Wiskott-Aldrich Syndrome
Conditions
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.
Interventions
GENETICAutologous 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
Sponsors
Genethon
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
OTHER
Masking
NONE
Eligibility
Sex/Gender
MALE
Healthy volunteers
No
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.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Reconstitution of cell mediated and humoral immunity | yearly from 3 years to 10 years (from 3 years to 5 years for PHA and candida ) | Immunophenotyping panel, whole blood lymphocytes proliferation assays, restoration of antibody production, humoral response to antigene |
| Change in medical conditions | yearly from 3 years to 10 years | Weight and complete clinical exam |
| Key medical events related to WAS | yearly from 3 years to 10 years | Eczema status, infections, bleeding symptoms, autoimmune manifestation |
| Hematological reconstitution | yearly from 3 years to 10 years | CBC including platelets count and size |
| Incidence and type of SAEs | yearly from 3 years to 15 years | Incidence and nature of delayed events such as malignancies, hematologic, autoimmune events, mortality |
| Lentiviral integration sites | yearly from 3 years to 15 years (from 11 to 15 yearly time points, only in case of Advers Events of Special Interest) | Presence of lentiviral integration sites in different cells sub-populations |
| Vector copy numbers | yearly from 3 years to 15 years (from 11 to 15 yearly time points, only in case of Advers Events of Special Interest) | Quantification of vector copy numbers on sorted cells population by q-PCR |
| Replication competent lentivirus (RCL) | yearly from 3 years to 15 years (from 11 to 15 yearly time points, only in case of Advers Events of Special Interest) | Presence of RCL |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Representation of TCR families | yearly from 3 years to 5 years | Representation of TCR families by PCR TREC (TCR excision circle) and TCR V beta panel |
| Bone marrow content | yearly from 3 years to 5 years (optional) | Numbers and type of cells in bone marrow |
| Need for associated treatments | yearly from 3 years to 15 years | Immunoglobulins, antibacterial, antifungal, antiviral drugs, transfusions |
Countries
France, United Kingdom
Outcome results
None listed