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 | NCT02021266 (ClinicalTrials.gov) | April 2013 | 19/12/2013 | Single Patient Expanded Access Protocol: Metabolic Boost | Single Patient Expanded Access Protocol: Phase I/II Pilot Study of Mixed Chimerism to Treat Inherited Metabolic Disorders | Metachromatic Leukodystrophy | Biological: Enriched Hematopoetic Stem Cell Infusion | Talaris Therapeutics Inc. | Duke University | No longer available | N/A | N/A | Female | Phase 1/2 | United States |