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.
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AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
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 )