251. 尿素サイクル異常症 Urea cycle disorder Clinical trials / Disease details
臨床試験数 : 54 / 薬物数 : 61 - (DrugBank : 15) / 標的遺伝子数 : 3 - 標的パスウェイ数 : 28
| 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 | NCT02991144 (ClinicalTrials.gov) | July 31, 2017 | 9/12/2016 | Safety and Dose-Finding Study of DTX301 (scAAV8OTC) in Adults With Late-Onset Ornithine Transcarbamylase (OTC) Deficiency | A Phase 1/2, Open-Label Safety and Dose-Finding Study of Adeno-Associated Virus (AAV) Serotype 8 (AAV8)-Mediated Gene Transfer of Human Ornithine Transcarbamylase (OTC) in Adults With Late-Onset OTC Deficiency | Ornithine Transcarbamylase (OTC) Deficiency | Genetic: scAAV8OTC;Drug: Reactive Corticosteroid Taper Regimen;Drug: Prophylactic Corticosteroid Taper Regimen | Ultragenyx Pharmaceutical Inc | NULL | Completed | 18 Years | N/A | All | 16 | Phase 1/Phase 2 | United States;Canada;Spain;United Kingdom |