Gene Therapy for WAS

Phase I/II Clinical Trial of Haematopoietic Stem Cell Gene Therapy for the Wiskott-Aldrich Syndrome

This is a phase I/II study to evaluate the safety and efficacy of Hematopoietic Stem Cell genetherapy for the Wiskott-Aldrich Syndrome.

This clinical trial is an ex vivo gene therapy trial. The investigational product corresponds to autologous CD34+ cells transduced with a lentiviral vector harboring the human WASP gene.

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