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

Interventional


Primary Outcome

Improvement in the eczema status

Secondary Outcome

 Occurrence and type of adverse events

Condition

Wiskott-Aldrich Syndrome

Intervention

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


Publications

* 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

Genetic

Estimated Enrollment

5

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
             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
      

Gender

Male

Ages

N/A - N/A

Accepts Healthy Volunteers

No

Contacts

, , 

Location Countries

France

Location Countries

France

Administrative Informations


NCT ID

NCT01347346

Organization ID

GTG003.08

Secondary IDs

2009-011152-22

Responsible Party

Sponsor

Study Sponsor

Genethon

Collaborators

 Hôpital Necker-Enfants Malades

Study Sponsor

, , 


Verification Date

May 2018