Genetic: scAAV8OTC ( DrugBank: - )
1 disease
| ID | Disease name (Link within this page) | Number of trials |
|---|---|---|
| 251 | Urea cycle disorder | 1 |
251. Urea cycle disorder
Clinical trials : 48 / Drugs : 52 - (DrugBank : 13) / Drug target genes : 4 - Drug target pathways : 29
| 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) | January 2017 | 9/12/2016 | Safety and Dose-Finding Study of DTX301 (scAAV8OTC) in Adults With Late-Onset 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: Oral prednisone | Ultragenyx Pharmaceutical Inc | NULL | Completed | 18 Years | N/A | All | 11 | Phase 1/Phase 2 | United States;Canada;Spain;United Kingdom |