Wiskott-Aldrich Syndrome Clinical Trial
— WASFUPOfficial 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).
Verified date | May 2021 |
Source | Genethon |
Contact | n/a |
Is FDA regulated | No |
Health authority | |
Study type | Interventional |
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.
Status | Active, not recruiting |
Enrollment | 10 |
Est. completion date | October 2032 |
Est. primary completion date | October 2032 |
Accepts healthy volunteers | No |
Gender | Male |
Age group | N/A and older |
Eligibility | 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. |
Country | Name | City | State |
---|---|---|---|
France | Hopital Necker - Enfants Malades | Paris | |
United Kingdom | UCL Institute of Child Health | London |
Lead Sponsor | Collaborator |
---|---|
Genethon |
France, United Kingdom,
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Primary | Incidence and type of SAEs | Incidence and nature of delayed events such as malignancies, hematologic, autoimmune events, mortality | yearly from 3 years to 15 years | |
Primary | Lentiviral integration sites | Presence of lentiviral integration sites in different cells sub-populations | yearly from 3 years to 15 years (from 11 to 15 yearly time points, only in case of Advers Events of Special Interest) | |
Primary | Vector copy numbers | Quantification of vector copy numbers on sorted cells population by q-PCR | yearly from 3 years to 15 years (from 11 to 15 yearly time points, only in case of Advers Events of Special Interest) | |
Primary | Replication competent lentivirus (RCL) | Presence of RCL | yearly from 3 years to 15 years (from 11 to 15 yearly time points, only in case of Advers Events of Special Interest) | |
Primary | Change in medical conditions | Weight and complete clinical exam | yearly from 3 years to 10 years | |
Primary | Key medical events related to WAS | Eczema status, infections, bleeding symptoms, autoimmune manifestation | yearly from 3 years to 10 years | |
Primary | Hematological reconstitution | CBC including platelets count and size | yearly from 3 years to 10 years | |
Primary | Reconstitution of cell mediated and humoral immunity | Immunophenotyping panel, whole blood lymphocytes proliferation assays, restoration of antibody production, humoral response to antigene | yearly from 3 years to 10 years (from 3 years to 5 years for PHA and candida ) | |
Secondary | Need for associated treatments | Immunoglobulins, antibacterial, antifungal, antiviral drugs, transfusions | yearly from 3 years to 15 years | |
Secondary | Representation of TCR families | Representation of TCR families by PCR TREC (TCR excision circle) and TCR V beta panel | yearly from 3 years to 5 years | |
Secondary | Bone marrow content | Numbers and type of cells in bone marrow | yearly from 3 years to 5 years (optional) |
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