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 | NCT01560182 (ClinicalTrials.gov) | April 9, 2010 | 16/3/2012 | Gene Therapy for Metachromatic Leukodystrophy (MLD) | A Phase I/II Clinical Trial of Hematopoietic Stem Cell Gene Therapy for the Treatment of Metachromatic Leukodystrophy | Lysosomal Storage Disease;Metachromatic Leukodystrophy | Genetic: OTL-200 Gene Therapy | Orchard Therapeutics | Ospedale San Raffaele | Active, not recruiting | N/A | 7 Years | All | 20 | Phase 1/Phase 2 | Italy |