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 |
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1 | NCT05422482 (ClinicalTrials.gov) | September 20, 2022 | 31/5/2022 | A Study to Evaluate the Safety, Tolerability, PK and PD of Intracerebroventricular GC1123 in Patients With MPS ? | Phase 1, Open-Label, Ascending Dose Study to Evaluate the Safety, Tolerability, PK and PD of Intracerebroventricular GC1123 in Patients With MPS ? Who Have Central Nervous System Involvement and Are Receiving Treatment With Intravenous Drug | Mucopolysaccharidosis II;Hunter Syndrome | Biological: GC1123 | GC Biopharma Corp | NULL | Recruiting | 18 Months | 18 Years | All | 12 | Phase 1 | Korea, Republic of |