Clinical Trial: Study of Blood Samples From Newborns With Down Syndrome

Study Status: Recruiting
Recruit Status: Recruiting
Study Type: Observational

Official Title: Biology Study of Transient Myeloproliferative Disorder (TMD) in Children With Down Syndrome (DS)

Brief Summary:

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.


Detailed Summary:

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-ass
    Sponsor: Children's Oncology Group

    Current Primary Outcome: Event-free survival [ Time Frame: length of study ]

    Original Primary Outcome: Event-free survival

    Current Secondary Outcome:

    • Overall survival [ Time Frame: length of study ]
    • Transient myeloproliferative disorder (TMD)-related mortality [ Time Frame: length of study ]
    • Incidence of subsequent leukemia for patients with resolved TMD [ Time Frame: length of study ]


    Original Secondary Outcome:

    • Overall survival
    • Transient myeloproliferative disorder (TMD)-related mortality
    • Incidence of subsequent leukemia for patients with resolved TMD


    Information By: Children's Oncology Group

    Dates:
    Date Received: August 13, 2009
    Date Started: February 2009
    Date Completion: January 2100
    Last Updated: February 13, 2017
    Last Verified: February 2017