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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.
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| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Autologous CD34+ cells transduced with WASP lentiviral vector | Genetic | Follow up of ex vivo gene therapy transplantation of patient's autologous CD34+ cells transduced with lentiviral vector containing human WASP gene |
| Measure | Description | Time Frame |
|---|---|---|
| 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 |
| 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) |
| 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) |
| 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) |
| Change in medical conditions | Weight and complete clinical exam | yearly from 3 years to 10 years |
| Key medical events related to WAS | Eczema status, infections, bleeding symptoms, autoimmune manifestation | yearly from 3 years to 10 years |
| Hematological reconstitution | CBC including platelets count and size | yearly from 3 years to 10 years |
| Measure | Description | Time Frame |
|---|---|---|
| Need for associated treatments | Immunoglobulins, antibacterial, antifungal, antiviral drugs, transfusions | 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 |
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Inclusion Criteria:
Exclusion Criteria:
• Parents, guardians, patients unwilling to return for the follow up study period.
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Hopital Necker - Enfants Malades | Paris | 75743 | France | |||
| UCL Institute of Child Health |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 35075289 | Derived | Magnani A, Semeraro M, Adam F, Booth C, Dupre L, Morris EC, Gabrion A, Roudaut C, Borgel D, Toubert A, Clave E, Abdo C, Gorochov G, Petermann R, Guiot M, Miyara M, Moshous D, Magrin E, Denis A, Suarez F, Lagresle C, Roche AM, Everett J, Trinquand A, Guisset M, Bayford JX, Hacein-Bey-Abina S, Kauskot A, Elfeky R, Rivat C, Abbas S, Gaspar HB, Macintyre E, Picard C, Bushman FD, Galy A, Fischer A, Six E, Thrasher AJ, Cavazzana M. Long-term safety and efficacy of lentiviral hematopoietic stem/progenitor cell gene therapy for Wiskott-Aldrich syndrome. Nat Med. 2022 Jan;28(1):71-80. doi: 10.1038/s41591-021-01641-x. Epub 2022 Jan 24. |
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| ID | Term |
|---|---|
| D014923 | Wiskott-Aldrich Syndrome |
| ID | Term |
|---|---|
| D025861 | Blood Coagulation Disorders, Inherited |
| D001778 | Blood Coagulation Disorders |
| D006402 | Hematologic Diseases |
| D006425 | Hemic and Lymphatic Diseases |
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| 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 ) |
| yearly from 3 years to 5 years |
| Bone marrow content | Numbers and type of cells in bone marrow | yearly from 3 years to 5 years (optional) |
| London |
| WC1N 1EH |
| United Kingdom |
| D008231 | Lymphopenia |
| D007970 | Leukopenia |
| D000095542 | Cytopenia |
| D006474 | Hemorrhagic Disorders |
| D007960 | Leukocyte Disorders |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D040181 | Genetic Diseases, X-Linked |
| D000081207 | Primary Immunodeficiency Diseases |
| D007153 | Immunologic Deficiency Syndromes |
| D007154 | Immune System Diseases |