Wiskott-Aldrich Syndrome Clinical Trial
Verified date | October 2003 |
Source | Office of Rare Diseases (ORD) |
Contact | n/a |
Is FDA regulated | No |
Health authority | United States: Federal Government |
Study type | Observational |
OBJECTIVES: I. Identify the molecular defects responsible for primary immunodeficiency
disorders.
II. Explore the mutations within each syndrome to better understand the genetics of these
disorders.
III. Study the function of the Wiskott-Aldrich syndrome proteins (WASP). IV. Design methods
to identify carriers and for prenatal diagnosis. V. Explore new avenues for therapy.
Status | Active, not recruiting |
Enrollment | 0 |
Est. completion date | |
Est. primary completion date | |
Accepts healthy volunteers | No |
Gender | Both |
Age group | N/A and older |
Eligibility |
PROTOCOL ENTRY CRITERIA: Primary immunodeficiency disease, e.g.: Leukocyte adhesion deficiency syndrome Wiskott-Aldrich syndrome X-linked agammaglobulinemia X-linked hyper IgM syndrome |
Primary Purpose: Screening
Country | Name | City | State |
---|---|---|---|
United States | University of Washington School of Medicine | Seattle | Washington |
Lead Sponsor | Collaborator |
---|---|
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) | University of Washington |
United States,
Miki H, Nonoyama S, Zhu Q, Aruffo A, Ochs HD, Takenawa T. Tyrosine kinase signaling regulates Wiskott-Aldrich syndrome protein function, which is essential for megakaryocyte differentiation. Cell Growth Differ. 1997 Feb;8(2):195-202. — View Citation
Zhu Q, Watanabe C, Liu T, Hollenbaugh D, Blaese RM, Kanner SB, Aruffo A, Ochs HD. Wiskott-Aldrich syndrome/X-linked thrombocytopenia: WASP gene mutations, protein expression, and phenotype. Blood. 1997 Oct 1;90(7):2680-9. — View Citation
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