Trial record 1 of 2 for:    COG AAML08B1
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Study of Blood Samples From Newborns With Down Syndrome

This study is currently recruiting participants. (see Contacts and Locations)
Verified August 2014 by Children's Oncology Group
Sponsor:
Collaborator:
Information provided by (Responsible Party):
Children's Oncology Group
ClinicalTrials.gov Identifier:
NCT00959283
First received: August 13, 2009
Last updated: August 4, 2014
Last verified: August 2014

August 13, 2009
August 4, 2014
February 2009
December 2016   (final data collection date for primary outcome measure)
Event-free survival [ Time Frame: length of study ] [ Designated as safety issue: No ]
Event-free survival [ Designated as safety issue: No ]
Complete list of historical versions of study NCT00959283 on ClinicalTrials.gov Archive Site
  • 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 ]
  • Overall survival [ Designated as safety issue: No ]
  • Transient myeloproliferative disorder (TMD)-related mortality [ Designated as safety issue: No ]
  • Incidence of subsequent leukemia for patients with resolved TMD [ Designated as safety issue: No ]
Not Provided
Not Provided
 
Study of Blood Samples From Newborns With Down Syndrome
Biology Study of Transient Myeloproliferative Disorder (TMD) in Children With Down Syndrome (DS)

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.

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.

Observational
Observational Model: Case Control
Time Perspective: Prospective
Not Provided
Retention:   Samples With DNA
Description:

blood

Non-Probability Sample

See Eligibility criteria.

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
Not Provided
Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruiting
180
Not Provided
December 2016   (final data collection date for primary outcome measure)

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
Both
up to 1 Year
No
United States,   Australia,   Canada
 
NCT00959283
AAML08B1, COG-AAML08B1, CDR0000636115
No
Children's Oncology Group
Children's Oncology Group
National Cancer Institute (NCI)
Principal Investigator: April D. Sorrell, MD Beckman Research Institute
Children's Oncology Group
August 2014

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP