65. 原発性免疫不全症候群 Primary immunodeficiency Clinical trials / Disease details
臨床試験数 : 500 / 薬物数 : 614 - (DrugBank : 119) / 標的遺伝子数 : 92 - 標的パスウェイ数 : 217
| No. | TrialID | Date_ enrollment | Date_ registration | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 1 | NCT03765632 (ClinicalTrials.gov) | January 3, 2018 | 8/8/2018 | Efficacy and Safety of the Cryopreserved Formulation of OTL-101 in Subjects With ADA-SCID | Efficacy and Safety of a Cryopreserved Formulation of Autologous CD34+ Haematopoietic Stem Cells Transduced ex Vivo With Elongation Factor 1a Short Form (EFS) Lentiviral Vector Encoding for Human ADA Gene in Subjects With Severe Combined Immunodeficiency (SCID) Due to Adenosine Deaminase Deficiency | Severe Combined Immunodeficiency Due to ADA Deficiency | Genetic: Infusion of autologous cryopreserved EFS-ADA LV CD34+ cells (OTL-101);Drug: Busulfan;Drug: Peg-Ada | Great Ormond Street Hospital for Children NHS Foundation Trust | Orchard Therapeutics | Completed | 30 Days | 17 Years | All | 13 | Phase 1/Phase 2 | United Kingdom |
| 2 | NCT01380990 (ClinicalTrials.gov) | November 15, 2012 | 23/6/2011 | Lentiviral (LV) Gene Therapy for Adenosine Deaminase (ADA) Deficiency | Phase I/II, Historical Controlled, Open-label, Non-randomised, Single-centre Trial to Assess the Safety and Efficacy of EF1aS-ADA Lentiviral Vector Mediated Gene Modification of Autologous CD34+ Cells From ADA-deficient Individuals | Adenosine Deaminase Deficiency;Severe Combined Immunodeficiencies (SCID) | Genetic: Infusion of autologous EFS-ADA LV CD34+ cells;Other: Haematopoietic Stem Cell Transplantation (HSCT);Drug: Busulfan;Drug: Peg-Ada | Great Ormond Street Hospital for Children NHS Foundation Trust | Orchard Therapeutics | Completed | N/A | 15 Years | All | 36 | Phase 1/Phase 2 | United Kingdom |
| 3 | NCT00794508 (ClinicalTrials.gov) | November 2008 | 19/11/2008 | MND-ADA Transduction of CD34+ Cells From Children With ADA-SCID | MND-ADA Transduction of CD34+ Cells From the Bone Marrow Of Children With Adenosine Deaminase (ADA)-Deficient Severe Combined Immunodeficiency (SCID): Effect of Discontinuation of PEG-ADA and Marrow Cytoreduction With Busulfan | Severe Combined Immunodeficiency | Biological: ADA gene transfer | Donald B. Kohn, M.D. | FDA Office of Orphan Products Development;National Institutes of Health (NIH) | Completed | 1 Month | 18 Years | All | 10 | Phase 2 | United States |