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 | NCT05092685 (ClinicalTrials.gov) | February 1, 2022 | 28/9/2021 | Halting Ornithine Transcarbamylase Deficiency With Recombinant AAV in ChildrEn | Phase I/II Open Label, Multicentre Clinical Trial to Assess Safety and Efficacy of AAVLK03hOTC for Paediatric Patients With Ornithine Transcarbamylase Deficiency. | Ornithine Transcarbamylase Deficiency | Genetic: AAVLK03hOTC | University College, London | NULL | Not yet recruiting | N/A | 16 Years | All | 12 | Phase 1/Phase 2 | NULL |