An open follow up study of patients enrolled in the Phase 1/2 clinical trial of haematopoietic stem cell gene therapy for the Wiskott-Aldrich Syndrome and treated with autologous CD34+ cells transduced with the w1.6\_hWASP\_WPRE (VSVg) lentiviral vector.
Sex
MALE
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
Incidence and type of SAEs
Timeframe: yearly from 3 years to 15 years
Lentiviral integration sites
Timeframe: yearly from 3 years to 15 years (from 11 to 15 yearly time points, only in case of Advers Events of Special Interest)
Vector copy numbers
Timeframe: yearly from 3 years to 15 years (from 11 to 15 yearly time points, only in case of Advers Events of Special Interest)
Replication competent lentivirus (RCL)
Timeframe: yearly from 3 years to 15 years (from 11 to 15 yearly time points, only in case of Advers Events of Special Interest)
Change in medical conditions
Timeframe: yearly from 3 years to 10 years
Key medical events related to WAS
Timeframe: yearly from 3 years to 10 years
Hematological reconstitution
Timeframe: yearly from 3 years to 10 years
Reconstitution of cell mediated and humoral immunity
Timeframe: yearly from 3 years to 10 years (from 3 years to 5 years for PHA and candida )