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 Type
INTERVENTIONAL
Allocation
NA
Purpose
OTHER
Masking
NONE
Enrollment
10
Follow up of ex vivo gene therapy transplantation of patient's autologous CD34+ cells transduced with lentiviral vector containing human WASP gene
Hopital Necker - Enfants Malades
Paris, France
UCL Institute of Child Health
London, United Kingdom
Incidence and type of SAEs
Incidence and nature of delayed events such as malignancies, hematologic, autoimmune events, mortality
Time frame: yearly from 3 years to 15 years
Lentiviral integration sites
Presence of lentiviral integration sites in different cells sub-populations
Time frame: yearly from 3 years to 15 years (from 11 to 15 yearly time points, only in case of Advers Events of Special Interest)
Vector copy numbers
Quantification of vector copy numbers on sorted cells population by q-PCR
Time frame: yearly from 3 years to 15 years (from 11 to 15 yearly time points, only in case of Advers Events of Special Interest)
Replication competent lentivirus (RCL)
Presence of RCL
Time frame: yearly from 3 years to 15 years (from 11 to 15 yearly time points, only in case of Advers Events of Special Interest)
Change in medical conditions
Weight and complete clinical exam
Time frame: yearly from 3 years to 10 years
Key medical events related to WAS
Eczema status, infections, bleeding symptoms, autoimmune manifestation
Time frame: yearly from 3 years to 10 years
Hematological reconstitution
CBC including platelets count and size
Time frame: yearly from 3 years to 10 years
Reconstitution of cell mediated and humoral immunity
Immunophenotyping panel, whole blood lymphocytes proliferation assays, restoration of antibody production, humoral response to antigene
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Time frame: yearly from 3 years to 10 years (from 3 years to 5 years for PHA and candida )
Need for associated treatments
Immunoglobulins, antibacterial, antifungal, antiviral drugs, transfusions
Time frame: yearly from 3 years to 15 years
Representation of TCR families
Representation of TCR families by PCR TREC (TCR excision circle) and TCR V beta panel
Time frame: yearly from 3 years to 5 years
Bone marrow content
Numbers and type of cells in bone marrow
Time frame: yearly from 3 years to 5 years (optional)