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Patients Treated for Wiskott-Aldrich Syndrome (WAS) Since 1990
Not Recruiting
Trial ID: NCT02064933
Purpose
Wiskott - Aldrich syndrome (WAS) is a rare serious medical condition that causes problems
both with the immune system and with easy bruising and bleeding. The immune abnormalities
cause patients with WAS to be very susceptible to infections. Depending on the specific type
of primary immune deficiency diseases, there are effective treatments, including antibiotics,
cellular therapy and gene therapy, but studies of large numbers of patients are needed to
determine the full range of causes, natural history, or the best methods of treatment for
long term success.
This multicenter study combines retrospective, prospective and cross-sectional analyses of
the transplant experiences for patients with WAS who have already received HCT since 1990, or
who will undergo Hematopoietic cell transplant (HCT) during the study period. The
retrospective and prospective portions of the study will address the impact of a number of
pre and post-transplant factors on post-transplant disease correction and ultimate benefit
from HCT and the cross-sectional portion of the study will assess the benefit of HCT 2 years
post-HCT in consenting surviving patients.
Official Title
Analysis of Patients Treated for Wiskott-Aldrich Syndrome Since January 1, 1990 (RDCRN PIDTC-6904)
Stanford Investigator(s)
Eligibility
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
Contact Information
Stanford University
School of Medicine
300 Pasteur Drive
Stanford,
CA
94305
Matthew Porteus, MD
650-725-6520