Stopping Tyrosine Kinase Inhibitors in Affecting Treatment-Free Remission in Patients With Chronic Phase Chronic Myeloid Leukemia
NCT ID: NCT03817398
Last Updated: 2025-12-22
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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ACTIVE_NOT_RECRUITING
PHASE2
110 participants
INTERVENTIONAL
2019-11-08
2026-06-30
Brief Summary
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Detailed Description
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I. To determine the 2-year treatment free remission (TFR) rate of children, adolescents, and young adults with chronic myeloid leukemia - chronic phase (CML-CP) following discontinuation tyrosine kinase inhibitor (TKI).
II. To estimate the re-induction rate and maintenance of molecular remission (BCR-ABL1 =\< 0.1%) at 1 year after restarting TKI for children, adolescents, and young adults.
SECONDARY OBJECTIVE:
I. To describe clinical factors and laboratory correlates affecting the persistence of major molecular remission (MMR) and re-initiation of treatment after stopping TKI (e.g. patient demographics, duration and level of prior molecular remission, duration and type of TKI, clinical presentation at diagnosis and immune studies).
EXPLORATORY OBJECTIVES:
I. To describe change in height standard deviation score over time in patients who are able to discontinue their TKI.
II. To describe the long-term health outcomes including but not limited to gonadal function, endocrine function, and bone metabolism in patients who are able to discontinue TKI as well as those that need to restart TKIs.
III. To describe differences in patient-reported health status after stopping TKIs, including those who need to resume TKI after stopping.
IV. To describe the incidence and characteristics of TKI withdrawal syndrome in children.
V. To evaluate changes in neurocognitive outcomes of patients enrolled on this study using a patient-completed, performance-based, computerized measure of neuropsychological functioning and a parent-report/self-report questionnaire.
OUTLINE:
Patients stop taking TKI medication within 10 days after enrollment. Patients undergo peripheral blood collection to monitor loss of MMR every 4 weeks in year 1, every 6 weeks in year 2, and every 12 weeks in year 3. Patients who lose their molecular remission may restart TKI medication and are monitored every 4 weeks in year 1, every 6 weeks in year 2, and every 12 weeks in year 3.
After completion of study treatment, patients are followed up annually.
Conditions
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Study Design
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NA
SINGLE_GROUP
TREATMENT
NONE
Study Groups
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Basic Science (stop taking TKI, biospecimen collection)
Patients stop taking TKI medication within 10 days after enrollment. Patients undergo peripheral blood collection to monitor loss of MMR every 4 weeks in year 1, every 6 weeks in year 2, and every 12 weeks in year 3. Patients who lose their molecular remission may restart TKI medication and are monitored every 4 weeks in year 1, every 6 weeks in year 2, and every 12 weeks in year 3.
Biospecimen Collection
Undergo blood collection
Drug Withdrawn
Stop taking TKI medication
Quality-of-Life Assessment
Ancillary studies
Questionnaire Administration
Ancillary studies
Tyrosine Kinase Inhibitor
Re-start TKI medication
Interventions
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Biospecimen Collection
Undergo blood collection
Drug Withdrawn
Stop taking TKI medication
Quality-of-Life Assessment
Ancillary studies
Questionnaire Administration
Ancillary studies
Tyrosine Kinase Inhibitor
Re-start TKI medication
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
* Patient must have histologic verification of CML-CP at original diagnosis
* Patient must be in molecular remission (MR) with a BCR-ABL1 level of =\< 0.01% BCR-ABL1 as measured using the International Scale (IS) by RQ-PCR for \>= 2 consecutive years at the time of enrollment
* Please note: The lab evaluating disease status and molecular response for this study must be College of American Pathology (CAP) and/or Clinical Laboratory Improvement Amendments (CLIA) certified (United States \[US\] only), sites in other countries must be certified by their accredited authorities. All labs must use the International Scale guidelines with a sensitivity of detection assay =\< 0.01% BCR-ABL1 and be able to report results in =\< 2 weeks
* Patient must have received any TKI for a minimum of 3 consecutive years at time of enrollment
* Patient agrees to discontinue TKI therapy
* REGULATORY REQUIREMENTS
* All patients and/or their 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 studies must be met
* ELIGIBILITY FOR PATIENT-REPORTED OUTCOMES (PROs):
* Age \>= 8 years at the time of enrollment
* Ability to understand English or Spanish
* Cognitive ability to complete instruments according to the primary team
* ELIGIBILITY FOR AAML18P1 NEUROCOGNITIVE STUDY:
* Patient must be 5 years or older at the time of enrollment
* English-, French- or Spanish-speaking
* No known history of neurodevelopmental disorder prior to diagnosis of CML (e.g., Down syndrome, Fragile X, William syndrome, mental retardation)
* No significant visual or motor impairment that would prevent computer use or recognition of visual test stimuli
Exclusion Criteria
* Additional clonal chromosomal abnormalities in Philadelphia chromosome (Ph) positive (+) cells at any time prior to enrollment that include "major route" abnormalities (second Ph, trisomy 8, isochromosome 17q, trisomy 19), complex karyotype or abnormalities of 3q26.2
* History of accelerated phase or blast crisis CML
* Female patients who are pregnant
* Lactating females are not eligible unless they have agreed not to breastfeed their infants
* Female patients of childbearing potential are not eligible unless a negative pregnancy test result has been obtained
25 Years
ALL
No
Sponsors
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National Cancer Institute (NCI)
NIH
Children's Oncology Group
NETWORK
Responsible Party
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Principal Investigators
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Sonali Chaudhury
Role: PRINCIPAL_INVESTIGATOR
Children's Oncology Group
Locations
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Children's Hospital of Alabama
Birmingham, Alabama, United States
USA Health Strada Patient Care Center
Mobile, Alabama, United States
Banner Children's at Desert
Mesa, Arizona, United States
Banner University Medical Center - Tucson
Tucson, Arizona, United States
Arkansas Children's Hospital
Little Rock, Arkansas, United States
Kaiser Permanente Downey Medical Center
Downey, California, United States
Miller Children's and Women's Hospital Long Beach
Long Beach, California, United States
Children's Hospital Los Angeles
Los Angeles, California, United States
Cedars Sinai Medical Center
Los Angeles, California, United States
Mattel Children's Hospital UCLA
Los Angeles, California, United States
Valley Children's Hospital
Madera, California, United States
UCSF Benioff Children's Hospital Oakland
Oakland, California, United States
Kaiser Permanente-Oakland
Oakland, California, United States
Children's Hospital of Orange County
Orange, California, United States
Lucile Packard Children's Hospital Stanford University
Palo Alto, California, United States
University of California Davis Comprehensive Cancer Center
Sacramento, California, United States
Rady Children's Hospital - San Diego
San Diego, California, United States
Children's Hospital Colorado
Aurora, Colorado, United States
Rocky Mountain Hospital for Children-Presbyterian Saint Luke's Medical Center
Denver, Colorado, United States
Connecticut Children's Medical Center
Hartford, Connecticut, United States
Alfred I duPont Hospital for Children
Wilmington, Delaware, United States
MedStar Georgetown University Hospital
Washington D.C., District of Columbia, United States
Children's National Medical Center
Washington D.C., District of Columbia, United States
Golisano Children's Hospital of Southwest Florida
Fort Myers, Florida, United States
UF Health Cancer Institute - Gainesville
Gainesville, Florida, United States
Memorial Regional Hospital/Joe DiMaggio Children's Hospital
Hollywood, Florida, United States
Nemours Children's Clinic-Jacksonville
Jacksonville, Florida, United States
University of Miami Miller School of Medicine-Sylvester Cancer Center
Miami, Florida, United States
Nicklaus Children's Hospital
Miami, Florida, United States
Arnold Palmer Hospital for Children
Orlando, Florida, United States
Nemours Children's Hospital
Orlando, Florida, United States
Sacred Heart Hospital
Pensacola, Florida, United States
Tampa General Hospital
Tampa, Florida, United States
Saint Joseph's Hospital/Children's Hospital-Tampa
Tampa, Florida, United States
Saint Mary's Medical Center
West Palm Beach, Florida, United States
Children's Healthcare of Atlanta - Arthur M Blank Hospital
Atlanta, Georgia, United States
Memorial Health University Medical Center
Savannah, Georgia, United States
Kapiolani Medical Center for Women and Children
Honolulu, Hawaii, United States
Saint Luke's Cancer Institute - Boise
Boise, Idaho, United States
Lurie Children's Hospital-Chicago
Chicago, Illinois, United States
University of Illinois
Chicago, Illinois, United States
University of Chicago Comprehensive Cancer Center
Chicago, Illinois, United States
Loyola University Medical Center
Maywood, Illinois, United States
Advocate Children's Hospital-Oak Lawn
Oak Lawn, Illinois, United States
Advocate Children's Hospital-Park Ridge
Park Ridge, Illinois, United States
Saint Jude Midwest Affiliate
Peoria, Illinois, United States
Southern Illinois University School of Medicine
Springfield, Illinois, United States
Northwestern Medicine Central DuPage Hospital
Winfield, Illinois, United States
Riley Hospital for Children
Indianapolis, Indiana, United States
Blank Children's Hospital
Des Moines, Iowa, United States
University of Iowa/Holden Comprehensive Cancer Center
Iowa City, Iowa, United States
University of Kentucky/Markey Cancer Center
Lexington, Kentucky, United States
Norton Children's Hospital
Louisville, Kentucky, United States
Children's Hospital New Orleans
New Orleans, Louisiana, United States
Ochsner Medical Center Jefferson
New Orleans, Louisiana, United States
Eastern Maine Medical Center
Bangor, Maine, United States
Maine Children's Cancer Program
Scarborough, Maine, United States
Sinai Hospital of Baltimore
Baltimore, Maryland, United States
Johns Hopkins University/Sidney Kimmel Cancer Center
Baltimore, Maryland, United States
Walter Reed National Military Medical Center
Bethesda, Maryland, United States
Tufts Children's Hospital
Boston, Massachusetts, United States
Dana-Farber Cancer Institute
Boston, Massachusetts, United States
UMass Memorial Medical Center - University Campus
Worcester, Massachusetts, United States
C S Mott Children's Hospital
Ann Arbor, Michigan, United States
Children's Hospital of Michigan
Detroit, Michigan, United States
Wayne State University/Karmanos Cancer Institute
Detroit, Michigan, United States
Michigan State University
East Lansing, Michigan, United States
Corewell Health Grand Rapids Hospitals - Helen DeVos Children's Hospital
Grand Rapids, Michigan, United States
Bronson Methodist Hospital
Kalamazoo, Michigan, United States
Corewell Health Children's
Royal Oak, Michigan, United States
Children's Hospitals and Clinics of Minnesota - Minneapolis
Minneapolis, Minnesota, United States
University of Minnesota/Masonic Cancer Center
Minneapolis, Minnesota, United States
University of Mississippi Medical Center
Jackson, Mississippi, United States
University of Missouri Children's Hospital
Columbia, Missouri, United States
Children's Mercy Hospitals and Clinics
Kansas City, Missouri, United States
Washington University School of Medicine
St Louis, Missouri, United States
Mercy Hospital Saint Louis
St Louis, Missouri, United States
Children's Hospital and Medical Center of Omaha
Omaha, Nebraska, United States
University of Nebraska Medical Center
Omaha, Nebraska, United States
University Medical Center of Southern Nevada
Las Vegas, Nevada, United States
Sunrise Hospital and Medical Center
Las Vegas, Nevada, United States
Alliance for Childhood Diseases/Cure 4 the Kids Foundation
Las Vegas, Nevada, United States
Summerlin Hospital Medical Center
Las Vegas, Nevada, United States
Renown Regional Medical Center
Reno, Nevada, United States
Hackensack University Medical Center
Hackensack, New Jersey, United States
Morristown Medical Center
Morristown, New Jersey, United States
Rutgers Cancer Institute of New Jersey-Robert Wood Johnson University Hospital
New Brunswick, New Jersey, United States
Saint Joseph's Regional Medical Center
Paterson, New Jersey, United States
Albany Medical Center
Albany, New York, United States
Roswell Park Cancer Institute
Buffalo, New York, United States
NYU Langone Hospital - Long Island
Mineola, New York, United States
The Steven and Alexandra Cohen Children's Medical Center of New York
New Hyde Park, New York, United States
NYP/Columbia University Medical Center/Herbert Irving Comprehensive Cancer Center
New York, New York, United States
Memorial Sloan Kettering Cancer Center
New York, New York, United States
NYP/Weill Cornell Medical Center
New York, New York, United States
University of Rochester
Rochester, New York, United States
Stony Brook University Medical Center
Stony Brook, New York, United States
State University of New York Upstate Medical University
Syracuse, New York, United States
Montefiore Medical Center - Moses Campus
The Bronx, New York, United States
New York Medical College
Valhalla, New York, United States
Mission Hospital
Asheville, North Carolina, United States
UNC Lineberger Comprehensive Cancer Center
Chapel Hill, North Carolina, United States
Carolinas Medical Center/Levine Cancer Institute
Charlotte, North Carolina, United States
Novant Health Presbyterian Medical Center
Charlotte, North Carolina, United States
East Carolina University
Greenville, North Carolina, United States
Wake Forest University Health Sciences
Winston-Salem, North Carolina, United States
Sanford Broadway Medical Center
Fargo, North Dakota, United States
Children's Hospital Medical Center of Akron
Akron, Ohio, United States
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, United States
Rainbow Babies and Childrens Hospital
Cleveland, Ohio, United States
Cleveland Clinic Foundation
Cleveland, Ohio, United States
Nationwide Children's Hospital
Columbus, Ohio, United States
Dayton Children's Hospital
Dayton, Ohio, United States
ProMedica Toledo Hospital/Russell J Ebeid Children's Hospital
Toledo, Ohio, United States
University of Oklahoma Health Sciences Center
Oklahoma City, Oklahoma, United States
Legacy Emanuel Children's Hospital
Portland, Oregon, United States
Oregon Health and Science University
Portland, Oregon, United States
Lehigh Valley Hospital-Cedar Crest
Allentown, Pennsylvania, United States
Geisinger Medical Center
Danville, Pennsylvania, United States
Penn State Children's Hospital
Hershey, Pennsylvania, United States
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States
Saint Christopher's Hospital for Children
Philadelphia, Pennsylvania, United States
Children's Hospital of Pittsburgh of UPMC
Pittsburgh, Pennsylvania, United States
Rhode Island Hospital
Providence, Rhode Island, United States
Prisma Health Richland Hospital
Columbia, South Carolina, United States
BI-LO Charities Children's Cancer Center
Greenville, South Carolina, United States
Sanford USD Medical Center - Sioux Falls
Sioux Falls, South Dakota, United States
T C Thompson Children's Hospital
Chattanooga, Tennessee, United States
East Tennessee Childrens Hospital
Knoxville, Tennessee, United States
Saint Jude Children's Research Hospital
Memphis, Tennessee, United States
The Children's Hospital at TriStar Centennial
Nashville, Tennessee, United States
Vanderbilt University/Ingram Cancer Center
Nashville, Tennessee, United States
Texas Tech University Health Sciences Center-Amarillo
Amarillo, Texas, United States
Dell Children's Medical Center of Central Texas
Austin, Texas, United States
Driscoll Children's Hospital
Corpus Christi, Texas, United States
Medical City Dallas Hospital
Dallas, Texas, United States
UT Southwestern/Simmons Cancer Center-Dallas
Dallas, Texas, United States
El Paso Children's Hospital
El Paso, Texas, United States
Baylor College of Medicine/Dan L Duncan Comprehensive Cancer Center
Houston, Texas, United States
M D Anderson Cancer Center
Houston, Texas, United States
Covenant Children's Hospital
Lubbock, Texas, United States
UMC Cancer Center / UMC Health System
Lubbock, Texas, United States
Children's Hospital of San Antonio
San Antonio, Texas, United States
Methodist Children's Hospital of South Texas
San Antonio, Texas, United States
Primary Children's Hospital
Salt Lake City, Utah, United States
University of Virginia Cancer Center
Charlottesville, Virginia, United States
Inova Fairfax Hospital
Falls Church, Virginia, United States
Children's Hospital of The King's Daughters
Norfolk, Virginia, United States
VCU Massey Comprehensive Cancer Center
Richmond, Virginia, United States
Carilion Children's
Roanoke, Virginia, United States
Seattle Children's Hospital
Seattle, Washington, United States
Providence Sacred Heart Medical Center and Children's Hospital
Spokane, Washington, United States
Mary Bridge Children's Hospital and Health Center
Tacoma, Washington, United States
Madigan Army Medical Center
Tacoma, Washington, United States
West Virginia University Charleston Division
Charleston, West Virginia, United States
University of Wisconsin Carbone Cancer Center - University Hospital
Madison, Wisconsin, United States
Marshfield Medical Center-Marshfield
Marshfield, Wisconsin, United States
John Hunter Children's Hospital
Hunter Regional Mail Centre, New South Wales, Australia
The Children's Hospital at Westmead
Westmead, New South Wales, Australia
Monash Medical Center-Clayton Campus
Clayton, Victoria, Australia
Royal Children's Hospital
Parkville, Victoria, Australia
Perth Children's Hospital
Perth, Western Australia, Australia
University of Alberta Hospital
Edmonton, Alberta, Canada
CancerCare Manitoba
Winnipeg, Manitoba, Canada
IWK Health Centre
Halifax, Nova Scotia, Canada
McMaster Children's Hospital at Hamilton Health Sciences
Hamilton, Ontario, Canada
Children's Hospital
London, Ontario, Canada
Children's Hospital of Eastern Ontario
Ottawa, Ontario, Canada
Hospital for Sick Children
Toronto, Ontario, Canada
The Montreal Children's Hospital of the MUHC
Montreal, Quebec, Canada
Centre Hospitalier Universitaire Sainte-Justine
Montreal, Quebec, Canada
CHU de Quebec-Centre Hospitalier de l'Universite Laval (CHUL)
Québec, , Canada
HIMA San Pablo Oncologic Hospital
Caguas, , Puerto Rico
Countries
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Other Identifiers
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NCI-2018-03439
Identifier Type: REGISTRY
Identifier Source: secondary_id
AAML18P1
Identifier Type: OTHER
Identifier Source: secondary_id
AAML18P1
Identifier Type: OTHER
Identifier Source: secondary_id
AAML18P1
Identifier Type: -
Identifier Source: org_study_id