineola New York, 11501, United States
Laura and Isaac Perlmutter Cancer Center at NYU Langone
New York New York, 10016, 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
University of Rochester
Rochester New York, 14642, United States
Stony Brook University Medical Center
Stony Brook New York, 11794, United States
State University of New York Upstate Medical University
Syracuse New York, 13210, United States
New York Medical College
Valhalla New York, 10595, United States
Mission Hospital
Asheville North Carolina, 28801, United States
UNC Lineberger Comprehensive Cancer Center
Chapel Hill North Carolina, 27599, United States
Carolinas Medical Center/Levine Cancer Institute
Charlotte North Carolina, 28203, United States
Novant Health Presbyterian Medical Center
Charlotte North Carolina, 28204, United States
Duke University Medical Center
Durham North Carolina, 27710, United States
East Carolina University
Greenville North Carolina, 27834, United States
Wake Forest University Health Sciences
Winston-Salem North Carolina, 27157, United States
Sanford Broadway Medical Center
Fargo North Dakota, 58122, United States
Children's Hospital Medical Center of Akron
Akron Ohio, 44308, United States
Cincinnati Children's Hospital Medical Center
Cincinnati Ohio, 45229, United States
Rainbow Babies and Childrens Hospital
Cleveland Ohio, 44106, United States
Cleveland Clinic Foundation
Cleveland Ohio, 44195, United States
Nationwide Children's Hospital
Columbus Ohio, 43205, United States
Dayton Children's Hospital
Dayton Ohio, 45404, United States
ProMedica Toledo Hospital/Russell J Ebeid Children's Hospital
Toledo Ohio, 43606, United States
University of Oklahoma Health Sciences Center
Oklahoma City Oklahoma, 73104, United States
Legacy Emanuel Children's Hospital
Portland Oregon, 97227, United States
Oregon Health and Science University
Portland Oregon, 97239, United States
Geisinger Medical Center
Danville Pennsylvania, 17822, United States
Penn State Children's Hospital
Hershey Pennsylvania, 17033, United States
Children's Hospital of Philadelphia
Philadelphia Pennsylvania, 19104, United States
Saint Christopher's Hospital for Children
Philadelphia Pennsylvania, 19134, United States
Children's Hospital of Pittsburgh of UPMC
Pittsburgh Pennsylvania, 15224, United States
Rhode Island Hospital
Providence Rhode Island, 02903, United States
Medical University of South Carolina
Charleston South Carolina, 29425, United States
BI-LO Charities Children's Cancer Center
Greenville South Carolina, 29605, United States
Sanford USD Medical Center - Sioux Falls
Sioux Falls South Dakota, 57117, United States
T C Thompson Children's Hospital
Chattanooga Tennessee, 37403, United States
East Tennessee Childrens Hospital
Knoxville Tennessee, 37916, United States
Saint Jude Children's Research Hospital
Memphis Tennessee, 38105, United States
The Children's Hospital at TriStar Centennial
Nashville Tennessee, 37203, United States
Vanderbilt University/Ingram Cancer Center
Nashville Tennessee, 37232, United States
Texas Tech University Health Sciences Center-Amarillo
Amarillo Texas, 79106, 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
Medical City Dallas Hospital
Dallas Texas, 75230, United States
UT Southwestern/Simmons Cancer Center-Dallas
Dallas Texas, 75390, United States
El Paso Children's Hospital
El Paso Texas, 79905, United States
Cook Children's Medical Center
Fort Worth Texas, 76104, United States
Baylor College of Medicine/Dan L Duncan Comprehensive Cancer Center
Houston Texas, 77030, United States
Covenant Children's Hospital
Lubbock Texas, 79410, United States
UMC Cancer Center / UMC Health System
Lubbock Texas, 79415, United States
Children's Hospital of San Antonio
San Antonio Texas, 78207, United States
Methodist Children's Hospital of South Texas
San Antonio Texas, 78229, United States
University of Texas Health Science Center at San Antonio
San Antonio Texas, 78229, United States
Primary Children's Hospital
Salt Lake City Utah, 84113, United States
University of Vermont and State Agricultural College
Burlington Vermont, 05405, United States
Inova Fairfax Hospital
Falls Church Virginia, 22042, United States
Children's Hospital of The King's Daughters
Norfolk Virginia, 23507, United States
Virginia Commonwealth University/Massey Cancer Center
Richmond Virginia, 23298, United States
Seattle Children's Hospital
Seattle Washington, 98105, United States
Providence Sacred Heart Medical Center and Children's Hospital
Spokane Washington, 99204, United States
Mary Bridge Children's Hospital and Health Center
Tacoma Washington, 98405, United States
Madigan Army Medical Center
Tacoma Washington, 98431, United States
West Virginia University Charleston Division
Charleston West Virginia, 25304, United States
Saint Vincent Hospital Cancer Center Green Bay
Green Bay Wisconsin, 54301, United States
University of Wisconsin Carbone Cancer Center
Madison Wisconsin, 53792, United States
Children's Hospital of Wisconsin
Milwaukee Wisconsin, 53226, United States
John Hunter Children's Hospital
Hunter Regional Mail Centre New South Wales, 2310, Australia
Sydney Children's Hospital
Randwick New South Wales, 2031, Australia
The Children's Hospital at Westmead
Westmead New South Wales, 2145, Australia
Royal Children's Hospital
Parkville Victoria, 3052, Australia
Princess Margaret Hospital for Children
Perth Western Australia, 6008, Australia
Perth Children's Hospital
Perth Western Australia, 6009, Australia
British Columbia Children's Hospital
Vancouver British Columbia, V6H 3, Canada
CancerCare Manitoba
Winnipeg Manitoba, R3E 0, Canada
IWK Health Centre
Halifax Nova Scotia, B3K 6, Canada
McMaster Children's Hospital at Hamilton Health Sciences
Hamilton Ontario, L8N 3, Canada
Kingston Health Sciences Centre
Kingston Ontario, K7L 2, Canada
Children's Hospital
London Ontario, N6A 5, Canada
Children's Hospital of Eastern Ontario
Ottawa Ontario, K1H 8, Canada
Hospital for Sick Children
Toronto Ontario, M5G 1, Canada
The Montreal Children's Hospital of the MUHC
Montreal Quebec, H3H 1, Canada
Centre Hospitalier Universitaire Sainte-Justine
Montreal Quebec, H3T 1, Canada
CHU de Quebec-Centre Hospitalier de l'Universite Laval (CHUL)
Quebec , G1V 4, Canada
Starship Children's Hospital
Grafton Auckland, 1145, New Zealand
Christchurch Hospital
Christchurch , 8011, New Zealand
San Jorge Children's Hospital
San Juan , 00912, Puerto Rico
University Pediatric Hospital
San Juan , 00926, Puerto Rico
King Faisal Specialist Hospital and Research Centre
Riyadh , 11211, Saudi Arabia

How clear is this clinincal trial information?

Study is for people with:

Acute Myeloid Leukemia

Phase:

Phase 3

Estimated Enrollment:

312

Study ID:

NCT02521493

Recruitment Status:

Active, not recruiting

Sponsor:


Children's Oncology Group

How clear is this clinincal trial information?

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