19. ライソゾーム病 Lysosomal storage disease Clinical trials / Disease details
臨床試験数 : 899 / 薬物数 : 684 - (DrugBank : 99) / 標的遺伝子数 : 51 - 標的パスウェイ数 : 182
| 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 | NCT00004294 (ClinicalTrials.gov) | November 1999 | 18/10/1999 | Phase I Study of Retrovirally Mediated Transfer of the Human Glucocerebrosidase Gene Into Peripheral Blood Stem Cells for Autologous Transplantation in Patients With Type I Gaucher Disease | Gaucher's Disease | Genetic: human glucocerebrosidase gene into autologous peripheral blood stem cells | National Center for Research Resources (NCRR) | National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK);University of Pittsburgh | Completed | 18 Years | 65 Years | Both | 15 | Phase 1 | United States |