Title
Gene Therapy for Wiskott-Aldrich Syndrome (WAS)
Phase I/II Clinical Trial of Haematopoietic Stem Cell Gene Therapy for the Wiskott-Aldrich Syndrome
Phase
Phase 1/Phase 2Lead Sponsor
GenethonStudy Type
InterventionalStatus
Completed No Results PostedIndication/Condition
Wiskott-Aldrich SyndromeIntervention/Treatment
autologous cd34 positive cells transduced with a lentiviral vector containing human was gene ...Study Participants
6This 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.
transplantation of patient's autologous CD34+ cells transduced with lentiviral vector containing human WAS gene
autologous CD34 positive cells transduced with a lentiviral vector containing the human WAS 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 or of a 10/10 antigen HLA-matched unrelated donor or 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 antigen HLA-matched unrelated donor or cord blood contraindication to leukapheresis contraindication to bone marrow harvest contraindication to administration of conditioning medication HIV positive patient
