This Phase 1/2 open follow-up study monitors patients who were enrolled in the hematopoietic stem cell gene therapy trial for Wiskott-Aldrich Syndrome and treated with autologous CD34+ cells transduced with the w1.6_hWASP_WPRE (VSVg) lentiviral vector. Its purpose is long-term safety follow-up. Participants are patients from the initial Phase 1/2 WAS studies conducted in France and the United Kingdom (GTG002.07 and GTG003.08) who, or whose parents or guardians, have provided informed consent.
The summary above is a plain-language rendering of the official record. The original English title is shown for reference.
Criteria are reproduced from the ClinicalTrials.gov record in the original English. Only the trial team can determine eligibility.
Inclusion Criteria: * Patients enrolled in the initial phase I/II WAS conducted in France and United Kingdom (GTG002.07 and GTG003.08). * Parents, guardians or patient signed informed consent, guardians or patient signed informed consent Exclusion Criteria: • Parents, guardians, patients unwilling to return for the follow up study period.