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| Name | Class |
|---|---|
| Great Ormond Street Hospital for Children NHS Foundation Trust | OTHER |
| Institute of Child Health | OTHER |
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This is a phase I/II study to evaluate the safety and efficacy of Hematopoietic Stem Cell genetherapy for the Wiskott-Aldrich Syndrome.
This clinical trial is an ex vivo gene therapy trial. The investigational product corresponds to autologous CD34+ cells transduced with a lentiviral vector harboring the human WASP gene.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| study treatment | Experimental | autologous CD34 positive cells transduced with a lentiviral vector containing the human WAS gene |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Autologous CD34 positive cells transduced with a lentiviral vector containing human WAS gene | Genetic | transplantation of patient's autologous CD34+ cells transduced with lentiviral vector containing human WAS gene |
| Measure | Description | Time Frame |
|---|---|---|
| Improvement in the eczema status | Improvement in the eczema status as compared with the baseline status at study entry on clinical evaluation | 2 years |
| Reduction in the frequency and severity of infection episodes | Reduction in the frequency and severity of infection episodes as compared with the baseline status and the patient's historical data collected over the 2 years prior to study entry | 2 years |
| Reduction in the frequency and severity of bruising and bleeding episodes | Reduction in the frequency and severity of bruising and bleeding episodes as compared with the baseline status and the patient's historical data collected over the 2 years prior to study entry | 2 years |
| Reduction in the frequency and severity of autoimmune disorders | Reduction in the frequency and severity of autoimmune disorders as compared with the baseline status at study entry | 2 years |
| Reduction in the number of disease related days of hospitalization | Reduction in the number of disease related days of hospitalization as compared with the patient's historical data collected over the 2 years prior to study entry | 2 years |
| Measure | Description | Time Frame |
|---|---|---|
| Occurrence and type of adverse events | Occurrence and type of adverse events reported during the course of the study | 2 years |
| Change in medical conditions | Assessment of weight, vital signs, ECG and laboratory exams during the course of the study |
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Inclusion Criteria:
Exclusion Criteria:
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Great Ormond Street Hospital | London | WC1N 1EH | United Kingdom | |||
| Royal Free Hospital |
| 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. | |
| 28716862 |
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| ID | Term |
|---|---|
| D014923 | Wiskott-Aldrich Syndrome |
| D000081207 | Primary Immunodeficiency Diseases |
| ID | Term |
|---|---|
| D025861 | Blood Coagulation Disorders, Inherited |
| D001778 | Blood Coagulation Disorders |
| D006402 | Hematologic Diseases |
| D006425 | Hemic and Lymphatic Diseases |
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| 2 years |
| Safety of lentivirus gene transfer into Hematopoietic Stem Cells | Detection of replication competent lentivirus (RCL) and lentivirus integration sites analysis | 3, 6, 12, 24 months / 6, 12, 18, 24 months |
| Improvement of microthrombocytopenia | Improvement of microthrombocytopenia as compared with the baseline evaluation at study entry | 3, 6, 12, 24 months |
| Decrease in the number and volume of platelets transfusions | Decrease in the number and volume of platelets transfusions as compared with patient's historical data collected over the 2 years prior to study entry | 2 years |
| Evidence of sustained engrafment of WASP-expressing transduced cells | Quantification of vector copy numbers and detection of vector-derived WASP expression | 6 weeks, 1, 3, 6, 9, 12, 18 & 24 months |
| Reconstitution of humoral and cell mediated immunity | Reconstitution of humoral and cell mediated immunity as compared with the baseline evaluation at study entry | 9, 12, 18 & 24 months |
| London |
| WC1N 1EH |
| United Kingdom |
| Derived |
| Morris EC, Fox T, Chakraverty R, Tendeiro R, Snell K, Rivat C, Grace S, Gilmour K, Workman S, Buckland K, Butler K, Chee R, Salama AD, Ibrahim H, Hara H, Duret C, Mavilio F, Male F, Bushman FD, Galy A, Burns SO, Gaspar HB, Thrasher AJ. Gene therapy for Wiskott-Aldrich syndrome in a severely affected adult. Blood. 2017 Sep 14;130(11):1327-1335. doi: 10.1182/blood-2017-04-777136. Epub 2017 Jul 17. |
| 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 |
| D007153 | Immunologic Deficiency Syndromes |
| D007154 | Immune System Diseases |