Eligibility Module
The Eligibility Module contains detailed information about who can participate in the clinical trial. This includes eligibility criteria, age restrictions, gender requirements, healthy volunteer status, and study population descriptions, helping researchers understand who is eligible to participate in the study.
Eligibility Module path is as follows:
Study -> Protocol Section -> Eligibility Module
Eligibility Module
Ignite Creation Date:
2025-12-25 @ 2:51 AM
Ignite Modification Date:
2025-12-25 @ 2:51 AM
NCT ID:
NCT02064933
Eligibility Criteria:
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.
Healthy Volunteers:
False
Sex:
MALE
Study:
NCT02064933
Study Brief:
Protocol Section:
NCT02064933