This study evaluates the safety and efficacy of hematopoietic stem cell gene therapy for Wiskott-Aldrich Syndrome (WAS). The patient's own CD34-positive stem cells are modified in the laboratory with a lentiviral vector carrying the human WAS gene. The Phase 1/2 trial enrolled males of all ages with severe WAS (clinical score 3-5) or absent WAS protein, confirmed by WAS gene sequencing, for whom no HLA-genotypically identical bone marrow donor was found after 3 months of searching.
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: * males of all ages * severe WAS (clinical score 3-5) or absence of WAS protein in peripheral blood mononuclear cells determined by Western blotting and flow cytometry * molecular confirmation by WAS gene DNA sequencing * lack of HLA-genotypically identical bone marrow after 3 month search * lack of a 10/10 or 9/10 antigen HLA-matched unrelated donor after 3 month search * lack of a HLA-matched cord blood after 3 month search * parental, guardian, patient signed informed consent/assent * willing to return for follow-up * only for patients who have received previous allogenic hematopoietic stem cell transplant: * failed allogenic hematopoietic stem cell transplant * contraindication to repeat transplantation Exclusion Criteria: * patient with HLA-genotypically identical bone marrow * patient with 10/10 or 9/10 antigen HLA-matched unrelated donor or with HLA-matched cord blood * contraindication to leukapheresis * contraindication to bone marrow harvest * contraindication to administration of conditioning medication * HIV positive patient