Gene Therapy for WAS

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Brief Title

Gene Therapy for WAS

Official Title

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

Brief Summary

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

Detailed 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.

Study Phase

Phase 1/Phase 2

Study Type


Primary Outcome

Improvement in the eczema status

Secondary Outcome

 Occurrence and type of adverse events


Wiskott-Aldrich Syndrome


Autologous CD34 positive cells transduced with a lentiviral vector containing human WAS gene


* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information

Recruitment Status


Estimated Enrollment


Start Date

May 2011

Completion Date

January 9, 2017

Primary Completion Date

January 13, 2016

Eligibility Criteria

        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

          -  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




N/A - N/A

Accepts Healthy Volunteers



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Location Countries


Location Countries


Administrative Informations



Organization ID


Secondary IDs


Responsible Party


Study Sponsor



 Hôpital Necker-Enfants Malades

Study Sponsor

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Verification Date

May 2018