19. Lysosomal storage disease Clinical trials / Disease details
Clinical trials : 899 / Drugs : 684 - (DrugBank : 99) / Drug target genes : 51 - Drug target pathways : 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 | JPRN-UMIN000009758 | 2013/06/24 | 15/01/2013 | Development of treosulfan-based conditioning regimen for congenital metabolic diseases; Phase I study | Development of treosulfan-based conditioning regimen for congenital metabolic diseases; Phase I study - Development of treosulfan-based conditioning regimen for congenital metabolic diseases; Phase I study | Mucopolysaccharidosis type I (Herler syndrome), type II (Hunter syndrome) | Treosulfan 14 g/m2, intravenous, day -6 -5 -4 | Tokai University School of Medicine | 1. School of Human Health Science Faculty of Medicine Kyoto University2. Department of Pediatrics, Japanese Red cross Nagoya Daiichi Hospital3. Department of Pediatrics, Nihon University | Complete: follow-up complete | Not applicable | Not applicable | Male and Female | 12 | Phase 1 | Japan |