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Autologous cd34+cells transduced with the w1.6_hwasp_wpre (vsvg) lentiviral vector    (DrugBank: -)

1 disease
告示番号疾患名（ページ内リンク）臨床試験数
65原発性免疫不全症候群2

65. 原発性免疫不全症候群　［臨床試験数：413，薬物数：581（DrugBank：97），標的遺伝子数：68，標的パスウェイ数：202］
Searched query = "Primary immunodeficiency", "X-SCID", "Reticular dysgenesis", "Adenosine deaminase deficiency", "Omenn syndrome", "Purine nucleoside phosphorylase deficiency", "CD8 deficiency", "ZAP-70 deficiency", "MHC class I deficiency", "MHC class II deficiency", "Combined immunodeficiency", "Wiskott-Aldrich syndrome", "Telangiectasia ataxia", "Nijmegen breakage syndrome", "Bloom syndrome", "Immunodeficiency, centromere region instability, facial anomalies syndrome", "ICF syndrome", "PMS2 deficiency", "Radiosensitivity, immunodeficiency, dysmorphic features, and learning difficulties syndrome", "RIDDLE syndrome", "Schimke syndrome", "Netherton syndrome", "Thymic hypoplasia", "DiGeorge syndrome", "22q11.2 deletion syndrome", "Hyper-IgE syndrome", "Hepatic venoocclusive immunodeficiency", "Immunodeficiency with central hepatic vein atresia", "Dyskeratosis congenita", "X-linked agammaglobulinaemia", "Common variable immunodeficiency", "Hyper-IgM syndrome", "Isolated IgG subclass deficiency", "Selective IgA deficiency", "Specific antibody production deficiency", "Infant transient hypogammaglobulinemia", "Chédiak-Higashi syndrome", "Chediak-Higashi syndrome", "X-linked lymphoproliferative syndrome", "SAP deficiency", "SH2D1A/SLAM-associated protein deficiency", "XIAP deficiency", "X-linked inhibitor of apoptosis deficiency", "Autoimmune lymphoproliferative syndrome", "ALPS", "Familial hemophagocytic syndrome", "Perforin deficiency", "Munc13-4 deficiency", "Syntaxin 11 deficiency", "Munc18-2 deficiency", "Autoimmune polyendocrinopathy-candidiasis-ectodermal dystrophy", "APECED", "Immune dysregulation, polyendocrinopathy, enteropathy, X-linked syndrome", "IPEX syndrome", "CD25 deficiency", "ITCH deficiency", "Primary phagocytic dysfunction", "Severe congenital neutropenia", "Cyclic neutropenia", "Hermanskyi-Pudlak syndrome type 2", "Hermanskyi-Pudlak syndrome 2", "Griscelli syndrome type 2", "Griscelli syndrome 2", "p14 deficiency", "Warts, hypogammaglobulinemia, infections, myelokathexis syndrome", "WHIM syndrome", "Glycogen storage disease type Ib", "Leukocyte adhesion deficiency", "Shwachman-Diamond syndrome", "Chronic granulomatous disease", "Myeloperoxidase deficiency", "Mendelian susceptibility to mycobacterial disease", "MSMD", "Anhidrotic ectodermal dysplasia with immunodeficiency", "EDA-ID", "Interleukin-1 receptor-associated kinase-4 deficiency", "IRAK4 deficiency", "IMyD88 deficiency", "Chronic mucocutaneous candidiasis", "Epidermodysplasia verruciformis", "Herpes simplex encephalitis", "Caspase recruitment domain family member 9 deficiency", "CARD9 deficiency", "Trypanosomiasis", "Congenital complement deficiency", "C1q deficiency", "CC1r deficiency", "CC1s deficiency", "CC2 deficiency", "CC3 deficiency", "CC4 deficiency", "CC5 deficiency", "CC6 deficiency", "CC7 deficiency", "CC8 deficiency", "CC9 deficiency", "Factor D deficiency", "Properdin deficiency", "Factor I deficiency", "Factor H deficiency", "MASP1 deficiency", "3MC syndrome", "Mannose-binding protein-associated serine protease 2 deficiency", "MASP2 deficiency", "FCN3", "Hereditary angioedema type 1", "Hereditary angioedema type I", "C1 inhibitor deficiency type 1", "C1 inhibitor deficiency type I", "Hereditary angioedema type 2", "Hereditary angioedema type II", "C1 inhibitor deficiency type 2", "C1 inhibitor deficiency type II", "Hereditary angioedema type 3", "Hereditary angioedema type III", "C1 inhibitor deficiency type 3", "C1 inhibitor deficiency type III"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: 03/15/2021. Trials are sorted by Date_enrollment from most recent to oldest in the table.
2 / 413 trials found
No.TrialIDDate_
enrollment		Date_
registration		Public_titleScientific_titleConditionInterventionPrimary_
sponsor		Secondary_
sponsor		Recruitment_
Status		Inclusion_
agemin		Inclusion_
agemax		Inclusion_
gender		Target_
size		PhaseCountries
1EUCTR2014-000274-20-GB
(EUCTR)		14/05/201412/03/2014Long-term follow-up of the WAS gene therapy studyLONG 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(GTG 002-07 AND GTG 003-08) - Long-term follow-up of the WAS gene therapy study, version 2.0 Wiskott-Aldrich syndrome (WAS) is a rare X-linked immunodeficiencycaused by mutations in a single gene ,the Wiskott-Aldrich SyndromeProtein (WASP). WAS is characterised by micro-thrombocytopenia,recurrent infections,eczema and associated with a high incidence ofauto-immunity and of lymphoid malignancies. Over 150 uniquemutations in the WAS gene have been identified.Loss-of-functionmutations in this gene have widespread consequences on hematopoieticlineages.
MedDRA version: 19.1;Level: PT;Classification code 10047992;Term: Wiskott-Aldrich syndrome;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]		Product Name: Autologous CD34+cells transduced with the w1.6_hWASP_WPRE (VSVg) lentiviral vector
Other descriptive name: AUTOLOGOUS CD34+CELLS TRANSDUCED WITH THE W1.6_HWASP_WPRE (VSVG) LENTIVIRAL VECTOR		GenethonNULLAuthorised-recruitment may be ongoing or finishedFemale: no
Male: yes		10Phase 2United Kingdom
2EUCTR2007-004308-11-GB
(EUCTR)		11/01/201006/07/2009Gene therapy for WASPHASE I/II CLINICAL TRIAL OF HAEMATOPOIETIC STEM CELL GENE THERAPYFOR THE WISKOTT-ALDRICH SYNDROME - Gene Therapy for WAS , version 5.0 Wiskott-Aldrich syndrome (WAS) is a rare X-linked immunodeficiency caused by mutations in a single gene ,the Wiskott-Aldrich Syndrome Protein (WASP). WAS is characterised by micro-thrombocytopenia, recurrent infections,eczema and associated with a high incidence of auto-immunity and of lymphoid malignancies. Over 150 unique mutations in the WAS gene have been identified.Loss-of-function mutations in this gene have widespread consequences on hematopoietic lineages.
MedDRA version: 19.1;Level: PT;Classification code 10047992;Term: Wiskott-Aldrich syndrome;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]		Product Name: Autologous CD34+cells transduced with the w1.6_hWASP_WPRE (VSVg) lentiviral vector
Other descriptive name: AUTOLOGOUS CD34+CELLS TRANSDUCED WITH THE W1.6_HWASP_WPRE (VSVG) LENTIVIRAL VECTOR
Product Name: Autologous CD34+cells transduced with the w1.6_hWASP_WPRE (VSVg) lentiviral vector
Other descriptive name: AUTOLOGOUS CD34+CELLS TRANSDUCED WITH THE W1.6_HWASP_WPRE (VSVG) LENTIVIRAL VECTOR
Product Name: Autologous CD34+cells transduced with the w1.6_hWASP_WPRE (VSVg) lentiviral vector
Other descriptive name: AUTOLOGOUS CD34+CELLS TRANSDUCED WITH THE W1.6_HWASP_WPRE (VSVG) LENTIVIRAL VECTOR		GenethonNULLAuthorised-recruitment may be ongoing or finishedFemale: no
Male: yes		Phase 1;Phase 2United Kingdom