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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/ct2/show/NCT00959283




Registration number
NCT00959283
Ethics application status
Date submitted
13/08/2009
Date registered
14/08/2009
Date last updated
3/10/2023

Titles & IDs
Public title
Study of Blood Samples From Newborns With Down Syndrome
Scientific title
Biology Study of Transient Myeloproliferative Disorder (TMD) in Children With Down Syndrome (DS)
Secondary ID [1] 0 0
NCI-2011-02193
Secondary ID [2] 0 0
AAML08B1
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Myeloid Proliferations Associated With Down Syndrome 0 0
Condition category
Condition code
Human Genetics and Inherited Disorders 0 0 0 0
Down's syndrome
Other 0 0 0 0
Research that is not of generic health relevance and not applicable to specific health categories listed above

Intervention/exposure
Study type
Observational
Patient registry
Target follow-up duration
Target follow-up type
Description of intervention(s) / exposure
Other interventions - Diagnostic Laboratory Biomarker Analysis
Other interventions - Pharmacological Study

Ancillary-correlative - 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.


Other interventions: Diagnostic Laboratory Biomarker Analysis
Correlative studies

Other interventions: Pharmacological Study
Correlative studies
Intervention code [1] 0 0
Other interventions
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Event-free survival
Timepoint [1] 0 0
Up to 5 years
Secondary outcome [1] 0 0
Overall survival
Timepoint [1] 0 0
Up to 5 years
Secondary outcome [2] 0 0
Incidence of TMD-related mortality
Timepoint [2] 0 0
Up to 5 years
Secondary outcome [3] 0 0
Incidence of subsequent leukemia for patients with resolved TMD
Timepoint [3] 0 0
Up to 5 years

Eligibility
Key inclusion criteria
- 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
Minimum age
No limit
Maximum age
90 Days
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria

Study design
Purpose
Duration
Selection
Timing
Prospective
Statistical methods / analysis

Recruitment
Recruitment status
Completed
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
23/02/2009
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
30/09/2023
Sample size
Target
Accrual to date
Final
180
Recruitment in Australia
Recruitment state(s)
VIC,WA
Recruitment hospital [1] 0 0
Monash Medical Center-Clayton Campus - Clayton
Recruitment hospital [2] 0 0
Royal Children's Hospital - Parkville
Recruitment hospital [3] 0 0
Princess Margaret Hospital for Children - Perth
Recruitment postcode(s) [1] 0 0
3168 - Clayton
Recruitment postcode(s) [2] 0 0
3052 - Parkville
Recruitment postcode(s) [3] 0 0
6008 - Perth
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
Alabama
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United States of America
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Alaska
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Arizona
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Arkansas
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California
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Colorado
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Connecticut
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Delaware
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District of Columbia
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Florida
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Georgia
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Hawaii
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Idaho
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Illinois
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Indiana
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Iowa
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Kentucky
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Louisiana
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Maine
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Mississippi
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Missouri
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Nebraska
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Nevada
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New Hampshire
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New Jersey
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New Mexico
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New York
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North Carolina
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North Dakota
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Ohio
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Oklahoma
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Oregon
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Pennsylvania
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Rhode Island
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South Carolina
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South Dakota
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Tennessee
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Texas
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Utah
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Vermont
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Washington
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West Virginia
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Wisconsin
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British Columbia
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Canada
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Manitoba
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Canada
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Newfoundland and Labrador
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Canada
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Nova Scotia
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Canada
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Ontario
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Quebec
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Puerto Rico
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San Juan

Funding & Sponsors
Primary sponsor type
Other
Name
Children's Oncology Group
Address
Country
Other collaborator category [1] 0 0
Government body
Name [1] 0 0
National Cancer Institute (NCI)
Address [1] 0 0
Country [1] 0 0

Ethics approval
Ethics application status

Summary
Brief summary
This research study is looking at blood samples from newborns with Down syndrome. Studying
the genes expressed in samples of blood from patients with Down syndrome may help doctors
identify biomarkers related to cancer.
Trial website
https://clinicaltrials.gov/ct2/show/NCT00959283
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
April D Sorrell
Address 0 0
Children's Oncology Group
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries