Last updated on May 2018

Gene Therapy for Wiskott-Aldrich Syndrome (WAS)


Brief description of study

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

Detailed Study Description

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.

Clinical Study Identifier: NCT01347242

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Recruitment Status: Open


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