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Study of Blood Samples From Newborns With Down Syndrome

This study has been completed.
Sponsor:
Collaborator:
National Cancer Institute (NCI)
Information provided by (Responsible Party):
Children's Oncology Group
ClinicalTrials.gov Identifier:
NCT00959283
First received: August 13, 2009
Last updated: May 13, 2016
Last verified: May 2016
  Purpose

RATIONALE: Studying the genes expressed in samples of blood from patients with Down syndrome may help doctors identify biomarkers related to cancer.

PURPOSE: This research study is looking at blood samples from newborns with Down syndrome.


Condition Intervention
Leukemia
Genetic: DNA analysis
Genetic: RNA analysis
Genetic: cytogenetic analysis
Genetic: fluorescence in situ hybridization
Genetic: gene expression analysis
Genetic: microarray analysis
Genetic: mutation analysis
Genetic: polymerase chain reaction
Genetic: polymorphism analysis
Other: flow cytometry
Other: laboratory biomarker analysis
Other: pharmacological study

Study Type: Observational
Study Design: Observational Model: Case Control
Time Perspective: Prospective
Official Title: Biology Study of Transient Myeloproliferative Disorder (TMD) in Children With Down Syndrome (DS)

Resource links provided by NLM:


Further study details as provided by Children's Oncology Group:

Primary Outcome Measures:
  • Event-free survival [ Time Frame: length of study ] [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • Overall survival [ Time Frame: length of study ] [ Designated as safety issue: No ]
  • Transient myeloproliferative disorder (TMD)-related mortality [ Time Frame: length of study ] [ Designated as safety issue: No ]
  • Incidence of subsequent leukemia for patients with resolved TMD [ Time Frame: length of study ] [ Designated as safety issue: No ]

Biospecimen Retention:   Samples With DNA
blood

Enrollment: 180
Study Start Date: February 2009
Study Completion Date: May 2016
Primary Completion Date: May 2016 (Final data collection date for primary outcome measure)
Detailed Description:

OBJECTIVES:

  • To further our biological understanding of the natural history of transient myeloproliferative disorder (TMD) and its relationship to subsequent leukemia by facilitating the development of a TMD cell and protein bank, and repository of DNA/RNA from megakaryoblasts for future biological studies.
  • To investigate the biology of TMD molecular changes associated with resolution of TMD or its conversion to acute myeloid leukemia within each mortality-risk group by conducting GATA1 mutational analyses, hematopoiesis clonality studies, assessment of RAS mutations, and genomic instability studies using glycophorin A assays.
  • To determine if high-resolution microarray genomic analysis of TMD blasts (using Affymetrix SNP Genechip technology to assess gene expression, copy number variation, and loss of heterozygosity) can predict the development of subsequent leukemia.
  • To determine the relationship of minimal residual disease (monitored by peripheral blood flow cytometry and GATA1 mutational studies) to clinical remission status and development of subsequent leukemia within each mortality-risk group of TMD patients.
  • To evaluate the relationship between karyotype (including FISH analysis) and subsequent leukemia in TMD patients.
  • To examine pharmacogenetics and in vitro drug sensitivity to cytarabine (MTT assay) in blasts from TMD patients.
  • To examine the relationship of functional polymorphisms in Phase I and Phase II drug detoxification genes, DNA repair, and DNA synthesis pathways that may modify susceptibility to leukemia and outcome in TMD patients.
  • To determine the relationship between fibrosis-associated serum factors (e.g., platelet-derived growth factor, transforming growth factor beta, N-terminal peptide of III procollagen, type IV collagen, and hyaluronic acid) and event-free survival.

OUTLINE: This is a multicenter study.

Patients undergo peripheral blood collection periodically for biomarker analysis. Samples are analyzed for GATA1 mutations by real-time PCR, polymorphisms, cytogenetics, - and K-RAS mutations, gene expression, drug sensitivity patterns, and minimal residual disease by flow cytometry.

Patients are followed up periodically for 5 years.

  Eligibility

Ages Eligible for Study:   up to 1 Year   (Child)
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population
See Eligibility criteria.
Criteria

DISEASE CHARACTERISTICS:

  • Diagnosis of transient myeloproliferative disorder (TMD) at < 90 days of age and meeting 1 of the following criteria:

    • A diagnosis of Down syndrome or Down syndrome mosaicism AND non-erythroid and non-lymphoid blasts (any amount) in the peripheral blood verified with a second sample

      • Patients with typical physical characteristics of Down syndrome are allowed before cytogenetic or FISH confirmation of the diagnosis
    • Trisomy 21-positive leukemic blasts documented by biopsy of any organ (including > 5% non-erythroid/non-lymphoid blasts documented by bone marrow aspirate or biopsy)

      • Infants with isolated trisomy 21 positivity identified only in the leukemic blasts are allowed
  • Institutional immunophenotype characterization is required for study enrollment

PATIENT CHARACTERISTICS:

  • Not specified

PRIOR CONCURRENT THERAPY:

  • Not specified
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT00959283

  Hide Study Locations
Locations
United States, Alabama
UAB Comprehensive Cancer Center
Birmingham, Alabama, United States, 35294
University of South Alabama Mitchell Cancer Institute
Mobile, Alabama, United States, 36604
United States, Arizona
Phoenix Children's Hospital
Phoenix, Arizona, United States, 85016-7710
United States, Arkansas
Arkansas Cancer Research Center at University of Arkansas for Medical Sciences
Little Rock, Arkansas, United States, 72205
United States, California
Loma Linda University Cancer Institute at Loma Linda University Medical Center
Loma Linda, California, United States, 92354
Jonathan Jaques Children's Cancer Center at Miller Children's Hospital
Long Beach, California, United States, 90801
Childrens Hospital Los Angeles
Los Angeles, California, United States, 90027
Southern California Permanente Medical Group
Los Angeles, California, United States, 90027
Samuel Oschin Comprehensive Cancer Institute at Cedars-Sinai Medical Center
Los Angeles, California, United States, 90048-1865
Children's Hospital Central California
Madera, California, United States, 93638-8762
Children's Hospital and Research Center Oakland
Oakland, California, United States, 94609
Children's Hospital of Orange County
Orange, California, United States, 92868
Lucile Packard Children's Hospital at Stanford University Medical Center
Palo Alto, California, United States, 95798
Sutter Cancer Center
Sacramento, California, United States, 95816
University of California Davis Cancer Center
Sacramento, California, United States, 95817
Rady Children's Hospital - San Diego
San Diego, California, United States, 92123-4282
UCSF Helen Diller Family Comprehensive Cancer Center
San Francisco, California, United States, 94115
United States, Colorado
Children's Hospital Colorado Center for Cancer and Blood Disorders
Aurora, Colorado, United States, 80045
Presbyterian - St. Luke's Medical Center
Denver, Colorado, United States, 80218
United States, Connecticut
Connecticut Children's Medical Center
Hartford, Connecticut, United States, 06106
Yale Cancer Center
New Haven, Connecticut, United States, 06520-8028
United States, Delaware
Alfred I. duPont Hospital for Children
Wilmington, Delaware, United States, 19803
United States, District of Columbia
Lombardi Comprehensive Cancer Center at Georgetown University Medical Center
Washington, District of Columbia, United States, 20007
United States, Florida
Lee Cancer Care of Lee Memorial Health System
Fort Myers, Florida, United States, 33901
Memorial Cancer Institute at Memorial Regional Hospital
Hollywood, Florida, United States, 33021
Nemours Children's Clinic
Jacksonville, Florida, United States, 32207
Florida Hospital Cancer Institute at Florida Hospital Orlando
Orlando, Florida, United States, 32803-1273
M.D. Anderson Cancer Center at Orlando
Orlando, Florida, United States, 32806
Nemours Children's Clinic - Orlando
Orlando, Florida, United States, 32806
Nemours Children's Clinic - Pensacola
Pensacola, Florida, United States, 32504
All Children's Hospital
Saint Petersburg, Florida, United States, 33701
St. Joseph's Cancer Institute at St. Joseph's Hospital
Tampa, Florida, United States, 33607
Kaplan Cancer Center at St. Mary's Medical Center
West Palm Beach, Florida, United States, 33407
United States, Georgia
AFLAC Cancer Center and Blood Disorders Service of Children's Healthcare of Atlanta - Egleston Campus
Atlanta, Georgia, United States, 30322
Curtis and Elizabeth Anderson Cancer Institute at Memorial Health University Medical Center
Savannah, Georgia, United States, 31403-3089
United States, Hawaii
Cancer Research Center of Hawaii
Honolulu, Hawaii, United States, 96813
United States, Idaho
Mountain States Tumor Institute at St. Luke's Regional Medical Center
Boise, Idaho, United States, 83712-6297
United States, Illinois
Children's Memorial Hospital - Chicago
Chicago, Illinois, United States, 60611
University of Illinois Cancer Center
Chicago, Illinois, United States, 60612-7243
University of Chicago Cancer Research Center
Chicago, Illinois, United States, 60637-1470
Keyser Family Cancer Center at Advocate Hope Children's Hospital
Oak Lawn, Illinois, United States, 60453
Simmons Cooper Cancer Institute
Springfield, Illinois, United States, 62794-9677
United States, Indiana
Riley's Children Cancer Center at Riley Hospital for Children
Indianapolis, Indiana, United States, 46202
St. Vincent Indianapolis Hospital
Indianapolis, Indiana, United States, 46260
United States, Iowa
Blank Children's Hospital
Des Moines, Iowa, United States, 50309
Holden Comprehensive Cancer Center at University of Iowa
Iowa City, Iowa, United States, 52242-1002
United States, Kentucky
Lucille P. Markey Cancer Center at University of Kentucky
Lexington, Kentucky, United States, 40536-0093
Kosair Children's Hospital
Louisville, Kentucky, United States, 40232
United States, Louisiana
Tulane Cancer Center Office of Clinical Research
Alexandria, Louisiana, United States, 71315-3198
Children's Hospital of New Orleans
New Orleans, Louisiana, United States, 70118
United States, Maine
CancerCare of Maine at Eastern Maine Medical Center
Bangor, Maine, United States, 04401
United States, Maryland
Alvin and Lois Lapidus Cancer Institute at Sinai Hospital
Baltimore, Maryland, United States, 21215
Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins
Baltimore, Maryland, United States, 21231-2410
National Naval Medical Center
Bethesda, Maryland, United States, 20889-5600
United States, Massachusetts
Floating Hospital for Children at Tufts - New England Medical Center
Boston, Massachusetts, United States, 02111
United States, Michigan
C.S. Mott Children's Hospital at University of Michigan Medical Center
Ann Arbor, Michigan, United States, 48109-0286
Barbara Ann Karmanos Cancer Institute
Detroit, Michigan, United States, 48201-1379
Hurley Medical Center
Flint, Michigan, United States, 48503
Helen DeVos Children's Hospital at Spectrum Health
Grand Rapids, Michigan, United States, 49503
Van Elslander Cancer Center at St. John Hospital and Medical Center
Grosse Pointe Woods, Michigan, United States, 48236
Bronson Methodist Hospital
Kalamazoo, Michigan, United States, 49007-5381
Breslin Cancer Center at Ingham Regional Medical Center
Lansing, Michigan, United States, 48910
United States, Minnesota
Children's Hospitals and Clinics of Minnesota - Minneapolis
Minneapolis, Minnesota, United States, 55404
Mayo Clinic Cancer Center
Rochester, Minnesota, United States, 55905
United States, Mississippi
University of Mississippi Cancer Clinic
Jackson, Mississippi, United States, 39216-4505
United States, Missouri
Ellis Fischel Cancer Center at University of Missouri - Columbia
Columbia, Missouri, United States, 65203
Children's Mercy Hospital
Kansas City, Missouri, United States, 64108
David C. Pratt Cancer Center at St. John's Mercy
Saint Louis, Missouri, United States, 63141
Cardinal Glennon Children's Hospital
St. Louis, Missouri, United States, 63104
Siteman Cancer Center at Barnes-Jewish Hospital - Saint Louis
St. Louis, Missouri, United States, 63110
United States, Nebraska
Children's Hospital
Omaha, Nebraska, United States, 68114-4113
UNMC Eppley Cancer Center at the University of Nebraska Medical Center
Omaha, Nebraska, United States, 68198-6805
United States, Nevada
CCOP - Nevada Cancer Research Foundation
Las Vegas, Nevada, United States, 89109-2306
United States, New Hampshire
Norris Cotton Cancer Center at Dartmouth-Hitchcock Medical Center
Lebanon, New Hampshire, United States, 03756-0002
United States, New Jersey
Hackensack University Medical Center Cancer Center
Hackensack, New Jersey, United States, 07601
St. Barnabas Medical Center Cancer Center
Livingston, New Jersey, United States, 07039
Saint Peter's University Hospital
New Brunswick, New Jersey, United States, 08901
Cancer Institute of New Jersey at UMDNJ - Robert Wood Johnson Medical School
New Brunswick, New Jersey, United States, 08903
Newark Beth Israel Medical Center
Newark, New Jersey, United States, 07112
St. Joseph's Hospital and Medical Center
Paterson, New Jersey, United States, 07503
Carol G. Simon Cancer Center at Morristown Memorial Hospital
Summit, New Jersey, United States, 07901
United States, New Mexico
University of New Mexico Cancer Center
Albuquerque, New Mexico, United States, 87131-5636
United States, New York
Albany Medical Center Hospital
Albany, New York, United States, 12208-3419
Albert Einstein Cancer Center at Albert Einstein College of Medicine
Bronx, New York, United States, 10461
Roswell Park Cancer Institute
Buffalo, New York, United States, 14263-0001
Winthrop University Hospital
Mineola, New York, United States, 11501
Schneider Children's Hospital
New Hyde Park, New York, United States, 11040
NYU Cancer Institute at New York University Medical Center
New York, New York, United States, 10016
Herbert Irving Comprehensive Cancer Center at Columbia University Medical Center
New York, New York, United States, 10032
James P. Wilmot Cancer Center at University of Rochester Medical Center
Rochester, New York, United States, 14642
Stony Brook University Cancer Center
Stony Brook, New York, United States, 11794-9446
SUNY Upstate Medical University Hospital
Syracuse, New York, United States, 13210
United States, North Carolina
Mission Hospitals - Memorial Campus
Asheville, North Carolina, United States, 28801
Lineberger Comprehensive Cancer Center at University of North Carolina - Chapel Hill
Chapel Hill, North Carolina, United States, 27599-7295
Blumenthal Cancer Center at Carolinas Medical Center
Charlotte, North Carolina, United States, 28232-2861
Presbyterian Cancer Center at Presbyterian Hospital
Charlotte, North Carolina, United States, 28233-3549
Duke Cancer Institute
Durham, North Carolina, United States, 27710
Leo W. Jenkins Cancer Center at ECU Medical School
Greenville, North Carolina, United States, 27834
Wake Forest University Comprehensive Cancer Center
Winston-Salem, North Carolina, United States, 27157-1096
United States, North Dakota
CCOP - MeritCare Hospital
Fargo, North Dakota, United States, 58122
United States, Ohio
Akron Children's Hospital
Akron, Ohio, United States, 44308-1062
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, United States, 45229-3039
Rainbow Babies and Children's Hospital
Cleveland, Ohio, United States, 44106-5000
Cleveland Clinic Taussig Cancer Center
Cleveland, Ohio, United States, 44195
Nationwide Children's Hospital
Columbus, Ohio, United States, 43205-2696
Dayton Children's - Dayton
Dayton, Ohio, United States, 45404-1815
Toledo Hospital
Toledo, Ohio, United States, 43606
Mercy Children's Hospital
Toledo, Ohio, United States, 43608
United States, Oklahoma
Oklahoma University Cancer Institute
Oklahoma City, Oklahoma, United States, 73104
Natalie Warren Bryant Cancer Center at St. Francis Hospital
Tulsa, Oklahoma, United States, 74136
United States, Oregon
Legacy Emanuel Children's Hospital
Portland, Oregon, United States, 97227
Knight Cancer Institute at Oregon Health and Science University
Portland, Oregon, United States, 97239-3098
United States, Pennsylvania
Geisinger Cancer Institute at Geisinger Health
Danville, Pennsylvania, United States, 17822-0001
Penn State Children's Hospital
Hershey, Pennsylvania, United States, 17033-0850
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States, 19104
Children's Hospital of Pittsburgh of UPMC
Pittsburgh, Pennsylvania, United States, 15213
United States, Rhode Island
Rhode Island Hospital Comprehensive Cancer Center
Providence, Rhode Island, United States, 02903
United States, South Carolina
Hollings Cancer Center at Medical University of South Carolina
Charleston, South Carolina, United States, 29425
Palmetto Health South Carolina Cancer Center
Columbia, South Carolina, United States, 29203
United States, South Dakota
Sanford Cancer Center at Sanford USD Medical Center
Sioux Falls, South Dakota, United States, 57117-5039
United States, Tennessee
T.C. Thompson Children's Hospital
Chattanooga, Tennessee, United States, 37403
East Tennessee Children's Hospital
Knoxville, Tennessee, United States, 37916
St. Jude Children's Research Hospital
Memphis, Tennessee, United States, 38105
Vanderbilt-Ingram Cancer Center
Nashville, Tennessee, United States, 37232-6838
United States, Texas
Texas Tech University Health Sciences Center School of Medicine - Amarillo
Amarillo, Texas, United States, 79106
Driscoll Children's Hospital
Corpus Christi, Texas, United States, 78411
Medical City Dallas Hospital
Dallas, Texas, United States, 75230
Simmons Comprehensive Cancer Center at University of Texas Southwestern Medical Center - Dallas
Dallas, Texas, United States, 75390
Brooke Army Medical Center
Fort Sam Houston, Texas, United States, 78234-6200
Cook Children's Medical Center - Fort Worth
Fort Worth, Texas, United States, 76104
Baylor University Medical Center - Houston
Houston, Texas, United States, 77030-2399
University of Texas Health Science Center at San Antonio
San Antonio, Texas, United States, 78207
United States, Utah
Primary Children's Medical Center
Salt Lake City, Utah, United States, 84113-1100
United States, Vermont
Fletcher Allen Health Care - University Health Center Campus
Burlington, Vermont, United States, 05401
United States, Virginia
University of Virginia Cancer Center
Charlottesville, Virginia, United States, 22908
Inova Fairfax Hospital
Falls Church, Virginia, United States, 22042-3300
Children's Hospital of The King's Daughters
Norfolk, Virginia, United States, 23507-1971
Carilion Medical Center for Children at Roanoke Community Hospital
Roanoke, Virginia, United States, 24014
United States, Washington
Children's Hospital and Regional Medical Center - Seattle
Seattle, Washington, United States, 98105
Providence Cancer Center at Sacred Heart Medical Center
Spokane, Washington, United States, 99220-2555
Mary Bridge Children's Hospital and Health Center - Tacoma
Tacoma, Washington, United States, 98405
United States, West Virginia
West Virginia University Health Sciences Center - Charleston
Charleston, West Virginia, United States, 25302
United States, Wisconsin
St. Vincent Hospital Regional Cancer Center
Green Bay, Wisconsin, United States, 54307-3508
Midwest Children's Cancer Center at Children's Hospital of Wisconsin
Milwaukee, Wisconsin, United States, 53226
Australia, Victoria
Royal Children's Hospital
Parkville, Victoria, Australia, 3052
Australia, Western Australia
Princess Margaret Hospital for Children
Perth, Western Australia, Australia, 6001
Canada, British Columbia
Children's and Women's Hospital of British Columbia
Vancouver, British Columbia, Canada, V6H 3V4
Canada, Manitoba
CancerCare Manitoba
Winnipeg, Manitoba, Canada, R3E 0V9
Canada, Newfoundland and Labrador
Janeway Children's Health and Rehabilitation Centre
St. John's, Newfoundland and Labrador, Canada, A1B 3V6
Canada, Nova Scotia
IWK Health Centre
Halifax, Nova Scotia, Canada, B3J 3G9
Canada, Ontario
McMaster Children's Hospital at Hamilton Health Sciences
Hamilton, Ontario, Canada, L8N 3Z5
Cancer Centre of Southeastern Ontario at Kingston General Hospital
Kingston, Ontario, Canada, K7L 2V7
Children's Hospital of Western Ontario
London, Ontario, Canada, N6A 5W9
Children's Hospital of Eastern Ontario
Ottawa, Ontario, Canada, K1H 8L1
Hospital for Sick Children
Toronto, Ontario, Canada, M5G 1X8
Canada, Quebec
Montreal Children's Hospital at McGill University Health Center
Montreal, Quebec, Canada, H3H 1P3
Hopital Sainte Justine
Montreal, Quebec, Canada, H3T 1C5
Canada
Centre Hospitalier Universitaire de Quebec
Quebec, Canada, G1V 4G2
Sponsors and Collaborators
Children's Oncology Group
National Cancer Institute (NCI)
Investigators
Principal Investigator: April D. Sorrell, MD City of Hope Comprehensive Cancer Center
  More Information

Responsible Party: Children's Oncology Group
ClinicalTrials.gov Identifier: NCT00959283     History of Changes
Other Study ID Numbers: AAML08B1  COG-AAML08B1  CDR0000636115  NCI-2011-02193 
Study First Received: August 13, 2009
Last Updated: May 13, 2016
Health Authority: United States: Food and Drug Administration

Keywords provided by Children's Oncology Group:
acute myeloid leukemia/transient myeloproliferative disorder

Additional relevant MeSH terms:
Down Syndrome
Myeloproliferative Disorders
Leukemoid Reaction
Intellectual Disability
Neurobehavioral Manifestations
Neurologic Manifestations
Nervous System Diseases
Abnormalities, Multiple
Congenital Abnormalities
Chromosome Disorders
Genetic Diseases, Inborn
Bone Marrow Diseases
Hematologic Diseases
Leukocytosis
Leukocyte Disorders

ClinicalTrials.gov processed this record on September 30, 2016