Genetic: UX701 ( DrugBank: - )
1 disease
| 告示番号 | 疾患名（ページ内リンク） | 臨床試験数 |
|---|---|---|
| 171 | ウィルソン病 | 1 |
171. ウィルソン病
臨床試験数 : 68 / 薬物数 : 79 - (DrugBank : 17) / 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 | NCT04884815 (ClinicalTrials.gov) | September 27, 2021 | 7/5/2021 | Clinical Study of UX701 AAV-Mediated Gene Transfer for the Treatment of Wilson Disease | A Randomized, Double-Blind, Placebo-Controlled, Multicenter, Seamless, Adaptive, Safety, Dose-Finding, and Phase 3 Clinical Study of UX701 AAV-Mediated Gene Transfer for the Treatment of Wilson Disease | Wilson Disease | Genetic: UX701;Other: Placebo;Drug: oral corticosteroids;Drug: Placebo for oral corticosteroids | Ultragenyx Pharmaceutical Inc | NULL | Recruiting | 18 Years | N/A | All | 90 | Phase 1/Phase 2 | United States |