Long Term Safety Follow up of Haematopoietic Stem Cell Gene Therapy for the Wiskott Aldrich Syndrome

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Brief Title

Long Term Safety Follow up of Haematopoietic Stem Cell Gene Therapy for the Wiskott Aldrich Syndrome

Official Title

Long Term Safety Follow up of Patients Enrolled in the Phase I/II Clinical Trial of Haematopoietic Stem Cell Gene Therapy for the Wiskott Aldrich Syndrome (GTG002-07 and GTG003-08).

Brief Summary

      An open follow up study of patients enrolled in the Phase 1/2 clinical trial of
      haematopoietic stem cell gene therapy for the Wiskott-Aldrich Syndrome and treated with
      autologous CD34+ cells transduced with the w1.6_hWASP_WPRE (VSVg) lentiviral vector.
    


Study Phase

Phase 1/Phase 2

Study Type

Interventional


Primary Outcome

Incidence and type of SAEs

Secondary Outcome

 Need for associated treatments

Condition

Wiskott-Aldrich Syndrome

Intervention

Autologous CD34+ cells transduced with WASP lentiviral vector


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

10

Start Date

September 2014

Completion Date

October 2032

Primary Completion Date

October 2032

Eligibility Criteria

        Inclusion Criteria:

          -  Patients enrolled in the initial phase I/II WAS conducted in France and United Kingdom
             (GTG002.07 and GTG003.08).

          -  Parents, guardians or patient signed informed consent, guardians or patient signed
             informed consent

        Exclusion Criteria:

        • Parents, guardians, patients unwilling to return for the follow up study period.
      

Gender

Male

Ages

N/A - N/A

Accepts Healthy Volunteers

No

Contacts

, , 

Location Countries

France

Location Countries

France

Administrative Informations


NCT ID

NCT02333760

Organization ID

GNT-WAS-03


Responsible Party

Sponsor

Study Sponsor

Genethon


Study Sponsor

, , 


Verification Date

May 2021