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Long Term Safety Follow up of Haematopoietic Stem Cell Gene Therapy for the Wiskott Aldrich Syndrome
Sponsor: Genethon
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.
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).
Key Details
Gender
MALE
Age Range
Any - Any
Study Type
INTERVENTIONAL
Enrollment
10
Start Date
2014-09
Completion Date
2032-10
Last Updated
2021-06-03
Healthy Volunteers
No
Conditions
Interventions
Autologous CD34+ cells transduced with WASP lentiviral vector
Follow up of ex vivo gene therapy transplantation of patient's autologous CD34+ cells transduced with lentiviral vector containing human WASP gene
Locations (2)
Hopital Necker - Enfants Malades
Paris, France
UCL Institute of Child Health
London, United Kingdom