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Not Recruiting
Trial ID: NCT02064933
Analysis of Patients Treated for Wiskott-Aldrich Syndrome Since January 1, 1990 (RDCRN PIDTC-6904)
Inclusion Criteria:
- WAS participants will be defined as males who have:
1. thrombocytopenia (< 100K) AND EITHER molecular diagnosis of WAS OR reduced WASP
expression; OR
2. thrombocytopenia (< 100K) AND positive family history consistent with WAS
diagnosis; OR
3. chronic thrombocytopenia (< 100K for minimum of 3 months) AND low mean platelet
volume (MPV below normal range for age) AND EITHER recurrent and/or severe
infections requiring treatment and/or eczema OR lack of antibody response to
polysaccharide antigens or low IgM.
- Longitudinal Analysis (Retrospective and Prospective)
1. Stratum A. Participants with WAS who have or will Receive HCT
- Participants with WAS who have received an HCT since January 1, 1990
2. Stratum B. Participants with WAS who have or will Receive Gene Transfer
- Participants in which the intention is to treat with gene transfer with
autologous modified cells
- Cross-Sectional Analysis (Strata A and B) 1. Participants with WAS who are surviving
and at least 2 years after the most recent HCT or gene therapy.
Exclusion Criteria:
- As this is a natural history study, for both the Longitudinal Analysis and the
Cross-Sectional Analysis we will not exclude any patients due to race or age who fit
the inclusion criteria.
Not Recruiting
Stanford University
School of Medicine
300 Pasteur Drive
Stanford,
CA
94305
Matthew Porteus, MD
650-725-6520